Campus Connections

New Connections (Posted February 1, 2012)...

Scottish Medical Charity and International Drug Consortium Form Partnership

Developing World Health (DWH), a leading medical charity based in Stirlingshire, Scotland and committed to developing effective treatments for neglected tropical diseases (NTDs), has signed a collaboration agreement with the internationally respected Consortium for Parasitic Drug Development (CPDD), based at the University of North Carolina (UNC) at Chapel Hill. The collaboration agreement to develop new drugs is believed to be the first between a U.K. medical charity and international scientific consortium focused on NTDs. Dr. Rick Tidwell, director of CPDD, commented: "This is a very welcome and exciting strategic partnership and brings together additional expertise to increase funding and collaboration opportunities for the consortium and enhance the rapid development of novel treatments for NTDs such as African sleeping sickness and leishmaniasis." The partnership means DWH and CPDD can effectively source funding from commercial, government, and not-for-profit organizations to develop and/or improve effective treatments for preventable, treatable diseases like leishmaniasis, African trypanosomiasis (sleeping sickness), Chagas disease (American trypanosomiasis), and dengue. The partnership enables the charity to bridge the "missing link" between academics requiring research funding and the pharmaceutical industry interested in tackling these devastating tropical diseases. The CPDD brings together some of the world's top experts in drug development and delivery from UNC-Chapel Hill, Georgia State University, the University of Glasgow, Ohio State University, the Swiss Tropical Institute, and the Kenya Trypanosomiasis Research Institute, among others. Source: EurekAlert! 1/26/12

Prostate Cancer Study Proves Drug Delays Disease Progression

For men diagnosed with low-risk, localized prostate cancer, being treated with the drug dutasteride (Avodart) delays disease progression and initiating active treatment, and also reduces anxiety, show the results of a three-year international clinical trial led by Dr. Neil Fleshner, head of the Division of Urology, University Health Network (UHN). The findings were published online January 24 in The Lancet. “The results prove that using active surveillance plus dutasteride is a viable, safe and effective treatment option for men who often undergo aggressive local treatment despite low risk of dying from the disease,” says Fleshner, a surgical oncologist in UHN’s Princess Margaret Cancer Program and Professor of Surgery at the University of Toronto. “This is very good news for men with low-risk disease because aggressive treatment can have a major impact on their quality of life, with risks of impotence and incontinence.” The three-year clinical trial enrolled 302 men between the ages of 48 and 82 diagnosed with low-risk localized prostate cancer and regularly monitored for clinical changes--a treatment option called “active surveillance.” In the trial, participants were randomized 1:1 to receive dutasteride or a matching placebo daily. The men also underwent biopsies at 18 months and three years. The study showed a significant delay in disease progression in the men treated with dutasteride, and the final biopsies showed the men treated with the drug were less likely to have cancer detected. The clinical research was funded by GlaxoSmithKline. Source: Newswise 1/24/12

Earlier Connections...

University of Kentucky Offers Stroke Stem Cell Trial

The University of Kentucky will be the first site in the state and one of a select few in the entire country participating in the first stages of a groundbreaking study to investigate the effects of MultiStem, a human adult stem cell product, on patients with acute ischemic stroke. The Phase II clinical trial, known as Atherys stroke protocol B01-02, was approved recently by the university's institutional review board. Source: EurekAlert! 1/13/12

Combination Therapy May Improve Prostate Cancer Survival

Thomas Jefferson University Hospital's Kimmel Cancer Center has started a Phase I clinical trial investigating the latest prostate cancer chemotherapy drug to extend survival, cabazitaxel, in combination with radiation and hormone therapy. This first-of-its-kind multimodality approach could improve disease control and eventually survival for locally advanced prostate cancer patients. Source: EurekAlert! 1/12/12

Conventional Nerve Repair Wisdom Defied in Donated Nerve Tissue Study

In the first-ever multicenter clinical trial on processed nerve allograft, researchers from the Buncke Clinic in San Francisco, Calif. found that treatment of severed peripheral nerve with processed nerve allograft showed meaningful recovery in 87 percent of patients, comparing favorably to traditional nerve repair (autograft nerve). The study findings were published in the January 2012 issue of Microsurgery. Source: PRNewswire 1/12/12

Parkinson's Treatment Shows Positive Results in Clinical Testing

Researchers from the University of Florida and 14 additional medical centers reported results in January in the online version of The Lancet Neurology indicating that deep brain stimulation (DBS) is effective at improving motor symptoms and quality of life in patients with advanced Parkinson's disease. The study, sponsored by St. Jude Medical Inc., tested the safety and effectiveness of a constant current DBS device developed by St. Jude Medical to manage the symptoms of Parkinson's disease. Source: EurekAlert! 1/11/12

UCSF, Sanofi Collaborate in Search of New Diabetes Cures

The University of California, San Francisco (UCSF) has signed an alliance with international pharmaceutical company Sanofi to share expertise in diabetes research and identify drug targets that could lead to new therapies for both type 1 and type 2 diabetes. The $3.1 million collaboration will bring together scientists in three UCSF labs with deep understanding of the biology of beta cells with Sanofi researchers who are experienced in developing potential drug candidates into actual therapies. Source: EurekAlert! 1/10/12

Kessler Foundation and USC Team Up on Disability Research

Kessler Foundation and the University of Southern California (USC) Institute for Creative Technologies will collaborate on clinical research projects applying virtual reality technology to cognitive and motor rehabilitation research. The goal is to conduct research to develop the evidence base to support the future of home-based rehabilitation that is effective, convenient, and affordable. Source: EurekAlert! 1/10/12

Researchers Trial New Device that May Support Improved Newborn Health

Despite the numerous medical advances that happen every day, the infant mortality rate in the United States is still higher than in most European countries. While experts believe this is closely linked to the growing rate of preterm births, researchers are committed to finding ways to make labor and delivery safer. Northwestern University researchers are examining a new device that may support improved newborn health at delivery through closer monitoring of infant oxygen use during labor. "Poor birth outcomes are often directly related to loss of oxygen during labor and delivery," explained Alan Peaceman, MD, the chief of maternal fetal medicine and professor of obstetrics and gynecology who is the lead investigator on the study. "Through more advanced monitoring, we hope to identify red flags sooner and prevent dips in oxygen that may lead to long-term health issues for the baby." STAN™ is fetal heart rate monitoring system that measures and tracks the electrical activity of the baby's heart via an internal electrode, along with uterine contractions and how well the baby uses oxygen during labor. It then interprets the data and signals clinicians when a significant change in oxygen levels or heart rate occurs. The monitor is approved by the Food and Drug Administration and is routinely used in Europe, but has not been widely adopted in the United States yet. At present, it is only available for patients enrolled in the study. If this study demonstrates improved outcomes, it is likely that the monitoring system will become more widely available to mothers giving birth in this country. Northwestern is one of 14 centers in the United States participating in the clinical trial, which researchers hope will enroll 11,000 women from across the country over the next three years. Subjects who opt to participate in this research study are randomly assigned to one of two groups. One will receive the standard fetal monitoring offered today, and the other will be monitored using the new system in addition to the existing method. The way in which fetal heart rate is monitored will be the only change to the labor and delivery experience, which will continue to be managed by the woman's doctor. Source: EurekAlert! 12/21/11

Human Research Protection Award Recipients Announced

Awards for Excellence in Human Research Protection for 2011 were announced in December by Dr. Peter G. Goldschmidt, president and founder of the Health Improvement Institute. The winner of the Annual Award for Best Practice was: Harvard Catalyst | The Harvard Clinical and Translational Science Center, Harvard Medical School (Boston, Mass.) for its best practice, Harvard Catalyst Master Common Reciprocal Institutional Review Board (IRB) Reliance Agreement. The agreement supports a framework that smoothes the review of proposed multicenter human studies by reducing the administrative burden on IRBs, increasing the efficiency of review, and, when utilized, facilitates best practices for research participants by preventing disparities among protocols and informed consent forms that often occur in multi-IRB reviews. The recipients of Awards of Excellence for Best Practice included Cedars-Sinai Medical Center (Beverly Hills, Calif.), for IRB educational initiatives; Mount Sinai School of Medicine (New York, N.Y.), for flexible IRB; Medical Research Management, Inc./CRA Solutions, Inc. (Coral Springs, Fla.), for monitoring; and the Quality Assurance Office for Clinical Trials at Dana-Farber/Harvard Cancer Center, Dana-Farber Cancer Institute (Boston, Mass.), for audit team. Awards are given for demonstrated excellence in promoting the well being of people who participate in research. The Health Improvement Institute created the award program. This year's judges were drawn from academic, compliance, consulting, health services, legal, and research review organizations. Source: PRNewswire 12/20/11

HIV/AIDS Vaccine Proceeding to Clinical Trials

The first and only preventative HIV vaccine based on a genetically modified killed whole virus has received approval by the United States Food and Drug Administration (FDA) to start human clinical trials. Developed by Dr. Chil-Yong Kang and his team at the University of Western Ontario, with the support of Sumagen Canada, the vaccine (SAV001) holds tremendous promise, having already proven to stimulate strong immune responses in preliminary toxicology tests with no adverse effects or safety risks. It is the only HIV vaccine currently under development in Canada, and one of only a few in the world. Since the virus was characterized in 1983, there have been numerous trials through pharmaceutical companies and academic institutions around the world to develop vaccines; however, no commercialized vaccine has been developed to date. Other HIV vaccines evaluated through human clinical trials have focused on either one specific component of HIV as an antigen, genetic vaccine using recombinant DNA, or recombinant viruses carrying the HIV genes. Kang's vaccine is unique in that it uses a killed whole HIV-1, much like the killed whole virus vaccines for polio, influenza, rabies, and hepatitis A. The HIV-1 is genetically engineered so it is nonpathogenic and can be produced in large quantities. Before it can be commercialized, the SAV001 vaccine must go through three phases of human clinical trials: Phase I, set to begin in January 2012, will double check the safety of the vaccine in humans, involving only 40 HIV-positive volunteers; Phase II will measure immune responses in humans, involving approximately 600 HIV-negative volunteers who are in the high-risk category for HIV infection; and Phase III will measure the efficacy of the vaccine, involving approximately 6,000 HIV-negative volunteers who are also in the high-risk category for HIV infection. Source: EurekAlert! 12/20/11

Discovery Could Significantly Change Biomedical Research

In a major step that could revolutionize biomedical research, scientists have discovered a way to keep normal cells as well as tumor cells taken from an individual cancer patient alive in the laboratory, which previously had not been possible. Normal cells usually die in the lab after dividing only a few times, and many common cancers will not grow, unaltered, outside the body. This new technique, described online in the American Journal of Pathology, could be the critical advance that ushers in a new era of personalized cancer medicine, and has potential application in regenerative medicine, says the study's senior investigator, Richard Schlegel, MD, PhD, chairman of the department of pathology at Georgetown Lombardi Comprehensive Cancer Center, a part of Georgetown University Medical Center. "Because every tumor is unique, this advance will make it possible for an oncologist to find the right therapies that both kills a patient's cancer and spares normal cells from toxicity," he says. "We can test resistance as well chemosensitivity to single or combination therapies directly on the cancer cell itself." The research team, which also includes several scientists from the National Institutes of Health (NIH), found that adding two different substances to cancer and normal cells in a laboratory pushes them to morph into stem-like cells--adult cells from which other cells are made. The two substances are a Rho kinase (ROCK) inhibitor and fibroblast feeder cells. ROCK inhibitors help stop cell movement, but it is unclear why this agent turns on stem cell attributes, Schlegel says. "We tried breast cells and they grew well. We tried prostate cells and their growth was fantastic, which is amazing because it is normally impossible to grow these cells in the lab," Schlegel says. "We found the same thing with lung and colon cells that have always been difficult to grow. In short, we discovered we can grow normal and tumor cells from the same patient forever, and nobody has been able to do that." The ability to immortalize cancer cells will also make biobanking both viable and relevant, Schlegel says. The researchers further discovered that the stem-like behavior in these cells is reversible. Withdrawing the ROCK inhibitor forces the cells to differentiate into the adult cells that they were initially. This "conditional immortalization" could help advance the field of regenerative medicine, Schlegel says. The research was funded by grants from the NIH, Department of Defense fellowship funding, and an internal grant from Georgetown Lombardi's Cancer Center Support Grant from the National Cancer Institute. Source: EurekAlert! 12/19/11

Ruxolinitib Better at Reducing Myelofibrosis Symptoms

In a major advance in treatment, a multicenter study found that ruxolinitib did a better job than off-label chemotherapy drugs reducing the terrible symptoms associated with myelofibrosis, including pain, enlarged spleen, anemia, fever, chills, fatigue, and weight loss. The results were presented in December at the annual meeting of the American Society of Hematology and Oncology in San Diego, Calif. Myelofibrosis is a bone marrow disorder that disrupts the body's normal production of blood cells, resulting in extensive scarring in the bone marrow. Patients tend to be over age 50. "For years, researchers have struggled to find effective medications to help patients struggling with symptoms of this chronic pre-leukemia condition," says lead investigator Ruben Mesa, MD, of Mayo Clinic in Arizona. There are several thousand new myelofibrosis patients in the U.S. every year. Only about 10 percent of myelofibrosis patients are eligible for a bone marrow transplant and chemotherapy often falls short, Mesa says. A handful of off-label chemotherapy drugs have been modestly helpful, he says. A randomized, double-blind clinical trial, known as the COMFORT-1 study, showed that ruxolinitib reduced spleen size by more than 35 percent in almost all of the 154 patients studied. An enlarged spleen, caused by sequestered over-proliferating blood cells, causes discomfort and can also lead to the need for blood transfusions and further medical complications for patients. In the study, patients treated with ruxolinitib also reported less severe symptoms than those reported by patients treated with a placebo. A tandem randomized investigation in Europe, known as the COMFORT-2 study, showed that ruxolinitib also was more effective at reducing symptoms than the best available chemotherapies--specifically, the off-label drugs doctors have been offering. Most surprising for researchers, however, was the ultimate comparison between the two clinical trials. "When we looked at the control arms of the two studies, we found that the best available chemotherapies we've been using up to this time are no more effective than a placebo," Mesa says. The comparison provided a wake-up call, he adds. "This was a unique piece of information we hadn't had in the past, and it offers further incentive and pressure to find better therapies for patients." Funding for the study was supported by Incyte and Novartis. Source: EurekAlert! 12/12/11

Neuroscience Clinical Trials Center Could Bring Treatments to Patients Faster

In a development that could pave the way for treatment for rare neurological diseases and clues to more common ones, physician-scientists at New York's Albert Einstein College of Medicine of Yeshiva University and at Montefiore Medical Center, the university hospital for the college, have secured a grant to establish a clinical site for the Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT). One of only 25 such federally funded centers in the country, the Einstein-Montefiore site was created in partnership with Einstein affiliates Beth Israel Medical Center in Manhattan and the North Shore-Long Island Jewish Health System. The NeuroNEXT network and its centers were established with grants from the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health. NeuroNEXT was created to facilitate Phase II clinical trials for rare neurological diseases, which face significant challenges securing funding from industry, as well as with recruiting and retaining participants. By establishing a network of sites and streamlining cumbersome logistics, the NeuroNEXT network and their centers aim to allow both experienced and novice researchers to tap into an existing infrastructure that can provide the research and regulatory expertise needed for them to participate in multicenter clinical trials and launch new studies more rapidly. NeuroNEXT centers are encouraged to submit their proposals for clinical trials to the network for evaluation. The inaugural study will focus on spinal muscular atrophy and recruitment will begin this year. Source: Newswise 12/1/11

Cancer Center Tests Novel Drug that Makes Brain Tumors Glow Hot Pink

Just 24 hours after Lisa Rek sang at her niece's wedding, her husband Brad was driving her to a local hospital. "The pain got worse. When we got to the emergency room, I said to Brad 'something is just not right,'" Rek remembers. After an MRI showed a suspected tumor, Rek was immediately flown to Seidman Cancer Center at University Hospitals (UH) Case Medical Center, where Andrew Sloan, MD, diagnosed her with stage 4 glioblastoma, the most aggressive form of brain cancer and the most difficult to treat. "The tumors are comprised of the brain itself. It looks like brain tissue, it sort of feels like brain tissue. It's hard to figure out necessarily where tumor ends and swollen brain tissue begins," says Sloan, director of the Brain Tumor and Neuro-Oncology Center and chair for neurosurgery at UH Case Medical Center and associate professor at Case Western Reserve University School of Medicine. To help identify the difference between tumors and healthy tissue and improve tumor resection, Sloan is testing an experimental drug called 5-aminolevulinic acid (5-ALA), which brain tumor cells glow hot pink when illuminated with a special blue light incorporated into his operating microscope. This novel technique enables surgeons to visualize the edges of the tumor more clearly, allowing them to remove it more completely from the brain. Patients take the drug by mouth prior to surgery and then during their operation, Sloan uses the blue light to identify and remove tumor cells, a process called fluorescent guided resection (FGR). Rek is now one of the volunteers in the research trial. Although 5-ALA is routinely used for FGR in Europe, it has not been approved by the Food and Drug Administration in the United States, and thus is not widely used. UH Case Medical Center is one of a handful of hospitals studying the drug in the U.S. for brain tumor surgery. The study is supported with funds from Sloan's Peter D. Cristal Chair in Neurosurgery and the Kimble Foundation. A video about the 5-ALA research and Lisa Rek's story can be viewed here. Source: EurekAlert! 12/1/11

Psychological Intervention Helps Adolescents with Fibromyalgia

A recent trial shows cognitive-behavioral therapy reduces functional disability and depressive symptoms in adolescents with juvenile fibromyalgia. The psychological intervention was found to be safe and effective, and proved to be superior to disease management education. Full findings from this multisite clinical trial are published in Arthritis & Rheumatism. Medical evidence reports that juvenile fibromyalgia syndrome affects 2 to 7 percent of school-age children. Similar to adult cases, the juvenile form of the disorder primarily strikes adolescent girls. Both adult and juvenile fibromyalgia patients experience widespread musculoskeletal pain, fatigue, and sleep and mood disturbances. Previous studies show that juvenile fibromyalgia patients are burdened with substantial physical, school, social, and emotional impairments. However, studies investing treatment for the juvenile form of the disorder are limited. For the current trial, led by Dr. Susmita Kashikar-Zuck from the Division of Behavioral Medicine and Clinical Psychology at Cincinnati Children's Hospital Medical Center in Ohio, investigators recruited 114 adolescents between the ages of 11 and 18 who were diagnosed with juvenile fibromyalgia. The trial was conducted at four pediatric rheumatology centers between December 2005 through 2009, with participants randomized to cognitive-behavioral therapy or fibromyalgia education, receiving eight weekly individual therapy sessions and two additional sessions in the six months following the end of active therapy. Analyses showed that both patient groups displayed significant reduction in functional disability, pain, and depressive symptoms at the end of the trial. Pediatric participants in the cognitive-behavioral therapy group reported a significantly greater reduction in functional disability compared to those receiving fibromyalgia education. The therapy group had a 37 percent improvement in disability compared to 12 percent in the education cohort. Both groups had scores in the nondepressed range by the end of the study, but pain reduction was not clinically significant—a decrease in pain of less than 30 percent for either group. Source: EurekAlert! 11/22/11

Novel ALS Drug Slows Symptom Progression, Reduces Mortality in Phase II Trial

Treatment with dexpramipexole--a novel drug believed to prevent dysfunction of mitochondria, the subcellular structures that provide most of a cell's energy--appears to slow symptom progression in the neurodegenerative disease amyotrophic lateral sclerosis (ALS). Promising results of a Phase II trial of dexpramipexole are receiving advance online publication in Nature Medicine. "Today there are only two [Food and Drug Administration]-approved drugs used to treat ALS--riluzole, which extends life about 10 percent, and Nuedexta, which treats the emotional instability that characterizes ALS and other neurological disorders," says Merit Cudkowicz, MD, director of the Massachusetts General Hospital Neurology Clinical Trials Unit and ALS Center, lead author of the study. "We need more therapies to slow, halt, and ultimately reverse the course of disease, [as well as] therapies to treat the symptoms." Initially developed by Knopp Biosciences of Pittsburgh, Pa., dexpramipexole appears to protect neurons from mitochondrial dysfunction. Several investigators from Knopp, which sponsored the study reported in the current article, collaborated with Cudkowicz and the Northeast ALS Consortium on the trial. Cofounded and codirected by Cudkowicz, the Northeast ALS Consortium includes more than 100 clinical sites throughout North America that work collaboratively on clinical trials and has established the infrastructure necessarily to quickly and efficiently bring forward new treatments for people with the disorder. The research team devised a two-stage study; in the first stage, 102 patients who had recently been diagnosed with ALS were randomized into four groups, receiving oral tablets of either a placebo or dexpramipexole at total daily dosages of either 50, 150, or 300 mg for 12 weeks. When that stage was completed, participants continuing in the trial received placebo only for four weeks and then were rerandomized into two different groups, receiving daily dosages of either 50 or 300 mg of the study drug for 24 weeks. Results of the first stage showed that receiving dexpramipexole appeared to slow the progression of symptoms measured both by the ALS Functional Rating Scale and by pulmonary capacity. The protective effect was greatest in the 300 mg group, in whom symptom progression was approximately 30 percent slower than in the placebo group, and little effect was seen in those receiving 50 mg. The second stage had similar results, with slower disease progression and a reduced risk of death in participants receiving the higher dosage. While results were not generally statistically significant, due to the small size and short duration of the study, all data trends were dose responsive. According to Cudkowicz, "Confirmation of these findings in the Phase III clinical trial, which is currently ongoing, would give us even more confidence in this study design for Phase II testing in ALS. The Phase III trial, sponsored by Biogen Idec, began earlier this year and has completed enrollment at locations around the world. Source: EurekAlert! 11/18/11

Malaria Drug Studied for Treatment of Metastatic Breast Cancer

An antimalaria drug used for more than 60 years is now being studied for use in breast cancer patients whose disease has not responded to traditional chemotherapy treatment. Dr. Jenny Chang, director of the Methodist Cancer Center in Houston, is leading an investigator-initiated study to look at the efficacy and safety of chloroquine, used in combination with chemotherapy, as a possible treatment for patients with advanced or metastatic breast cancer. The combination of chloroquine and standard chemotherapy already has proven effective in mice models with this disease. The primary goal of this clinical study is to better understand how cancer therapy works on different patients. Chang’s team is focusing on the response of chloroquine used in combination with Taxane or Taxane-like drugs, the active ingredient in which is paclitaxel, a natural product with antitumor activity. With limiting funds flowing into new drug development, Chang said it is important to look at existing—and more affordable—drugs to treat a variety of diseases. “We’re very hopeful that this is a new paradigm that we can apply, repurposing old drugs for 5 cents a day that may make an impact in reversing treatment-resistance in women with breast cancer,” said Chang, a breast medical oncologist. Approximately 47 patients will participate in this 24-week clinical trial. The study will focus on breast cancer patients who were previously treated with an anthracycline regimen, and have either locally advanced cancer (involves the breast and lymph nodes) or their cancer has spread to other parts of the body (metastasized). Source: Newswise 11/17/11

Researchers Discover Achilles' Heel in Lethal Form of Prostate Cancer

An international team of researchers led by clinicians at Weill Cornell Medical College have discovered a genetic Achilles' heel in an aggressive type of prostate cancer--a vulnerability they say can be attacked by a targeted drug that is already in clinical trials to treat other types of cancers. In a recent issue of Cancer Discovery, the researchers report that the investigational drug had a dramatic response in animal models of neuroendocrine prostate cancer, and so provides the first hope of an effective human therapy for this lethal cancer. The study is the largest in-depth analysis of neuroendocrine prostate cancer yet undertaken, and the findings "are very exciting, because our bench-to-bedside approach identified a new molecular target for a subtype of prostate cancer for which a drug is now available," says the study's senior investigator, Dr. Mark A. Rubin, a professor of pathology and laboratory medicine at Weill Cornell Medical College and a pathologist at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. The finding is especially important because many men are now being treated with new, highly potent androgen suppression therapy, which these researchers believe will significantly increase the risk of future development of neuroendocrine tumors. The researchers undertook the study to see if they could find a way to target neuroendocrine tumors, which is considered an orphan disease among other types of prostate cancer. This study demonstrated that the aurora kinase inhibitor PHA-739358 worked against human neuroendocrine prostate cells in the laboratory, and that it had a dramatic response in animal models of neuroendocrine prostate cancer. While PHA-739358 was studied in prostate cancer without success, the researchers suspect that few of the patients who participated had neuroendocrine prostate tumors. Dr. Beltran is preparing a clinical trial to test an aurora kinase inhibitor in prostate cancer patients whose tumors contain neuroendocrine cancer cells or similar molecular alterations involving AURKA and MYCN. "Not only are we eager to test the drug in patients diagnosed with neuroendocrine prostate cancer, we hope to develop biomarkers that can help us screen patients for these cells before the cancer advances," says Dr. Beltran. The research was sponsored by the Ann and William Bresnan Foundation, the Prostate Cancer Foundation, the NCI Early Detection Research Network, and the Department of Defense. Source: Newswise 11/17/11

Collaborators Plan Joint Trials of New Personalized Cancer Treatments

The Virginia G. Piper Cancer Center at Scottsdale Healthcare, serving the greater Phoenix, Ariz. area, is expanding its efforts to accelerate advances in cancer care with the addition of Cedars-Sinai's Samuel Oschin Comprehensive Cancer Institute in Los Angeles as a collaborator in clinical trials to develop personalized therapies that could lead to more effective cancer treatments. The collaboration will focus on Phase I clinical trials of new personalized therapies for gastrointestinal and genitourinary malignancies, and for rare cancers such as adrenal, neuroendocrine, and thyroid cancer. Virginia G. Piper Cancer Center Clinical Trials, a partnership between the Translational Genomics Research Institute (TGen) and Scottsdale Healthcare, will provide local coordination for the program. The partnership offers patients access to promising new cancer treatments through clinical trials, emphasizing collaboration among leading organizations to speed innovation from laboratory bench to patient bedside. The collaborative endeavor also will study new approaches to improve patients' quality of life during cancer treatment and create innovative models to deliver supportive care and services to cancer survivors. Source: EurekAlert! 11/7/11

New Drug Shows Promise Against Multiple Sclerosis

An experimental drug called ocrelizumab has shown promise in a Phase II clinical trial involving 220 people with multiple sclerosis (MS). The study, carried out by researchers at the University of California, San Francisco Medical Center, and involving hospitals in the United States, Canada, and Europe, is described in a recent issue of Lancet. The study involved patients with relapsing-remitting MS, a form of the disease marked by the accumulation of lesions in the brain and spinal cord and periodic “attacks” of neurological impairment. The 220 patients were randomly enrolled into four groups--two that received injections of the monoclonal antibody ocrelizumab at two different doses, one that received the standard MS drug interferon-beta, and one group that was given a placebo. The doctors gauged the effectiveness of each treatment by performing monthly MRI brain scans of the patients and counting the number of visible marks that indicate inflamed lesions, a hallmark of the disease. They also compared the severity and frequency of neurological “attacks” that cause loss of vision and coordination, weakness, and numbness, among other symptoms. The results of this trial showed that patients who received the drug generally fared well and showed fewer signs of the disease than patients who receive a placebo or the standard interferon treatment. Overall, the trial found that ocrelizumab led to a 89 percent reduction in the formation of brain lesions, and it also reduced the number of new MS attacks over 24 weeks. During this relatively short-term study, interferon performed no better than placebo on these outcomes. One patient taking ocrelizumab died, but the relationship with the study drug, if any, is as yet unclear. Whether the drug’s effect and positive safety profile will be sustained over time will be addressed in two parallel Phase III trials, now enrolling a larger number of patients who will receive the drug regimens for a longer period of time. The trials are sponsored by Roche, the company that owns ocrelizumab. Besides the obvious question of whether the drug would work, also under scrutiny in the Phase II trial was whether a drug like ocrelizumab would work in the first place. Its mechanism of action is fundamentally different than other existing therapies for MS, which work by targeting a person’s immune system. Instead of the T cells targeted by other drugs, ocrelizumab targets a molecule called CD20 found on the surface of B cells, a separate component of the immune system. B cells seem to induce the T cells to attack. If T cells are pulling the trigger on nerve fibers in the brain, B cells seem to be the ones that are loading the gun. The Phase II trial showed, in essence, that if you block one, you may be able to stop the other. Source: Newswise 11/1/11

Targeted Antibiotic Drug Safest Among Recommended Treatments for IBS

Among the most commonly used treatments for irritable bowel syndrome (IBS), a targeted antibiotic was shown to be the safest in a new study by Cedars-Sinai researchers, based on an analysis of 26 large-scale clinical trials. The study examined drug interventions for IBS deemed to be of merit by a task force of experts, and compared the therapies based on “number needed to harm statistics” from large clinical trials. Three of these treatments are for diarrhea-predominant IBS (tricyclic antidepressants, alosetron, and the antibiotic rifaximin). The difference among these drugs was striking: For tricyclic inhibitors, the number needed to harm was 18.3, for alosetron it was 19.4, and for rifaximin it was 8,971. The study also found that for every 2.3 and 2.6 patients who benefitted from tricyclic inhibitors and alosetron, respectively, one patient was harmed by the study medication and had to withdraw. For rifaximin, this number was 897. Thus, not only have previous clinical studies shown that the selectively absorbed antibiotic is the first treatment for IBS patients in which patients continue to have relief from their symptoms after stopping the drug, but when compared to other available treatments for the condition, it statistically has caused fewer adverse side effects. The ground-breaking therapy developed at Cedars-Sinai was found through two double-blind trials to provide patients who suffered from diarrhea-prominent IBS relief from bloating, less abdominal pain, and improved stool consistency for up to 10 weeks. Rifaximin is marketed by Salix Pharmaceuticals Inc. Cedars-Sinai holds patent rights to the discovery of its use for IBS, and has licensed rights to the invention to Salix. Source: Newswise 11/1/11

Consortium Gets Contract Renewal to Promote Phase II Trials

The National Cancer Institute (NCI) recently awarded $7.2 million for the competitive renewal of the Southeast Phase 2 Consortium (SEP2C), led by Moffitt Cancer Center’s Daniel Sullivan, MD, executive vice president and associate center director for clinical investigations. The renewal is through September 2016. The SEP2C enrolls patients in Phase II clinical trials and some Phase I trials across six member sites. Moffitt is the lead site for the large contract, which is awarded through the NCI’s N01 research and development contract mechanism. The first year is projected to provide nearly $1.5 million. The other member sites are the University of North Carolina's Lineberger Comprehensive Cancer; Vanderbilt-Ingram Cancer Center; Winship Cancer Institute of Emory University; Virginia Commonwealth University's Massey Cancer Center; and the Cancer Institute of New Jersey. Patient accrual at Moffitt will be focused on acute myeloid leukemia; breast, colorectal, and lung cancers; myelodysplastic syndrome; melanoma and multiple myeloma; non-Hodgkin's lymphoma; ovarian cancer and sarcoma; as well as on immunotherapy and novel Phase I trials. The consortium provides full management of the clinical trials, including initiation, assessing eligibility, recruitment, evaluation of toxicity and response, auditing, follow-up, and analysis of results. Source: Newswise 10/28/11

Institute, Corporation Collaborate to Speed Drug Discovery

Sanford-Burnham Medical Research Institute is the latest research organization to partner with Pfizer, Inc. as part of Pfizer's commitment to transforming research and development through a focus on translational medicine. The Centers for Therapeutic Innovation (CTI) is a research unit at Pfizer dedicated to open innovation through establishing global partnerships with academic research institutions to bridge the gap between discovery science and clinical applications. Using a combination of grant funding, philanthropy, and strategic partnerships, Sanford-Burnham has built a sophisticated infrastructure for advanced drug discovery in the last few years, including recruiting industry-trained leadership with a track record of success in putting drugs into clinical development. Working with the CTI unit at Pfizer will provide Sanford-Burnham investigators with access to additional resources including select Pfizer compound libraries, proprietary screening methods, and antibody development technologies that are directly relevant to the investigators' work. Sanford-Burnham is one of only two participating CTI institutions that are not academic medical centers. Given Sanford-Burnham's proximity to the University of California, San Diego (UCSD), it is anticipated that the institute will collaborate with UCSD when a project reaches the clinical stage. Source: EurekAlert! 11/3/11

Once-Promising Kidney Drug Fails in Large Clinical Trial

What was hoped to be a promising new drug to protect the kidneys has failed to benefit diabetes patients with kidney disease, according to a study appearing in an upcoming issue of the Journal of the American Society Nephrology. The results call into question the usefulness of the drug sulodexide. Kidney disease due to diabetes is the most common cause of kidney failure in developed countries. The number of patients with type 2 diabetes is expected to double and reach 366 million individuals worldwide by 2030. Kidney disease cases are sure to rise in parallel. Investigators have wondered whether sulodexide, which belongs to a class of drugs called glycosaminoglycans, may protect the kidneys. The drug is actually a naturally occurring compound and has been used for more than 20 years to treat various heart conditions. Previous research indicates that sulodexide reduces excretion of protein in the urine, which is a hallmark of kidney disease. To test the effects of sulodexide on the kidneys in a large and extensive trial, David Packham, MD, of the Melbourne Renal Research Group in Australia, and his colleagues within the Collaborative Study Group (a large clinical trial group comprised of various kidney care centers) conducted a randomized, double-blind, placebo-controlled study in patients with diabetes and kidney disease. The investigators planned to enroll 2,240 patients in the Sun-MACRO trial over a period of two years, but they stopped the study early after enrolling 1,248 patients because they did not detect any significant differences between sulodexide and placebo for preventing kidney failure. Also, the trial did not confirm the potentially beneficial effect of sulodexide in reducing urinary protein excretion that was previously reported in smaller studies. The study was funded entirely by Keryx Biopharmaceuticals. Source: EurekAlert! 10/27/11

Clinical Trial Shows First Evidence that Anal Cancer is Preventable

A large, international clinical trial led by doctors at the University of California, San Francisco (UCSF) indicates that a vaccine to prevent anal cancer is safe and effective, according to a study reported in the October 27 issue of New England Journal of Medicine. Though anal cancer is less common than other forms of the disease in the United States, the number of cases has increased in recent years, and is particularly common among men who have sex with men and HIV-infected individuals. Anal cancer is caused by infection with human papilloma virus (HPV), the most common sexually transmitted pathogen in the United States. The virus also causes cervical cancer in women, and the vaccine is already approved and routinely recommended to prevent this condition. The new clinical trial suggests that the same vaccine would also protect men, and likely women, against anal cancer. UCSF professor Joel Palefsky, MD, FRCPC, led the clinical trial, and recently founded a new professional society devoted to the study of the disease. The trial involved a group of 602 men who have sex with men from Australia, Brazil, Canada, Croatia, Germany, Spain, and the United States, all of whom had at least one, but no more than five, sexual encounters and who were between the ages of 16 and 26 years. All were randomized into groups that either received a placebo or a three-shot injection of the vaccine Gardasil, which protects against HPV 16 and 18, the most common HPV types involved in anal cancer, and HPV 6 and 11, the most common types in anogenital warts. The patients were enrolled in the trial from 2006 to 2008, and they were followed for three years after their last shot. As described in the paper, the vaccine proved effective at reducing anal infections with HPV and precancerous lesions known as high-grade anal intraepithelial neoplasia, which are anal cancer precursors. The trial showed that the vaccine reduced the incidence of these cancer precursors by nearly 75 percent among those who had not been previously exposed to any of the HPV types in the vaccine. Among those who were previously exposed to one or more of the types in the vaccine, the vaccine reduced the incidence of the precancerous lesions by 54 percent. The study was funded by Merck and Co. Source: EurekAlert! 10/26/11

Through-the-Nipple Breast Cancer Therapy Shows Promise in Early Tests

Delivering anticancer drugs into breast ducts via the nipple is highly effective in animal models of early breast cancer, and has no major side effects in human patients, according to a report by Johns Hopkins Kimmel Cancer Center researchers in Science Translational Medicine on October 26. The results of the study are expected to lead to more advanced clinical trials of so-called intraductal treatment for early breast cancer. "Our results support the theory that by treating the breast tissue directly we can reach a much more potent drug concentration where it is needed, with far fewer adverse effects on tissues outside the breasts," says oncologist Vered Stearns, MD, PhD, the Breast Cancer Chair in Oncology and co-director of the Breast Cancer Program at the Kimmel Cancer Center, who supervised the clinical part of the study. "This has been a classic translational medicine collaboration between a bench researcher and a clinician scientist," says cancer biologist Saraswati Sukumar, PhD, who supervised the animal tests. Sukumar, the Barbara B. Rubenstein Professor of Oncology at the Kimmel Cancer Center and co-director with Stearns of the Breast Cancer Program, began intraductal research more than a decade ago, reasoning that because most breast cancers originate from cells lining the milk ducts, early or preventive therapies should be delivered directly to the ducts via the nipple, rather than intravenously. In 2006, in the journal Cancer Research, Sukumar and her colleagues reported on an initial successful test of the technique using the chemotherapy drug doxorubicin against early ductal breast cancers in rats. For the current study, Stearns set up a small clinical trial to determine the feasibility of Sukumar's technique in 17 breast cancer patients. Starting first with dextrose--essentially sugar water--and later with escalating doses of the same doxorubicin formulation used on Sukumar's rats (pegylated liposomal doxorubicin, or PLD), she was able to infuse patients' breast ducts via a small catheter placed into the nipple. The technique wasn't used in this case to treat cancer; the patients in the study all had established breast tumors and were awaiting mastectomies. However, Stearns was able to establish that single doses of PLD to breast ducts caused only mild side effects, including mild nipple pain and breast fullness. A comparison of 12 patients receiving PLD intraductally with three patients treated with PLD by the standard intravenous route also was revealing, Stearns said. "Intraductal delivery of PLD resulted in much higher concentration in the breast compared to the circulation, whereas in the women with intravenous doses we saw relatively high concentrations in the blood, but very little if any in the breast," she noted. Sukumar and Stearns say the next step is to set up a further clinical study with a different anticancer drug, 5FU, based on the new findings. The goal is to use intraductal therapy to suppress tumors in patients with a high genetic risk for breast cancer or premalignant lesions in their breast ducts. "In principle, one could do such a procedure every 10 years or so to keep one's breasts tumor-free, as an alternative to having the breasts removed," Sukumar says. The study was funded by the National Cancer Institute, Windy Hill Medical Center, the Mary Kay Ash Foundation and the Susan Love Research Foundation. Source: EurekAlert! 10/26/11

University Begins Clinical Trial of Breast Cancer Vaccine

The University of Arkansas for Medical Sciences (UAMS) in October announced the start of a Phase I clinical trial to study the safety of a novel vaccine to prevent the recurrence of breast cancer. UAMS has three research subjects enrolled in the trial, and plans on completing it sometime in 2012 after studying the safety of the vaccine in up to 18 research subjects. After the safety testing phase is completed, UAMS expects to conduct another clinical trial using the vaccine on up to 50 research subjects. The vaccine was developed by Thomas Kieber-Emmons, PhD, professor of pathology and holder of the Josetta Wilkins Chair of Breast Cancer Research at the Winthrop P. Rockefeller Cancer Institute at UAMS. His research has been federally funded since 1992. Here’s how the vaccine would work: The surface of a cancer cell is covered with carbohydrates. If the vaccine could trigger an immune response to carbohydrates, it would destroy the cancer cells. However, it is difficult to get the immune system to recognize carbohydrates on cancer cells. So Kieber-Emmons used a chemical compound called a peptide to mimic a carbohydrate, in hopes that the body would create an immune response to the peptide that then cross-reacts with the carbohydrate on the tumor cell and destroys the cell. “It’s not quite a bait and switch, but you’re trying to elicit a set of responses you just don’t see with carbohydrates. The true test will come during the clinical trial, but we anticipate our little deception will work to the advantage of breast cancer patients,” Kieber-Emmons said. The successful effort to initiate the clinical evaluation of this novel approach to vaccine development involved a collaborative effort between the university and three well-known service providers--AmbioPharm Inc., a manufacturer of peptide active pharmaceutical ingredients; Advantar Laboratories, a laboratory offering analytical and formulation development services; and NextPharma Technologies, a contract manufacturer for clinical trial materials. Source: Newswise 10/24/11

Institutes Collaborate on Cancer Drug Development

Cedars-Sinai has combined efforts with the Translational Genomics Research Institute (TGen) in Arizona so researchers may offer joint clinical trials and collaborate to develop personalized therapies that could lead to more effective cancer treatments. Physician-scientists at the two institutions, as part of this collaboration, will team up for Phase I clinical trials of new anticancer therapies aimed at molecular targets in prostate, kidney, bladder, and colorectal cancers. Research also will be conducted on drugs for the less common adrenal, neuroendocrine, and thyroid cancers. “Our two organizations share the same goal: to greatly improve cancer treatment with therapies that attack the disease in new and innovative ways,” said Steven Piantadosi, MD, PhD, Phase One Foundation chair and director of the Samuel Oschin Comprehensive Cancer Institute. “Translating new research into effective therapies will improve the lives of cancer patients and, ultimately, lead to a time when cancer is a manageable condition, not a feared disease.” The collaborative endeavor also will study new approaches to improve patients’ quality of life during cancer treatment and create innovative models to deliver supportive care and services to cancer survivors. Cedars-Sinai has one of the largest clinical research trial facilities of any private hospital in the nation. Its outpatient cancer center treats more than 9,000 patients each year, making it one of the busiest treatment facilities in California. The drug development research collaboration will be coordinated by Cedars-Sinai with Clinical Trials, a partnership with TGen and the Virginia G. Piper Cancer Center at Scottsdale Healthcare. Source: Newswise 10/18/11

FDA Grant Launches Atlanta Pediatric Device Consortium

The U.S. Food and Drug Administration (FDA) has awarded the Georgia Institute of Technology, Children’s Healthcare of Atlanta, Emory University, and Saint Joseph’s Translational Research Institute a two-year, $1.8 million grant to foster the development of medical devices focused on the special needs of children. The award will launch the new Atlanta Pediatric Device Consortium, which will provide assistance with engineering design, prototype development, preclinical and clinical studies, and commercialization for novel pediatric medical devices. “By developing, testing, and refining medical devices specifically for children, we hope to produce safer, more effective devices that will improve their lives,” said Barbara Boyan, a tissue engineering expert at Georgia Tech and Emory University. The consortium will be led by Boyan, along with consortium codirectors Kevin Maher, a cardiologist and researcher specializing in pediatrics with appointments at the Children’s Healthcare of Atlanta Sibley Heart Center and Emory University, and Wilbur Lam, a pediatric hematologist/oncologist and bioengineer with appointments at Emory, the Aflac Cancer Center of Children’s Healthcare of Atlanta, and Georgia Tech. Historically, devices designed for adults have been used in children. However, differences in body size and immune system responses between adults and children, and the lack of appropriate models to assess how a device might function in a growing child, can result in poor device performance and responses that are less than optimal. In addition, the high cost of clinical trials for a small market like pediatrics has made conducting pediatric trials cost-prohibitive for many manufacturers. The consortium will try to reduce these barriers by creating a product development pathway that will provide support for commercialization of devices for pediatric healthcare from initial concept to the completed product. The consortium will provide assistance for pediatric medical devices from academic institutions and small businesses. The three technologies that will be investigated initially are: a smartphone attachment designed for at-home ear examinations, a renal dialysis device, and a gel designed to delay the refusion of a child’s skull bones after surgery for craniosynostosis. Future projects will be selected through the consortium’s seed grant competition, which will provide awards between $25,000 and $50,000 to inventors in the partnering institutions and the business community to develop a pediatric medical device through the consortium. Additional devices will be identified through various technology development and commercialization programs. Source: Newswise 10/12/11

Trial Will Use Drug to Boost Immune System Function in Critically Injured Children

Thanks to funding from the National Institutes of Health, Nationwide Children’s Hospital will test the ability of a drug commonly used to improve immune function in pediatric cancer patients to help prevent hospital-acquired infection after severe trauma. It will be the first clinical trial aimed at improving immune function in critically injured children. Traumatic injury remains the leading cause of death for children outside the neonatal period. Beyond the life-threatening damage caused by initial injury, traumatically injured children are at high risk of developing life-threatening nosocomial infection, also known as hospital-acquired infection. The incidence of nosocomial infection is especially high in injured children who require treatment in the intensive care unit. “Adult studies have shown that innate immune function, which is responsible for protecting the body by identifying and killing pathogens, is impaired following critical injury,” said Mark Hall, MD, critical care medicine physician and principal investigator in the Center for Perinatal Research at Nationwide Children’s Hospital. “Using a unique immune surveillance approach at Nationwide Children’s, we have shown a similar finding in children and are able to tell, in a matter of hours, whether or not a child is at high risk for developing nosocomial infection.” Hall’s studies have identified specific thresholds of immune function, by measuring the ability of patients’ blood to produce certain chemicals when stimulated outside the body that predict infection risk; what has been missing is a possible intervention. With funding from the National Institute of General Medical Sciences,  Hall is set to lead a clinical trial to help determine whether GM-CSF, a drug commonly used to reconstitute bone marrow in leukemia and bone marrow transplant patients, could be the intervention needed to help protect critically ill children from infection. Several small studies in adults suggest that GM-CSF can reverse critical-illness-induced immune suppression. Source: Newswise 10/11/11 http://www.newswise.com/articles/view/581582/?sc=mwhn

Results of Trial for Pancreatic Cancer Patients Lead to Worldwide Phase III Study

Patients at Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare were the first in the nation to participate in a clinical trial to determine the safety, tolerability, and effectiveness for usage of a new drug combination consisting of a standard drug called gemcitabine and a drug called nab-paclitaxel for patients with advanced pancreatic cancer. The results of this study, headed by pancreatic cancer expert Dr. Daniel Von Hoff, were published online October 3 in the Journal of Clinical Oncology. Nab-paclitaxel (Abraxane), an albumin-bound formulation of paclitaxel, is a drug manufactured by Abraxis BioScience, a wholly owned subsidiary of Celgene, and is approved for the treatment of patients with advanced breast cancer. The use of this agent in patients with pancreatic cancer is investigational. Scientists at TGen and the International Genomics Consortium, in collaboration with Abraxis scientists, found that in pancreatic cancer an albumin-binding protein called SPARC was present at high levels in cells within the pancreatic tumor microenvironment. It was hypothesized that the albumin formulation of nab-paclitaxel may be taken up by tumor cells with high SPARC expression. Based on these findings, Dr. Von Hoff--joined by colleagues from Johns Hopkins University Hospital, Baltimore; University of Alabama, Birmingham; and South Texas Oncology-Hematology, San Antonio--conducted a clinical trial in patients with advanced pancreatic cancer. The results of this pilot study in which 67 patients were treated showed impressive results. Following completion of a safety and dose finding phase, 44 patients were treated in the Phase II group. About half the patients showed reductions in tumor size measured by CT scans, and about 50 percent lived at least a year. "Compared to the average survival of six months seen typically in this group of patients, this is very encouraging," said Dr. Ramesh Ramanathan, medical director, Virginia G. Piper Cancer Center Clinical Trials. He added that the results of this study need to be confirmed. A large worldwide study of 842 patients comparing the standard treatment of gemcitabine to the new regimen of gemcitabine and nab paclitaxel is under way, led by Dr. Von Hoff and Dr. Ramanathan. Source: EurekAlert! 10/4/11

Study Finds Promising Treatment for Improving Language, Social Function in Autism

Most drug therapy interventions for people with autism have targeted psychiatric problems, including aggression, anxiety, and obsessive behavior. Now, University of Missouri researchers are examining the use of propranolol (a drug used to treat high blood pressure and control heart rate as well as to reduce test anxiety) to improve the primary traits associated with autism--difficulty with normal social skills, language, and repetitive behaviors. The researchers say the drug is a promising new avenue for improving language and social function. “We can clearly say that propranolol has the potential to benefit language and may help people with autism function appropriately in social situations, including making eye contact with others,” said David Beversdorf, associate professor and Thompson Endowed Chair at the university's Thompson Center for Autism and Neurodevelopmental Disorders. “Enhancing both language and social function is significant because those are two of the three main features of autism. Clinical trials will assess the drug’s effect on all three features, including repetitive behaviors.” Propranolol has been used for decades with minimal side effects reported in healthy individuals. The researchers are the first to study the benefits of the drug in autism in a controlled manner. The next step is to conduct clinical trials to determine if the benefits are sustained over time and if the benefits outweigh other effects. In previous studies, the researchers found that propranolol helped people with autism solve simple anagrams (word unscrambling tasks). It also increased semantic word fluency, which requires understanding the definition of words and connectivity among different brain regions. It did not help with letter fluency, which involves identifying words that start with specific letters and requires less distributed connectivity among brain regions. “We are interested to see if we can predict who will or will not respond to this drug among those with autism,” Beversdorf said. “In the follow-up study, we’re looking at markers of increased stress reactivity. If we find that those with higher stress reactivity are more sensitive to the effects of propranolol, it might help to identify who will benefit most from the drug.” Source: University of Missouri News 9/29/11

Cancer Center is Among Earliest Sites in Landmark Multiple Myeloma Study

The John Theurer Cancer Center at New Jersey's Hackensack University Medical Center is one of the first four clinical sites enrolling patients in a landmark study designed to uncover the molecular segments and variations of multiple myeloma. The study is the centerpiece of the Multiple Myeloma Research Foundation’s (MMRF) Personalized Medicine Initiative, CoMMpass (Relating Clinical Outcomes in MM to Personal Assessment of Genetic Profile), aimed to accelerate translational research into therapeutic breakthroughs for patients. According to David S. Siegel, MD, PhD, chief for multiple myeloma at the center and a principal investigator of the study, “This study is designed to build upon our most recent discovery--the completion of the first genomic portrait of multiple myeloma. We hope to gain a greater understanding of the mechanisms of the disease to develop personalized treatments for our patients.” The study will enroll at least 1,000 newly diagnosed multiple myeloma patients who have not yet initiated therapy for their disease. Researchers will track patients from initial diagnosis through their course of treatment, over a minimum of five years, and conduct sequential tissue sampling to identify how a patient’s molecular profile may affect his or her clinical progression and individual response to treatment. The MMRF plans to expand the network of academic and community cancer centers enrolling patients for the study. The current participating sites also include Virginia Cancer Specialists in Fairfax, Va.; Waverly Hematology Oncology in Cary, N.C.; and Mount Sinai School of Medicine in New York City. Source: John Theurer Cancer Center 9/20/11

New Strategy Likely to Speed Drug Development for Rare Cancers

Researchers have identified promising new therapies for ependymoma, a rare tumor with few treatment options. St. Jude Children's Research Hospital investigators led the effort, which used a new, faster drug development system that combines the latest drug screening technology with the first accurate animal model of the tumor. Investigators identified several dozen new and existing drugs as possible ependymoma treatment candidates. The drugs were found by screening 5,303 existing medicines, natural products, and other compounds for activity against the tumor, which develops in the brain and spine of children and adults. The work is published in the current edition of Cancer Cell. The list of candidate drugs included 5-fluorouracil (5-FU). 5-FU has been widely used to treat a variety of adult cancers, but has not been formally tested against ependymoma. Based on study results, St. Jude is planning a clinical trial of 5-FU in young ependymoma patients, said senior author Richard Gilbertson, MD, PhD, director of the St. Jude Comprehensive Cancer Center. Gilbertson credited the method used in this study with highlighting 5-FU's potential. Researchers hope to use the same system to expand chemotherapy options for patients with other cancers. Rather than waiting years for clinical trial results, this system promises to take just months to provide key information about a drug's effectiveness and optimal administration, Gilbertson said. The study was funded in part by the National Institutes of Health, the Collaborative Ependymoma Research Network and ALSAC. Source: EurekAlert! 9/15/11

Grant Helps Families of Children Participating in Brain Injury Trial

The Newborn Possibilities Fund, a grant making program established by Cord Blood Registry (CBR), in September announced it has provided a grant to the Memorial Hermann Foundation in collaboration with the University of Texas Health Science Center at Houston (UTHealth) and Children's Memorial Hermann Hospital (UTHealth's children's teaching hospital). The UTHealth Medical School is conducting an innovative Food and Drug Administration (FDA)-regulated clinical trial evaluating the use of a child's own cord blood stem cells as a potential medical intervention for traumatic brain injury. The grant will provide financial support to help with travel and other expenses for families with a child participating in the trial. The study will include 10 children, ages 18 months to 17 years who have suffered moderate to severe traumatic brain injury within the past six to 18 months. After trial eligibility has been confirmed, study participants will travel to Houston to undergo a medical history and physical exam, neuropsychiatric evaluation, DT-MRI imaging of the brain, and baseline laboratory evaluation. The participant's cord blood will be infused intravenously and they will be monitored as an in-patient for 24 hours, returning the following day for a final examination. Follow-up visits will occur back at UTHealth at 180 days, one year, and two years post-infusion. This important clinical study will provide new insights into the potential of cord blood stem cells to help children recover from nerve tissue damage to the brain," said Heather Brown, vice president of scientific and medical affairs at CBR. "However, the study design requires a family to make trips at their own expense to the study center. The goal of the Newborn Possibilities Fund is to remove financial barriers that may prevent eligible children from participating in this cutting-edge research and receiving investigational treatments that may improve their quality of life." The fund was created to help advance clinical research investigating the use of a child's own cord blood stem cells as a treatment for conditions like cerebral palsy and traumatic brain injury; it directs financial grants to nonprofit organizations to help cover the cost of travel for families who have the chance to participate in FDA-regulated trials, and is administered by Tides, a public charity, on behalf of CBR. Source: PRNewswire 9/14/11

Computerized Anxiety Therapy Found Helpful in Small Trial

A small clinical trial suggests that cognitive bias modification (CBM), a potential anxiety therapy that is delivered entirely on a computer, may be about as effective as in-person therapy or drugs for treating social anxiety disorder. The Brown University-led research also found that participants believed the therapy to be credible and acceptable. Participants in the pilot study, published in advance online in the journal Depression and Anxiety, improved their scores on a standardized measure of anxiety and on a public speaking task after completing two simple exercises twice a week for four weeks. The hope for CBM is that it can provide a new option for anxiety sufferers who cannot find or pay for a qualified therapist, who are afraid to try cognitive behavior therapies where they directly confront their fears, or who can't or don't want to try medications. Still, the idea of computer-based therapy has been controversial, acknowledged Courtney Beard, the new study's lead author and assistant professor (research) of psychiatry and human behavior in the Warren Alpert Medical School of Brown University. "A lot of people are skeptical, particularly people like me who are clinicians and know how hard it is to help people with anxiety and how much effort and time it takes in therapy," Beard said. "It just doesn't seem possible that a computer program could produce similar effects. But I'm more of a scientist than a clinician so I want to see data." Beard's study is the first randomized clinical trial, albeit with a small sample size of 20 people who received the therapy and 12 placebo controls, to combine two techniques of CBM to treat social anxiety disorder: one that seeks to enhance subjects' control over what they pay attention to and another that trains them to interpret situations less anxiously. Beard worked on the study with coauthors Risa Weisberg at Brown and Nader Amir at the University of California, San Diego. Studies of CBM began in healthy subjects in 2002, but clinical trials of CBM as a therapy for people with anxiety only began to appear in the literature in 2009, Beard said. Hers is the first clinical trial to add a behavioral performance measure, such as public speaking, and is the first to assess whether patients found the treatment credible (e.g., did the therapy make sense?) and acceptable (was this something you could do?). On both issues, participants provided moderately positive assessments. Although the study's results agree with many of the other small studies published to date, Beard said CBM still must be subjected to larger trials with longer follow-up times before the therapy's seeming effectiveness can be considered convincing. Funding for the study came from the National Institute of Mental Health. Source: EurekAlert! 9/14/11

$1.1 Million Grant Targets Disparities Among Medical Researchers

A Michigan State University (MSU) professor is using a $1.1 million federal grant to help overcome the racial and socioeconomic disparities seen among health and medical researchers. Elahe Crockett, an associate professor in the Department of Medicine at MSU's College of Human Medicine, will use the National Institutes of Health (NIH) funding to establish a short-term research education program aimed at under-represented, minority, and disadvantaged students at MSU. The goal of the program, known as the Research Program to Increase Diversity in Health Researchers, is to inspire those students to pursue research careers in cardiovascular, pulmonary, and hematologic disease disciplines. For many decades, little attention was given to diversity and the disparities that exist in health-related research and clinical practice in the United States. "This was especially true for individuals who suffer disproportionately from cardiovascular disease, diabetes, asthma and cancer, among other conditions, yet rarely were represented in research," Crockett said. Despite attempts to address the problem, including a 1993 NIH requirement for clinical trials to include women and minorities, large gaps remain in knowledge and in practice. As a result, racial and ethnic minorities continue to have higher rates of disease, disability, and premature death than nonminorities. As part of the program, 16 students will be recruited: four undergraduate students and 12 health professional students (medical, public health, and nursing students). They will be exposed to team mentoring and research training by mentors across campus, an introductory biomedical research course, and a summer hands-on research experience. The students will receive stipend as well as financial assistance to attend a biomedical conference. Source: EurekAlert! 9/14/11

Whole-Parasite Malaria Vaccine Shows Promise

For the first time, a malaria vaccine that uses the entire malaria parasite has proven safe and shown promise to produce a strong immune response in a clinical trial, according to a new study coauthored by researchers at the University of Maryland School of Medicine Center for Vaccine Development in Baltimore. The vaccine is unique in that it employs the entire malaria parasite, while most experimental malaria vaccines consist of just one or at most a few proteins found in the parasite. Researchers found that the vaccine—the first whole-parasite vaccine to be approved by the U.S. Food and Drug Administration for clinical trials—could provide unprecedented immune responses against malaria when administered intravenously. The study was published online in the journal Science in early September. The study also involved the U.S. Military Malaria Vaccine Program at the Naval Medical Research Center, the Vaccine Research Center at the National Institutes of Health, and the Rockville, Md.-based biotechnology firm Sanaria, Inc., which developed and manufactured the vaccine. "No vaccine has completely protected against malaria in a challenge trial, in which vaccinated volunteers are subjected to the bite of an infected mosquito to measure their immunity," says Kirsten Lyke, MD, associate professor of medicine and a research scientist at University of Maryland vaccine center. "This vaccine showed strong promise. We hope that with further study it could help revolutionize the field and prevent death and illness from malaria worldwide, and be used to eliminate malaria from certain areas." Researchers found that the vaccine produced a partial protective response in the 80 volunteers who were immunized subcutaneously by traditional needle and syringe in the Phase I trial. However, this response was significantly less than the 80 percent to 90 percent protective immunity the research team is intent on achieving. Researchers suspect that administering the vaccine more directly into the bloodstream, accelerating its path to the liver, might produce an even stronger response. "Our hope is that we can optimize the delivery of this vaccine to prevent and eliminate malaria on a global level," says Lyke. She and her colleagues are already at work designing new studies to find the best way to administer the vaccine. Source: EurekAlert! 9/7/11

Sorting Out Major Brain Stent Study: Experts Say Procedure Effective for Some

An article appearing in the September 7 New England Journal of Medicine, reporting on National Institutes of Health research on brain stents, says aggressive medical treatment without stenting is better for high-risk stroke patients. However, experts at Cedars-Sinai Medical Center who were involved in the study believe this procedure is appropriate for some patients. They express concern that those who might benefit from minimally invasive placement of a mesh tube or stent to open blocked brain arteries may be discouraged by this report. They say this study is a helpful start, but not likely to be the final word on understanding when stenting may be appropriate, and raise concerns about several study limitations and exclusions. "Patients in the trial received aggressive management of their risk factors--far greater than patients usually get. Only with such heroic measures will the average patient benefit the way those in the trial did," says Patrick D. Lyden, MD, chairman of the Department of Neurology and the Carmen and Louis Warschaw Chair in Neurology at Cedars-Sinai. In the study, sicker patients--such as those with multiple blockages of the arteries or long-length blockages--were excluded; these are among patients most likely to benefit from stenting. Furthermore, most patients in the trial had blockages in smaller arteries, which are more difficult to treat with stenting. Also, it is common in clinical trials of interventional or surgical procedures for patients in the treatment arm to have a higher "event rate" in the first 30 days; long-term results provide more valid information. The Wingspan stent evaluated in the study was designed to be more flexible to accommodate fragile brain arteries. The device is approved by the Food and Drug Administration. Source: EurekAlert! 9/7/11 See also www.eurekalert.org/pub_releases/2011-09/uof-amt090211.php and www.eurekalert.org/pub_releases/2011-09/nion-nsp090111.php

Children's Hospital Now Part of Pediatric Research Network

The emergency medicine department at Hasbro Children's Hospital, the pediatric division of Rhode Island Hospital in Providence and a pediatric teaching and research hospital of the Warren Alpert Medical School of Brown University, has received a grant that will support its participation in the Pediatric Emergency Care Applied Research Network (PECARN). This is the nation's first federally funded pediatric research network focused on the prevention and management of acute illnesses and injuries in children of all ages. Hasbro Children's Hospital is now one of only 18 pediatric emergency departments in the country to be part of PECARN. Under the direction of Thomas Chun, MD, MPH, a pediatric emergency medicine physician and researcher, Hasbro Children's Hospital will join a "node" of PECARN along with children's hospitals in Pittsburgh, Pa. and Delaware to form the PRIDENET Node. The annual funding for the node is $630,000. "The significance of PECARN's work lies in the fact that many important problems in pediatrics are difficult to answer because they occur infrequently," Chun says. "Multicenter studies are necessary to be able to identify enough patients and investigate these problems." Current research under way through PECARN includes studies on head trauma, bronchiolitis, pediatric diabetic ketoacidosis, and other important areas of children's health. Source: EurekAlert! 9/7/11

New Half-Match Bone Marrow Transplant Procedure Yields Promising Outcomes

Half-matched bone marrow or stem cell transplants for blood cancer patients have typically been associated with disappointing clinical outcomes. However, a clinical trial conducted at the Kimmel Cancer Center at Jefferson testing its unique, two-step half-match procedure has produced some promising results: the probability of overall survival was 45 percent in all patients after three years and 75 percent in patients who were in remission at the time of the transplant. Reporting in the journal Blood in a published-ahead-of-print article dated August 25, Neal Flomenberg, MD, Chair of the Department of Medical Oncology at Thomas Jefferson University Hospital, Dolores Grosso, DNP, coprincipal investigator and lead author of the article, and colleagues discuss the results of 27 patients treated on this Phase I/II trial who had diagnoses that included leukemia, lymphoma, and myelodysplasia. The patients received their transplant in two steps. First, after receiving radiation therapy to further treat their disease, the patients were given a specified dose of T cells (a type of immune cell that fights infection) from their half-matched family donor. The donors were parents, siblings, or children of the patient. The patients next received the drug cyclophosphamide to help the newly infused donor T cells to be more tolerant to the patient’s body. The second step of the transplant occurred when the patients received a dose of their donors’ stem cells to help their blood counts return to normal and further strengthen their new immune system. Dr. Flomenberg and his team found that after a follow-up of 28 to 56 months, overall survival for the patients after one year was 54 percent and 48 percent at three years. Patients in remission at the time of the transplant fared better, with an overall survival of 75 percent. Seventeen of the 27 patients—with a median age of 52 years old—were alive six months after their transplant, which was the official endpoint of the trial. Jefferson medical oncologists’ approach is unique in that the dosage, timing, and treatment of donor T cells was carefully controlled and optimized. No other transplant regimen controls the exact amount of donor T cells given. The investigators believe that dosing the T cells in this way helped avoid many of the life-threatening side effects of this type of transplant. Source: Newswise 9/1/11

Excitement Follows World-First Viral Therapy Trial in Cancer Patients

Researchers from the Ottawa Hospital Research Institute (OHRI), the University of Ottawa (uOttawa), Jennerex Inc., and several other institutions in late August reported promising results of a world-first cancer therapy trial in renowned journal Nature. The trial is the first to show that an intravenously delivered viral therapy can consistently infect and spread within tumors without harming normal tissues in humans. It is also the first to show tumor-selective expression of a foreign gene after intravenous delivery. The trial involved 23 patients (including seven at the Ottawa Hospital), all with advanced cancers that had spread to multiple organs and failed to respond to standard treatments. The patients received a single intravenous infusion of a virus called JX-594, at one of five dose levels, and biopsies were obtained eight to 10 days later. Seven of eight patients (87 percent) in the two highest dose groups had evidence of viral replication in their tumor, but not in normal tissues. All of these patients also showed tumor-selective expression of a foreign gene that was engineered into the virus to help with detection. "We are very excited because this is the first time in medical history that a viral therapy has been shown to consistently and selectively replicate in cancer tissue after intravenous infusion in humans," said Dr. John Bell, a senior scientist at OHRI, professor of medicine at uOttawa, and senior coauthor on the publication. "Intravenous delivery is crucial for cancer treatment because it allows us to target tumors throughout the body, as opposed to just those that we can directly inject. The study is also important because it shows that we can use this approach to selectively express foreign genes in tumors, opening the door to a whole new suite of targeted cancer therapies." JX-594 is derived from a strain of vaccinia virus that has been used extensively as a live vaccine against smallpox. It has a natural ability to replicate preferentially in cancer cells, but it has also been genetically engineered to enhance its anticancer properties. Although the current trial was designed primarily to assess safety and delivery of JX-594, antitumor activity was also evaluated. Six of eight patients in the two highest dose groups experienced a shrinking or stabilization of their tumor, while those in lower dose groups were less likely to experience this effect. "[W]e will need to do more trials to know if this virus can truly make a difference for patients," Bell said. "We are working hard to get these trials started, and at the same time, we are also working in the laboratory to advance our understanding of these viruses and figure out how best to use them." This research was supported by Jennerex Inc., the Terry Fox Foundation, the Canadian Institutes of Health Research, the Ontario Institute for Cancer Research, the Ottawa Hospital Foundation, the Canada Foundation for Innovation, the Natural Sciences and Engineering Research Council of Canada, and the Republic of Korea. Source: EurekAlert! 8/31/11

Hospital is First in Michigan to Enroll Patient in Worldwide Drug-Coated Balloon Trial

Physicians at St. John Hospital and Medical Center have enrolled the first patient in Michigan in LEVANT2, a global, multicenter, randomized clinical trial evaluating the safety and effectiveness of the Moxy™ drug-coated balloon for the treatment of peripheral arterial disease. Thomas Davis, MD, an interventional cardiologist with the center, performed the case. LEVANT 2, sponsored by medical device manufacturer Lutonix, Inc., is the first drug-coated balloon pivotal trial to be approved by the Food and Drug Administration. St. John Hospital is one of 55 centers around the world participating in the trial, which is expected to randomize 476 patients with diseased leg arteries. The trial will investigate whether the Moxy balloon is more effective than standard angioplasty at keeping leg arteries open and free from reblockage over time. LEVANT 2 is the largest randomized peripheral drug-coated balloon clinical trial to date, and one of the largest peripheral vascular studies ever conducted. Randomized patients in LEVANT 2 will be followed for a total of five years, and independent core laboratories will be utilized to verify trial outcomes. Drug-coated balloons are similar to standard angioplasty balloons, except they are coated with an antirestenosis drug aimed at preventing the artery from becoming blocked again. During the procedure, the Moxy balloon is inserted inside the narrowed area of the artery and then inflated in order to open the blockage and deliver the drug to the artery. After this short inflation, the balloon is removed from the body, leaving nothing but the drug behind in the artery. LEVANT 2 is a follow-on trial to LEVANT 1, which was a 101-patient, multicenter, randomized trial. Patients either received the Moxy balloon or standard angioplasty for the treatment of diseased femoropopliteal arteries. Based on the success and positive results of the LEVANT 1 trial, LEVANT 2 was designed to investigate the device in a larger patient population. Source: EurekAlert! 8/29/11

Universities Announce Drug Discovery Partnership

The University of Rochester Medical Center (URMC) and Temple University School of Pharmacy have announced a partnership that will help translate novel medical research into new drugs for treating diseases. The partnership reflects a growing trend in medical research in which academic institutions have become more directly involved in the drug discovery process, a role that has historically been filled by the pharmaceutical and biotech industries. As drug companies cut back on in-house research and development and become more risk-averse, they are increasingly looking to universities to conduct the early stage research necessary to identify promising new discoveries. For academic institutions, this means playing a more active role in identifying and guiding new compounds from the earliest stages of research along the path to becoming a new drug. The agreement enables scientists at the two institutions to collaborate and move these discoveries to the next stage of research by identifying compounds that act upon these new targets and may ultimately form the basis for new therapeutics. URMC scientists will work with Temple University's Moulder Center for Drug Discovery Research to rapidly screen large numbers of compounds to identify novel drug candidates that can undergo a battery of preclinical tests in the lab and in animal models necessary to ensure that they are stable, not toxic, and act as intended. Some 30 URMC research projects have already been identified as potential candidates for this collaboration. These include novel approaches that could lead to new treatments for bacterial pathogens such as methicillin-resistant Staphylococcus aureus and Acinetobacter baumannii, which are notorious for their ability to evade conventional drug treatments and are widely regarded as two of the most important bacterial threats to health. Other ongoing URMC research is aimed at developing treatments for fungal diseases caused by Candida albicans and Cryptococcus neoformans, pathogens that affect premature infants, patients receiving cancer chemotherapy and organ transplants, and those living with HIV/AIDS. Source: EurekAlert! 8/18/11

$3.4 Million Grant Initiates Study of Personalized Medicine

Mount Sinai School of Medicine has been awarded a $3.4 million grant over four years from the National Human Genome Research Institute of the National Institutes of Health to begin the largest study of its kind, in which a patient’s genomic risk for disease is revealed in a lab, and then entered into an electronic medical record for use in determining treatment in the clinical care setting. Using DNA and plasma samples provided by patients, Mount Sinai researchers from the Charles R. Bronfman Institute for Personalized Medicine will identify genetic markers of disease for each patient enrolled in the study and input them into Mount Sinai’s new electronic medical records system in a safe and secure way. Physicians who are treating these patients in the clinical setting may then electronically access this genomic information and determine susceptibility for heart disease, responsiveness to certain medications, and a personalized course of treatment. The study, called the “Biorepository for Genomic Medicine in Diverse Communities,” is part of a consortium of seven leading genomic medicine institutions called Electronic Medical Records and Genomics (eMERGE). As a member of the consortium, the Mount Sinai team hopes to enroll up to 20,000 patients from the Mount Sinai Biobank, which consists of consented patients representing the diverse communities surrounding the Mount Sinai Medical Center. The research team will create a database containing each of these individual’s genetic-disease risk profiles for heart, kidney, and liver disease, as well as their likely response to different medications and potential side effects. The study will include one group of physicians and patients that will be randomized to a genomic-risk assessment, and another that will be randomized to a traditional risk assessment, using risk factors like cholesterol and high blood pressure when determining a patient’s risk for heart disease. The team will evaluate whether the genomic-risk assessment, based on the presence of certain SNP genotypes as well as traditional risk factors, will lead to improved management of risk factors for heart disease in each patient and help prevent the onset of heart disease, as compared to the using traditional risk factors alone. Additionally, the research team will enter 3,000 biobank patients into a pool of 32,000 patients as part of genome-wide association studies that will be conducted by the eMERGE consortium with the goal of identifying genetic variants associated with 40 additional disease characteristics and symptoms. Source: Newswise 8/18/11

Outcomes Vary in Global Heart Failure Trials by Geographic Region

A comparison of several international clinical trials of beta-blocker drugs has shown there are notable differences in how well the drugs prevent deaths in heart failure patients, based on where the patients were treated. In this study, U.S. patients apparently had a lower survival rate with beta-blocker treatment compared to patients outside the U.S. The analysis was published online in mid-August in the Journal of the American College of Cardiology. The findings point to a consistent pattern of geographical differences within clinical trials that needs to be addressed, according to the study's lead author. The dissimilarity in multinational heart failure trial findings is being published at a time when globalization of clinical trials is on the rise. Although global clinical trials enable researchers to achieve enrollment goals within a reasonable time frame, and ensure new therapies are adequately tested in broad populations who will likely be exposed to them once market approval has been obtained, researchers must be aware that geographical discrepancies in results may exist, and future trials must be adapted accordingly. Factors that may contribute to the geographic discrepancies in clinical outcome include population differences, genetics, and cultural, social, or administrative differences in disease management. The results may also reflect how different population groups, including ethnicities, respond to the drugs, as well as differences in standards of care and the use of evidence-based therapies that may have contributed to differences in patient outcomes. Source: EurekAlert! 8/15/11

NIH Awards $84 Million for Comparative Effectiveness Heart Disease Trial

The New York University (NYU) Langone Medical Center announced it has received an $84 million grant from the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) to study the comparative effectiveness of two initial management strategies for patients with coronary artery disease. The International Study of Comparative Health Effectiveness with Medical and Invasive Approaches (ISCHEMIA) is a randomized controlled trial that will study 8,000 patients with stable ischemic heart disease and moderate to severe ischemia. The study, funded by one of the largest grants ever awarded by the NIH for a comparative effectiveness trial, will enlist the collaboration of more than 150 medical centers around the U.S. and hundreds of sites in 33 countries worldwide. The grant is the largest ever awarded to NYU Langone Medical Center. The trial, which will be clinically coordinated from the Cardiovascular Clinical Research Center at NYU Langone, will determine whether a routine early invasive strategy with cardiac catheterization followed by revascularization plus optimal medical therapy and lifestyle changes is superior to a conservative strategy of optimal medical therapy, reserving invasive procedures for failure of this strategy in patients with moderate to severe ischemia. The study will also assess whether the early invasive strategy improves angina-related quality of life. The other clinical trial core functions of ISCHEMIA will take place at different U.S. sites. Duke Clinical Research Institute will be the statistical and data coordinating center, and it will also serve as the coordinating center for the cost economics and quality of life. Emory School of Medicine will be the ischemia imaging coordinating center. In addition to the study chair at NYU Langone, the ISCHEMIA leadership committee includes faculty from Vanderbilt University, Columbia University, the University of New York at Buffalo, East Carolina University, Duke University, Brigham and Women's Hospital, and staff from the NHLBI. Source: EurekAlert! 8/1/11

Medical Center to Begin Trial on First-in-Class Anti-TB Drug

University Hospitals (UH) Case Medical Center in Cleveland, Ohio, will begin a Phase I clinical trial on a new experimental anti-tuberculosis (TB) drug called TMC207. This drug represents the first new class of anti-TB drugs in the past 60 years, and it has activity against both drug-susceptible and drug-resistant TB. The study is the first trial under a seven-year, $16.8 million contract awarded from the National Institutes of Health through the research organization Clinical Research Management to UH Case Medical Center, Case Western Reserve University, and Johns Hopkins University for first-in-human studies of new drugs for emerging infectious diseases. TMC207 will be tested in 32 healthy individuals for its safety and tolerability, and to determine if its pharmacological activity is affected by repeat doses of other anti-TB drugs, rifabutin and rifampin, which are already in use and are often used in combination for treating TB. Source: EurekAlert! 7/29/11

UH Rainbow Babies and Children's Hospital Opens Asthma Studies

Physicians at University Hospitals (UH) Rainbow Babies and Children's Hospital are participating in two new clinical trials with the national research consortium AsthmaNet. UH Rainbow, one of the 27 clinical sites in the United States, has partnered with University of Pittsburgh Medical Center (through a subcontract with Case Western Reserve University), to collaborate on these studies and future asthma clinical research studies in children and adults. The first study will focus on new treatments for wheezing in young children who wheeze during colds, while a second will examine if Vitamin D can help adolescents and adults who cannot control their asthma through standard inhaled controller medications. The first study, referred to as APRIL/OCELOT, has two parts. The purpose of the first part of the randomized, double-blind study is to determine if starting the anti-inflammatory drug azithromycin at the first signs of a cold will prevent the development of significant breathing symptoms such as frequent coughing, trouble breathing, or wheezing. If wheezing does begin, the second part of the study is focused on whether steroids are effective at reducing the severity of the wheezing and other respiratory symptoms. UH Rainbow hopes to enroll 30 to 40 children through UH Rainbow pediatric offices; 600 children will participate in the nation. The second study, VIDA (Vitamin D add-on therapy enhances corticosteroid responsiveness in Asthma), concentrates on adolescents and adults ages 18 years of age and older who have been diagnosed with asthma and are nonsmokers. The purpose of this study is to learn if taking Vitamin D in addition to an inhaled steroid--the most effective treatment for asthma available today--will help prevent worsening asthma symptoms and asthma exacerbations in people who have low Vitamin D levels. UH Rainbow will enroll 20 patients; 400 patients will be enrolled nationally. This study will also be a double-blind, randomized trial. These studies at UH Rainbow are supported by a grant awarded to the University of Pittsburgh, one of nine centers in the AsthmaNet research program. It is funded by the National Heart, Lung, and Blood Institute of the National Institutes of Health. Source: EurekAlert! 7/29/11

Trial of Molecular Therapy for Muscular Dystrophy Yields Positive Results

A molecular technique originally developed at the University of North Carolina at Chapel Hill has taken one step closer to becoming a treatment for the devastating genetic disease Duchenne muscular dystrophy. The novel treatment uses strips of genetic code--called antisense oligonucleotides--to restore the function of a defective dystrophin gene. In a study published July 25 in the journal The Lancet, researchers from the U.K., U.S., and Australia demonstrated that a Phase Ib/IIa trial of the approach restored production of the critical muscle protein missing in patients with the progressive neuromuscular condition. In Duchenne muscular dystrophy, which affects one in 3,500 newborn boys, cells fail to make a functional muscle protein and patients eventually lose their ability to walk and breathe. Scientists administered the gene-based treatment intraveneously to 19 Duchenne muscular dystrophy patients over the course of 12 weeks. They found that the drug was well tolerated and appeared to increase the levels of dystrophin protein in a statistically significant dose-dependent manner. The best three responders showed an increase following treatment of protein levels from 2 percent to 18 percent, from 0.9 percent to 17 percent, and from 0 percent to 7.7 percent of normal muscle, respectively. The researchers now plan to expand their studies in a new trial, increasing both the dose and the duration of the treatment to see if the approach has clinical impact. In the future, the researchers would like to develop alternative formulations of the drug that can be administered subcutaneously, like for diabetes, or perhaps even in pill form. The research was funded in part by the National Institute of General Medical Sciences and the National Heart, Lung, and Blood Institute. Source: EurekAlert! 7/25/11

Drug Improves Sight for Patients with Inherited Blindness

A clinical trial led by Newcastle University shows that idebenone (Catena®) improved the vision and perception of color in patients with Leber's hereditary optic neuropathy (LHON). The inherited mitochondrial disorder means patients, who can see normally, lose the sight in one eye and then, within three to six months, lose the sight in their other eye. In some severely affected patients, such as those who were unable to read any letters on an eye chart, the treatment with idebenone resulted in a marked improvement in vision. In nine patients (12 eyes) out of 36 patients (61 eyes) taking idebenone, vision improved to the extent that patients were able to read at least one row of letters on a chart. In contrast, not a single patient of the 26 who were taking the placebo improved to that extent. In the journal Brain, the authors describe how patients with LHON were recruited from Newcastle Hospitals in the U.K., Germany, and Canada for a double-blind trial. Patients were either given idebenone for 24 weeks or a placebo. Idebenone penetrates into the mitochondria and is thought to mop-up toxic free radicals and enhance mitochondrial function. Previous research had provided anecdotal reports of improvements in vision, but this is the first time it had been put to the test in a clinical trial. The drug company sponsoring the trial, Santhera Pharmaceuticals, is now seeking marketing approval from the European Medicines Agency for it to be offered as a standard form of treatment. Source: EurekAlert! 7/25/11

Existing Anti-Seizure Treatment Helps in Condition that Leads to Alzheimer's

An existing anti-seizure drug improves memory and brain function in adults with a form of cognitive impairment that often leads to full-blown Alzheimer's disease, a Johns Hopkins University study has found. The findings raise the possibility that doctors will someday be able to use the drug, levetiracetam, already approved for use in epilepsy patients, to slow the abnormal loss of brain function in some aging patients before their condition becomes Alzheimer's. The researchers emphasize, however, that more studies are necessary before any such recommendation can be made to doctors and patients. The new study, presented July 20 at the International Congress on Alzheimer's Disease in Paris, also shows that excess brain activity in patients with a condition known as amnestic mild cognitive impairment, or aMCI, contributes to brain dysfunction that underlies memory loss. Previously, it had been thought that this hyperactivity was the brain's attempt to "make up" for weakness in its ability to form new memories. The clinical study, funded by the National Institutes of Health, tested 34 participants, some healthy older adults and others with aMCI, meaning that they had memory difficulties greater than would be expected at their age. Each person participated in a sequence of two treatment phases lasting two weeks each. Patients received a low dose of levetiracetam during one phase and a placebo during the other. After each treatment phase, the researchers evaluated subjects' memory and conducted functional magnetic resonance imaging of their brains. These scans were used to map brain activity during performance of a memory task, allowing the researchers to compare each individual's status both on and off the drug. Compared to the normal participants, subjects with aMCI who took the placebo had excess activity in the hippocampus, a part of the brain essential for memory. However, when they had been taking levetiracetam for two weeks, the excess activity was reduced to the same level as that of the control subjects; memory performance in the task they performed also was improved to the level of the controls. Source: EurekAlert! 7/20/11

Gene Therapy to Reverse Heart Failure Ready for Clinical Trials

A promising gene therapy developed, in part, at Thomas Jefferson University's Center for Translational Medicine in Philadelphia, Pa., to prevent and reverse congestive heart failure, is on the verge of clinical trials after years of proving itself highly effective in the lab and a large animal study. Reporting in the online July 20 issue of Science Translational Medicine, cardiology researchers have demonstrated feasibility, the long-term therapeutic effectiveness, and the safety of S100A1 gene therapy in a large animal model of heart failure under conditions approximating a clinical setting. "This is the last step you have to take to finish a very long line of research," said Patrick Most, MD, adjunct assistant professor of medicine at Thomas Jefferson University, and lead author of the study who now heads the Institute for Molecular and Translational Cardiology at the University of Heidelberg, Germany. "The reversal of cardiac dysfunction in this preclinical heart failure model in the pig by restoring S100A1 levels in practically the same setting as in a patient is remarkable and will pave the way for a clinical trial." The therapy works by raising diminished levels of the protein S100A1, a calcium-sensing protein in the diseased heart muscle cell, to normal. Previous research suggests this will prevent heart failure development, particularly in people who have had a heart attack. Work on S100A1 started benchside 15 years ago, with Dr. Most and Walter J. Koch, PhD, now director of the Center for Translational Medicine in the Department of Medicine in Jefferson Medical College of Thomas Jefferson University. Five years ago, Jefferson researchers showed that increasing levels of the protein above normal helped protect mouse hearts from further damage after simulated heart attacks. The hearts worked better and had stronger contractile force. In their latest study, Drs. Koch and Most and their team of researchers used a pig model—this type more closely resembles human physiology, function, and anatomy—to determine the effectiveness and safety of the S100A1 gene therapy. Researchers were also able to administer it with certified catheters and delivery routes, just as a human patient would receive it. Heart failure was induced in the pigs, which at 14 weeks showed significantly decreased S100A1 levels. However, treatment with the gene therapy prevented and reversed development of heart failure by restoring the S100A1 protein levels or getting them above normal. This is the final set of preclinical data needed to apply for investigational new drug status with the U.S. Food and Drug Administration and advance to a Phase I clinical trial. Researchers say one of the next steps is to find industry or private partners to help fund the work, as well as recruit eligible patients to enroll in the clinical trial. Source: EurekAlert! 7/20/11

New Trial to Examine Social Withdrawal in Fragile X and Autism

Children and adults with social withdrawal due to fragile X syndrome, the most common cause of inherited intellectual disability and the most common known single-gene cause of autism, may benefit from an experimental drug under study by pediatric neurologists at Rush Children's Hospital at Rush University Medical Center in Chicago, Ill. Rush is the only site in Illinois and one of 21 hospitals in the U.S. participating in the trial. Fragile X syndrome is a neurodevelopmental disorder characterized by impaired social function, cognition, and speech, as well as attention deficits and anxiety. People with fragile X, autism, or autism spectrum disorders often display social impairment, including social withdrawal and anxiety, and have difficulty communicating and interacting with others. Although there are behavioral and psychological interventions, there are no approved medications for the treatment of social or communication difficulties in fragile X, autism, and autism spectrum disorders. The study is sponsored by Seaside Therapeutics, Inc., and will test the efficacy, safety, and tolerability of the drug called STX209 (arbaclofen). Racemic baclofen (a mixture of arbaclofen and esbaclofen) is approved by the U.S. Food and Drug Administration (FDA) to treat spasticity and stiff muscles due to cerebral palsy or other forms of brain or spinal cord injury, but arbaclofen, the more active form of baclofen, is not FDA-approved. Participants in the double-blind, placebo-controlled Phase III trial will be randomized to receive either the study drug or a placebo. The clinical trial will include screening, treatment, withdrawal of medication, and a follow-up period. Subjects who complete the study may be eligible to enroll in a subsequent open-label study, in which all subjects will be treated with STX209. STX209 has already been studied in a small placebo-controlled trial in children and adults with fragile X syndrome, and showed evidence of benefit for social withdrawal. Source: EurekAlert! 7/20/11

Medical Center Receives $38.9 Million to Help Translate Science into Treatment

A Columbia University institute whose goal is to accelerate the pace of translating science into real-life treatments for patients received $38.9 million from the National Institutes of Health (NIH) to expand its work over the next five years. The Irving Institute for Clinical and Translational Research is among 10 institutes nationwide to receive renewed funding, in recognition of their successes during the first five years of the Clinical and Translational Science Awards (CTSA) program, which is administered by the NIH's National Center for Research Resources. The other institutions are Mayo Clinic; Oregon Health & Science University; Rockefeller University; University of California, Davis; University of California, San Francisco; University of Pennsylvania; University of Pittsburgh; University of Rochester; and Yale University. Together, the institutes represent a $498 million renewed commitment on NIH's part to speed translational research nationwide. NIH will release a progress report on the program in August, highlighting research that has emerged from Columbia University Medical Center and other institutes in the CTSA consortium. The grants, which have now been awarded to 60 academic health centers, help scientists collaborate on research that applies to a broad range of diseases. CTSA-funded institutions also work with industry, manufacturers, patient groups, and nonprofit organizations to ensure that potentially life-saving new drugs and devices reach the public faster. Source: EurekAlert! 7/18/11

Standard Therapy Beats Newer Regimens Against H. Pylori in Latin America Study

Helicobacter pylori, the bacterium known to cause peptic ulcers, is also the primary cause of gastric cancer, which is a leading cancer killer globally. A large clinical trial at seven sites across Latin America has now found that a standard three-drug regimen for treating H. pylori is more effective, at least in the population studied, than either of two four-drug regimens that proved superior in studies in Europe and Asia. “This study turns recent literature a bit on its head,” says study coauthor William D. Chey, MD, of the University of Michigan. “Specifically, virtually all other randomized, controlled trials that have tested the four-drug therapy, either sequentially over ten days or concomitantly over five days, have found it superior.” However, most of that literature reports on research done in Italy and Taiwan, Chey points out. The new study, published online July 20 by The Lancet, suggests H. pylori eradication approaches need to be validated locally rather than relying on findings from studies of other populations. The authors speculate that geographic variations in H. pylori’s resistance to antibiotics might account for part of the discrepancy between populations. Coordinated by SWOG, one of the National Cancer Institute’s cooperative groups, the study took place at both urban and rural sites in Chile, Colombia, Costa Rica, Honduras, Mexico, and Nicaragua. Researchers randomly assigned 1,463 volunteers infected with H. pylori to one of three treatment regimens, making this the largest trial of its type ever conducted. Six weeks after starting treatment, each participant was tested for H. pylori infection. Of those volunteers who took a three-drug, 14-day treatment, 82.2 percent were infection-free. Only 73.6 percent of the group on a five-day regimen and 76.5 percent of those on the 10-day regimen--the same therapies earlier studies had found superior--had overcome their infection. The trial was part of an initiative exploring ways to reduce the incidence of gastric cancer. No large studies comparing the effectiveness of H. pylori treatment regimens have been conducted in the United States, and a pressing question now is which regimen should be recommended to U.S. doctors treating H. pylori. “Before this trial I thought I knew the answer to that question,” Chey says. “Now I’m not so sure.” Source: Newswise 7/15/11

Study Uses New Stem Cell Therapy Up to 19 Days After Stroke

The first Texas patient has been enrolled by researchers at the University of Texas Health Science Center at Houston (UTHealth) in the country's first double-blind clinical trial studying the safety and efficacy of an innovative stem cell therapy that can be given up to 19 days after an ischemic stroke. The Phase II study, cleared by the Federal Drug Administration, examines a regenerative therapy developed by Aldagen that uses a patient's own bone marrow stem cells. The therapy, called ALD-401, consists of stem cells that are identified using Aldagen's proprietary technology to isolate cells that express high levels of an enzyme that serve as a marker of stem cells. The cells are administered into the carotid artery. "This represents a new approach using stem cells for stroke," said Sean Savitz, MD, senior investigator for the multicenter study and associate professor of neurology at the UTHealth Medical School. "A major question in the field of stem cell research is whether we can extend the time window for administering stem cells. A longer window increases the number of patients that might be helped." The Houston resident received either placebo or ALD-401 on June 8 at Memorial Hermann-Texas Medical Center after suffering a stroke May 23. Her stroke was caused by previously undiagnosed atrial fibrillation, and while she doesn't know whether or not she received the stem cells in the study, she did not hesitate to join the trial. "I did a lot of research on stem cells online. I was very excited when I heard about the trial. I wanted to participate in the research for me, if possible, and for other people behind me," she said. Savitz' team is working to enroll patients at other area hospitals and facilities into the study. The trial, funded by Aldagen, will enroll a total of 100 patients with unilateral (one-sided) cortical ischemic strokes. Source: EurekAlert! 7/14/11

University to Study Medication for Autism Spectrum Disorders

An experimental drug to treat social withdrawal in children and young adults with autism is being studied in a clinical trial at the University of Illinois at Chicago's (UIC's) Institute for Juvenile Research. Children with autism--or autism spectrum disorders (ASD)--often have difficulty communicating and interacting with others. Although behavioral and psychological interventions are often beneficial, currently there is no medication to address social communication difficulties, a core symptom of ASD. A drug treatment is needed that would address symptoms that are "often disabling for patients and families," says Dr. Edwin Cook, professor of psychiatry and director of autism and genetics at UIC. UIC is the only study site in Illinois and one of 25 sites nationwide. The study is sponsored by Seaside Therapeutics, Inc. The clinical trial will enroll approximately 150 patients diagnosed with autism spectrum disorders between ages 5 and 21 to evaluate the efficacy, safety, and tolerability of STX209 (arbaclofen). Participants in the 22-week study will be randomized to receive either the study drug, STX209, or a placebo. The clinical trial will include screening, treatment, withdrawal of medication, and a follow-up period. Subjects who complete the study may be eligible to enroll in a subsequent open-label study, in which all subjects are treated with STX209. STX209 has been studied in children with fragile X syndrome, a genetic disorder that is the most common identified cause of autism. Previous research has found that from one-quarter to one-half of people with fragile X have autism spectrum disorders, said Cook, principal investigator of the UIC study. Cook's UIC study team includes co-investigator Dr. Fedra Najjar, assistant professor of psychiatry, and study coordinators Sarah Youngkin and Clare Tessman. Source: Newswise 7/12/11

Partners Join Forces to Help University Researchers

For University of Alberta medical drug researchers, the long and winding road from pharmaceutical laboratory discoveries to commercial opportunity has just become straighter and faster. The Centre for Drug Research and Development (CDRD) and TEC Edmonton have signed an affiliation agreement allowing University of Alberta researchers with promising health discoveries to access CDRD's expertise and infrastructure. CDRD is a not-for-profit public/private organization headquartered in Vancouver that is recognized by the Government of Canada as a Centre of Excellence for Commercialization and Research. As such, it provides drug development expertise and infrastructure to researchers from more than 20 leading academic and health research institutions from across Canada to advance commercially promising, early-stage drug candidates. TEC Edmonton is a not-for-profit joint venture between the University of Alberta and the Edmonton Economic Development Corporation. TEC Edmonton is Greater Edmonton's business incubator and accelerator for fledgling high-tech companies of all stripes, including bio-health. On campus, TEC Edmonton helps researchers turn their scientific solutions into business opportunities. It also serves as the exclusive technology transfer agent for the University of Alberta. Says CDRD President and CEO Natalie Dakers: "We are very pleased to be entering into this agreement with our western Canadian neighbors. Through this collaboration, CDRD's pipeline of new health technologies will be enhanced while University of Alberta researchers will gain access to our state-of-the-art drug development platforms coupled with our specialized scientific and business expertise to advance their technologies to a stage where they are sufficiently de-risked for private sector consideration." Responds TEC Edmonton President and CEO Chris Lumb: "TEC Edmonton, as part of its mandate to commercialize emerging technologies in the region, is pleased to partner with CDRD to bring new funding and resources to University of Alberta researchers. Medical innovations drive both better health and new business creation, and the University of Alberta is one of Canada's leading medical research universities. CDRD's specialized expertise will be an important addition to the University of Alberta's capabilities." Source: Marketwire 7/13/11

Pivotal Study in Africa Finds that HIV Medications Prevent HIV Infection

In a result that will fundamentally change approaches to HIV prevention in Africa, an international study has demonstrated that individuals at high risk for HIV infection who took a daily tablet containing an HIV medication--either the antiretroviral medication tenofovir (TDF) or tenofovir in combination with emtricitabine (FTC/TDF)--experienced significantly fewer HIV infections than those who received a placebo pill. These findings are clear evidence that this new HIV prevention strategy, called pre-exposure prophylaxis (or PrEP), substantially reduces HIV infection risk. The study is led by the University of Washington's International Clinical Research Center and involves 4,758 HIV serodiscordant couples, in which one partner has HIV and the other does not, from nine research sites in Kenya and Uganda. "This study is the largest study to date looking at the effectiveness of PrEP," said Dr. Connie Celum, a professor of global health and medicine and the principal investigator of the study, known as the Partners PrEP Study. The study is funded by the Bill & Melinda Gates Foundation. "This study demonstrates that antiretrovirals are a highly potent and fundamental cornerstone for HIV prevention and should become an integral part of global efforts for HIV prevention." Those who received TDF had an average of 62 percent fewer HIV infections and those who received FTC/TDF had 73 percent fewer HIV infections than those who received placebo. TDF and FTC/TDF were statistically similar in their levels of protection against HIV and reduced HIV risk in both women and men. Importantly, PrEP was found to be safe: the rate of serious medical events was similar for those assigned to TDF, FTC/TDF, and placebo. The study was designed to find out whether TDF or FTC/TDF would reduce the risk of acquiring HIV for persons who had an HIV infected sexual partner. Of the couples enrolled in the study, one-third of the HIV uninfected partners were randomly allocated to receive TDF, one-third FTC/TDF, and one-third a matching placebo. The study was double-blinded, and all study participants received a comprehensive package of HIV prevention services, which included intensive safer sex counseling (both individually and as a couple), HIV testing, free condoms, testing and treatment for sexually transmitted infections, and monitoring and care for HIV infection. In the study, adherence to the daily PrEP medication was very high--more than 97 percent of dispensed doses of the study medications were taken. More than 95 percent of participants were retained in study follow-up. The medications used in the Partners PrEP Study, TDF (300 mg) and combination FTC (200 mg)/TDF (300 mg), are marketed by Gilead Sciences, Inc. under the brand names Viread® and Truvada®. They are available generically in many countries at prices as low as approximately 25 cents (U.S.) per tablet. Gilead Sciences donated study medication for, but did not provide funding or otherwise participate in the design, implementation, or analysis of the Partners PrEP Study. Source: EurekAlert! 7/13/11

New Psychotherapy Intervention Improves End-of-Life Experience

Dignity therapy has substantial benefits over standard palliative care and client-centered care, significantly improving quality of life and enhancing the dignity of patients at the end of life while helping their families. The findings of the first randomized trial of this new psychotherapy, published online in The Lancet Oncology, suggest that dignity therapy should be made widely available to all individuals nearing the end of their lives. Despite much progress in improving physical comfort for dying patients, few interventions have been developed to address the emotional, social, and spiritual needs of patients and their families, and this remains an ongoing challenge. Dignity therapy (a unique, individualized psychotherapy) was designed to relieve distress and improve the end-of-life experience by allowing patients to discuss and document things that they would most want known or remembered. A previous Phase I trial suggested that dignity therapy is helpful to nearly all patients. In this study, an international team led by Harvey Max Chochinov from the University of Manitoba, Winnipeg, Canada examined whether dignity therapy could reduce distress and enrich the end-of-life experience in 326 terminally ill patients aged 18 years or older enrolled from hospitals and community settings across Canada, the U.S., and Australia. Patients were randomly assigned to dignity therapy (108 patients), client-centered care (107), or palliative care (111). Scores on scales measuring spiritual wellbeing, dignity, depression, and quality of life were completed at the start and end of the study. Patients' end-of-life experiences were also assessed using a self-reported survey after the study ended. Patients receiving dignity therapy were significantly more likely to report that the approach was helpful, improved their quality of life, enhanced their sense of dignity, changed how their family saw and appreciated them, and provided benefits to their relatives compared to those given conventional care. Dignity therapy was also significantly better than client-centered care at improving spiritual wellbeing, and significantly better than palliative care at reducing sadness and depression. However, no significant differences were noted in distress levels between the groups. Source: EurekAlert! 7/6/11

Massage Eases Low Back Pain in Randomized Controlled Trial

Massage therapy helps ease chronic low back pain and improve function, according to a randomized controlled trial that the Annals of Internal Medicine published in its July 5 issue. The first study to compare structural and relaxation (Swedish) massage, the trial found that both types of massage worked well, with few side effects. "We found that massage helps people with back pain to function even after six months," said trial leader Daniel C. Cherkin, PhD, a senior investigator at Group Health Research Institute, a Seattle-based, consumer-governed, nonprofit healthcare system. Better function means they are more able to work, take care of themselves, and be active. "This is important because chronic back pain is among the most common reasons people see doctors and alternative practitioners, including massage therapists. It's also a common cause of disability, absenteeism, and 'presenteeism,' when people are at work but can't perform well." The trial enrolled 400 Group Health Cooperative patients who had had low back pain for at least three months. Their pain was "nonspecific," meaning with no identified cause. They were randomly assigned to one of three treatments: structural massage, relaxation massage, or usual care. Usual care was what they would have received anyway, most often medications. The hour-long massage treatments were given weekly for 10 weeks. At 10 weeks, more than one in three patients who received either type of massage—but only one in 25 patients who got usual care—said their back pain was much better or gone. Also at 10 weeks, a questionnaire showed nearly twice as many massage patients (around two thirds) as usual-care patients (more than one third) were functioning significantly better than at the trial's outset. Patients in the massage groups spent fewer days in bed, were more active, and used less anti-inflammatory medication than did those with usual care. "As expected with most treatments, the benefits of massage declined over time," Dr. Cherkin said. "But at six months after the trial started, both types of massage were still associated with improved function." After one year, the benefits of massage were no longer significant. Drs. Cherkin's coauthors are Karen J. Sherman, PhD, MPH, a senior investigator at Group Health Research Institute; Andrea J. Cook, PhD, an assistant investigator of Group Health Research Institute and the University of Washington; biostatisticians Robert Wellman, MS, and Eric A. Johnson, MS, project manager Janet Erro, RN, MN, and analyst/programmer Kristin Delaney, MPH, of Group Health Research Institute; Janet Kahn, PhD, of the University of Vermont School of Medicine in Burlington; and Richard A. Deyo, MD, MPH, of Oregon Health and Science University in Portland. The National Center for Complementary and Alternative Medicine, part of the National Institutes of Health, funded this study. Source: EurekAlert! 7/4/11

Clinical Study Targeting Insomnia Uses New Technology

Wake Forest Baptist Medical Center is conducting the first ever, randomized, controlled clinical research study in the country using Brainwave Optimization™ to treat people with insomnia. Brainwave Optimization is a noninvasive technology that helps the brain balance itself for optimal performance. The study is made possible by a research grant from Brain State Technologies®, a company based in Scottsdale, Ariz., whose founder developed Brainwave Optimization, or what is technically known as High-resolution, Relational, Resonance-based, Electroencephalic Mirroring™. “Energetic imbalance of the brain, with dominance of the right or left hemispheres, can be seen in conditions such as trauma or stress, for which insomnia is often an accompanying symptom,” said Charles Tegeler IV, MD, professor of neurology and the primary investigator for the study. “This new technology is intended to facilitate greater balance and harmony in brain functioning, which may result in improved symptoms. In effect, we are allowing the brain to look at itself in the mirror and see itself in an optimized, energetic state. Those areas that are out of balance then begin to work toward a more functional state.” Tegeler said the study is focusing only on insomniacs because it is such a common condition in the general population and because it is a condition for which improvement can be easily measured. Twenty people who tested for moderate to severe insomnia were enrolled and underwent baseline testing. The study participants were randomly placed into two separate groups. The first group underwent eight to 12 Brainwave Optimization sessions each while the second group continues as is, acting as the control group. Once the data have been collected, the second group will also undergo the sessions. One study participant described his experience as having “a big reset button that’s been hit.” While Brainwave Optimization has already been used by more than 32,000 people around the world, this clinical study at Wake Forest Baptist is the first attempt to look at a specific health problem in a controlled setting in order to be able to measure outcomes and results. Source: Newswise 6/30/11

UCB Launches Research Alliance with Harvard University

Belgium-based biopharmaceutical company UCB and Harvard University in the U.S. officially launched their Research Alliance during a signing ceremony that took place in Boston in late June in the presence of his Royal Highness Prince Philippe of Belgium. The alliance creates a drug discovery bridge between industry and academia, with Harvard scientists continuing their research that holds potential for the development of new therapeutic modalities in clinical medicine. The strategy underlying the collaborative alliance is to advance ongoing Harvard research projects along the drug development pathway. "Harvard's world-class academic research is an excellent fit with UCB's world-class antibody research and central nervous system pharmacology," said Ismail Kola, president of UCB NewMedicines. The first research project funded under the Harvard-UCB alliance is a collaboration with Gokhan S. Hotamisligil, MD, PhD, chair of the Department of Genetics and Complex Diseases in the Harvard School of Public Health, to generate and develop antibodies against a target with potential application in a range of diseases, including diabetes and metabolic disorders. UCB will bring its expertise in antibody generation and medicinal chemistry into the alliance, and will provide up to $6 million over two years to fund specific innovative research projects led by Harvard scientists. The collaboration focuses on central nervous system and immunology, two key research domains for UCB. Source: PRNewswire 6/30/11

Researchers Develop New Gene Therapy for Heart Failure

Researchers at Mount Sinai School of Medicine have found in a Phase II trial that a gene therapy developed at Mount Sinai stabilized or improved cardiac function in people with severe heart failure. Patients receiving a high dose of the therapy, called SERCA2a, experienced substantial clinical benefit and significantly reduced cardiovascular hospitalizations, addressing a critical unmet need in this population. The data are published online in the June 27 issue of the American Heart Association journal Circulation. SERCA2a is delivered via an adeno-associated virus vector—an inactive virus that acts as a medication transporter—into cardiac cells. The therapy stimulates production of an enzyme within these cells that enables the heart to pump more effectively in people with advanced heart failure. After one year, patients who were administered a high dose SERCA2a demonstrated improvement or stabilization. Gene therapy with SERCA2a was also found to be safe in this sick patient population, with no increases in adverse events, disease-related events, laboratory abnormalities, or arrhythmias compared to placebo. The CUPID (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) trial is a randomized, double-blind, placebo-controlled study, which enrolled 39 patients with advanced heart failure to study the safety and efficacy of SERCA2a. Patients were randomized to receive SERCA2a gene delivery in one of three doses or placebo, and were evaluated over one year. The treatment is delivered directly to the patient's heart during a routine outpatient cardiac catheterization procedure. The CUPID Trial is funded by Celladon Corp. Source: EurekAlert! 6/28/11

Drug Shows Improved Kidney Function for Type 2 Diabetics

A new anti-inflammatory drug used by patients with type 2 diabetes improved their kidney function during a year-long study involving researchers from University of Texas (UT) Southwestern Medical Center. The study findings, reported in the New England Journal of Medicine, mark the first time a drug therapy has led to improved kidney function for patients with type 2 diabetes and chronic kidney disease. Previous studies have identified drugs that slowed the deterioration of kidney function, said Dr. Robert Toto, director of the Houston J. and Florence A. Doswell Center for the Development of New Approaches for the Treatment of Hypertension at UT Southwestern. The study involved 227 adult patients with type 2 diabetes and chronic kidney disease. They were divided into four groups--three receiving different dosages of bardoxolone methyl, an anti-inflammatory drug, and the fourth group receiving a placebo and acting as a control. The patients were tracked for 56 weeks, with measurements of their kidney function taken every four weeks. At study-highlighted weeks 24 and 52, researchers saw an overall significant increase in the estimated glomerular filtration rates, which are measurements of how well the kidneys are functioning, for the patients receiving the drug. At 56 weeks, four weeks after researchers stopped administering the drug, a third measurement showed that patients continued to maintain a slightly higher level of kidney function compared to baseline measurements taken at the study's start. The next step, Dr. Toto said, will be a longer-term clinical trial with a larger patient pool, necessary to confirm the effectiveness and safety of using the anti-inflammatory in people with type 2 diabetes and chronic kidney disease. He noted that it is important to interpret the current study's results cautiously, given the small number of patients and one-year duration of the study. Dr. Philip Raskin, professor of internal medicine who leads the University Diabetes Treatment Center and the diabetes clinic at Parkland Memorial Hospital, also participated in the study, as did researchers at Reata Pharmaceuticals, Renal Associates in San Antonio, the University of Alabama-Birmingham, and Statistics Collaborative in Washington, D.C. The study was funded by Reata. Source: EurekAlert! 6/24/11

New Drug Represents Breakthrough in Treatment of Hepatitis C

The drug telaprevir (Incivek) provides a dramatic improvement in the treatment of the most common form of hepatitis C virus (HCV) infection, says an international team of investigators led by Dr. Ira M. Jacobson of NewYork-Presbyterian Hospital/Weill Cornell Medical Center. Their study, published in the New England Journal of Medicine, led to approval of the agent for patient use by the U.S. Food and Drug Administration on May 23. Results of the ADVANCE trial showed that telaprevir combined with standard therapy (pegylated-interferon and ribavirin) cured the virus in 75 percent of patients treated compared to 44 percent of patients who received standard therapy alone. Furthermore, of the nearly 60 percent of telaprevir-treated patients who had undetectable viral levels at weeks 4 and 12 of treatment, and who were eligible by the terms of the study to receive 24 weeks of total treatment--half the time required for standard treatment--approximately 90 percent were cured. In the ADVANCE clinical trial, 1,088 untreated patients diagnosed with HCV genotype 1 were assigned to one of three treatment arms: standard therapy for 48 weeks, or telaprevir combined with standard therapy for eight or for 12 weeks, followed by standard therapy alone for a total treatment duration of either 24 or 48 weeks. The researchers found that sustained virologic response occurred in significantly more patients receiving 12 weeks (75 percent) or eight weeks (69 percent) of telaprevir than with standard therapy alone (44 percent). In all, 58 percent of telaprevir-treated patients received 24 weeks of total treatment. There were substantial benefits of telaprevir in subgroups of patients who do not generally respond well to standard therapy. For example, 62 percent of participating African-American patients achieved a viral cure with the telaprevir-based regimen, compared to 25 percent of African-Americans treated with standard therapy. In addition, 62 percent of patients with advanced liver cirrhosis achieved a viral cure with telaprevir compared to 33 percent of similar patients on standard therapy. Telaprevir was developed by Vertex Pharmaceuticals Inc. in collaboration with Tibotec Pharmaceuticals and Mitsubishi Tanabe Pharma. Vertex provided funding for the study. Source: Newswise 6/23/11

TB Trial Highlights Challenges of Childhood Immunization Schedule

A new vaccine to combat tuberculosis (TB) is less effective at stimulating an immune response when administered to Gambian infants in combination with the routine immunization schedule, according to clinical trial results published in Science Translational Medicine. The findings may have important implications for designing the most effective immunization schedules for children, and also for the design of future clinical trials of the new vaccine. Standard childhood vaccinations are routinely given as part of a schedule known as the Expanded Program on Immunization (EPI), which helps to improve vaccine coverage in the population by reducing the number of visits to the clinic required. The schedule includes vaccines for diphtheria, tetanus, and whooping cough, as well as the current vaccine for TB, Bacille Calmette-Guérin (BCG). The aim is to vaccinate children in early infancy, in order to protect them from disease as early as possible. MVA85A is a vaccine designed to be given after BCG to boost the body's immune response and improve protection against TB. Originally developed by Dr. Helen McShane at the University of Oxford with funding from the Wellcome Trust and the Medical Research Council (MRC), it has already been shown to be safe and capable of eliciting powerful immune responses in clinical trials in adults in the U.K., Gambia, and South Africa. This is the first trial to evaluate safety of the vaccine in infants. The purpose of the study was to assess whether MVA85A can stimulate immune responses against the TB bacteria in infants and whether it could feasibly be given at the same time as other childhood vaccines as part of the EPI. The randomized trial, funded by the Wellcome Trust, the MRC, and the European Commission, involved 214 healthy, 4-month-old infants who had already received BCG at birth. Children were given either EPI alone, MVA85A alone, or MVA85A in conjunction with EPI. Overall, MVA85A was deemed to be safe and well-tolerated, and it induced a strong immune response. Importantly, the responses to the standard EPI vaccines were not affected by giving MVA85A at the same time. However, the immune response to MVA85A was lower in infants who received it in conjunction with EPI vaccines compared to those that received the new vaccine alone. The study will help researchers determine the best way to integrate MVA85A into routine infant immunization schedules in future. Source: EurekAlert! 6/21/11

$5 Million Agreement Links University to Translational Research Program

University of Missouri (MU) officials announced in June a $5 million grant agreement with the Wallace H. Coulter Foundation, through the Foundation's Translational Partnership Award Program. The goal of the partnership is to develop technologies that will save, extend, and improve patient lives and suffering from any disease or condition in any country around the world. The funds will be used to support collaborative projects between biomedical engineers and clinicians to take research discoveries to clinical practice. "This is a major recognition of the University of Missouri and its researchers, as only about 15 universities have been given this award," said Jinglu Tan, professor and chair of biological engineering. "This award will position us to become a national leader in translational biomedical research by building on existing successes in biomedical engineering research, cooperation between engineering and medicine researchers, and technology transfer projects. This award will allow our scientists to take their discoveries and develop them for use in the hospital or other clinical settings in an effort to improve the quality of life for many people." The agreement between MU and the Coulter Foundation stipulates that the foundation will provide $666,667 per year for five years, while MU will match that money with $333,333 each year, for a total amount of $5 million over the next five years. An oversight committee, consisting of stakeholders in translational research, will be formed to decide how to award the money to MU bioengineering faculty who are seeking to commercialize their research discoveries in partnership with clinicians. Source: EurekAlert! 6/21/11

Research Foundation Awards $3.45 Million to Top Young Clinical Investigators

The Damon Runyon Cancer Research Foundation named five new Damon Runyon Clinical Investigators at its spring 2011 Clinical Investigator Award Committee review. The recipients of this prestigious three-year award are outstanding early-career physician-scientists conducting patient-oriented cancer research at major research centers under the mentorship of the nation's leading scientists and clinicians. Each will receive $450,000 to support the development of his/her cancer research program. The foundation also awarded Continuation Grants to four Damon Runyon Clinical Investigators. Each award will provide an additional two years of funding totaling $300,000. The Continuation Grant is designed to support clinical investigators who are approaching the end of their original awards and need extra time and funding to complete a promising avenue of research or initiate/continue a clinical trial. This program is possible through the generous support of the William K. Bowes, Jr. Foundation, and Connie and Robert Lurie. The Clinical Investigator Award program is specifically intended to help address the shortage of physicians capable of translating scientific discovery into new breakthroughs for cancer patients. In partnerships with industry sponsors and through its new Accelerating Cancer Cures initiative, the Damon Runyon Cancer Research Foundation has committed more than $38 million to support the careers of 58 physician-scientists across the United States since 2000. Source: EurekAlert! 6/21/11

Landmark Clinical Trial of Catheter Treatment for Atrial Fibrillation

Loyola University Medical Center in Illinois is enrolling patients in a major clinical trial of treatments for a common heart rhythm disorder called atrial fibrillation (A-fib). The study is comparing traditional drug therapy with a newer treatment called catheter ablation. Standard drug therapy has been available for more than 30 years. It includes drugs to prevent the heart rate from going too fast and drugs to stop the abnormal heart rhythm. However, medications do not always work, and they can cause side effects that significantly impair patients' quality of life. Moreover, medications do not halt the underlying progression of the disease. In the newer ablation procedure, an electrophysiologist destroys areas of heart tissue that are responsible for the erratic electrical signals. A catheter (thin flexible tube) is guided through blood vessels to the heart. The tip of the catheter delivers radiofrequency energy that heats and destroys tissue. Loyola is participating in an international, multicenter trial called CABANA (Catheter Ablation versus Antiarrhythmic Drug Therapy for Atrial Fibrillation Trial). The study will enroll 3,000 A-fib patients. Half will be randomly assigned to receive drug therapy and half will receive ablation. A-fib is the most common form of irregular heartbeat. Electrical signals, which regulate the heartbeat, become erratic. Instead of beating regularly, the upper chambers of the heart quiver. Not all the blood gets pumped out, so clots can form. A-fib can lead to strokes and heart failure. A-fib symptoms include heart palpitations, dizziness, chest pain, fatigue, shortness of breath, fainting, and lightheadedness. Patients who were enrolled in an earlier catheter ablation trial had already failed one or more drug treatments. By comparison, patients in CABANA need not have previously failed a drug; they begin treatment at an earlier stage of their disease. The main outcome studied in the earlier trial was the percentage of patients who remained free of A-fib episodes or symptoms. CABANA is designed primarily to determine whether ablation patients live longer than patients treated by medications. Source: Newswise 6/14/11

Drug Shows Promise for Treating Dermatomyositis

A multicenter pilot study of etanercept for treatment of dermatomyositis found no major safety concerns and many patients treated with the drug were successfully weaned from steroid therapy. These results are encouraging, but larger studies are needed to further investigate the safety and efficacy of etanercept. Results of this clinical trial are available in Annals of Neurology, a journal published by Wiley-Blackwell on behalf of the American Neurological Association. Dermatomyositis is a type of inflammatory myopathy that causes inflammation and progressive weakness in muscles, and is often accompanied by a purple or red skin rash. In some patients, the standard treatment for dermatomyositis is prednisone, but many patients continue to experience disabling weakness and unpleasant side affects associated with the steroids. "Given the positive response of other inflammatory diseases to etanercept, our pilot study aimed to assess the safety and tolerability of this drug in dermatomyositis, along with the feasibility of a forced prednisone taper," said Anthony Amato, MD, with the Department of Neurology at Brigham and Women's Hospital and Harvard Medical School in Boston, Mass. Researchers conducted a double-blind, placebo-controlled trial of etanercept (50 mg weekly) in 16 patients with dermatomyositis. The year-long trial randomized 11 participants to etanercept and five to placebo, with subjects tapered off prednisone in a standardized schedule over the first 24 weeks of the study. Trial results revealed no significant increase in adverse event rates in the treatment group compared to placebo. Of the 11 subjects treated with etanercept, five were successfully tapered from steroid therapy, with a median time to treatment failure (inability to wean off prednisone on schedule) in this group of 358 days. All placebo subjects were treatment failures with a median of 148 days to failure of treatment. Researchers reported that five etanercept-treated participants and one placebo-treated participant developed a worsening rash. After week 24, the median of the average prednisone dosage was 29.2 mg/day in the placebo group and 1.2 mg/day in the etanercept cohort. The authors noted that the small sample size and allowance of rescue treatment in this trial limit conclusions of etanercept efficacy to those related to steroid-sparing. "Etanercept was safe and well-tolerated in participants in our one-year study," concluded Dr. Amato. "Further study of the safety and efficacy of etanercept as a dermatomyositis treatment is warranted." Source: EurekAlert! 6/17/11

Glowing Dots a Potential Cancer Diagnostic Tool

The U.S. Food and Drug Administration (FDA) has approved the first clinical trial in humans of a new technology: Cornell Dots, brightly glowing nanoparticles that can light up cancer cells in PET-optical imaging. A paper describing this new medical technology appears in the July 2011 issue of the Journal of Clinical Investigation. This is a collaboration between Memorial Sloan-Kettering Cancer Center, Cornell University, and Hybrid Silica Technologies, a Cornell business start-up. For the first time, scientists report a uniquely advanced and comprehensive characterization of Cornell Dots--an ultra-small, cancer-targeted, multimodal silica nanoparticle that was approved recently as an investigational new drug by the Food and Drug Administration for a first-in-human clinical trial. Cornell Dots are silica spheres less than 8 nanometers in diameter that enclose several dye molecules. (A nanometer is one-billionth of a meter, about the length of three atoms in a row.) The silica shell, essentially glass, is chemically inert and small enough to pass through the body and out in the urine. For clinical applications, the dots are coated with polyethylene glycol (PEG) so the body will not recognize them as foreign substances. To make the dots stick to tumor cells, organic molecules that bind to tumor surfaces or even specific locations within tumors can be attached to the PEG shell. When exposed to near-infrared light, the dots fluoresce much brighter than dye to serve as a beacon to identify the target cells. The technology, the researchers say, enables visualization during surgical treatment, showing invasive or metastatic spread to lymph nodes and distant organs, and can show the extent of treatment response. The inventors of the dots have formed the company Hybrid Silica Technologies (HST) to commercialize the invention. The combined team of cancer center, Cornell, and HST researchers is now in the process of forming a new commercial entity in New York City that will help transition the research into commercial products that will benefit cancer patient care. The scientists have performed real-time imaging of lymphatic drainage patterns and particle clearance rates, as well as sensitively detected nodal metastases. Nodal mapping is now being pursued under a new award of a BioAccelerate NYC Prize from the Partnership for New York City and the New York City Economic Development Corp., which is expected to lead to another clinical trial in humans. Source: EurekAlert! 6/13/11

Breast Cancer Drug Pushes Colon Cancer Cells to Their Death

A new treatment for colon cancer that combines a chemotherapy agent approved to treat breast cancer and a cancer-fighting antibody is ready for clinical trials, according to Penn State College of Medicine researchers. Wafik S. El-Deiry, MD, PhD, a professor and chief of hematology/oncology, and his team have tested lapatinib, a targeted chemotherapy agent currently approved for breast cancer treatment, in a new combination with artificial antibodies that mimic a natural cancer-fighting protein produced in the human body. The monoclonal antibodies mapatumumab and lexatumumab act similarly to TRAIL--tumor necrosis factor [TNF]-related apoptosis-inducing ligand--a naturally occurring molecule in the body that tells a cell it is time to die. TRAIL sets a process in motion that targets and shuts down tumor cells and keeps them from spreading. "These are therapeutic antibodies that are manufactured very efficiently, and given to patients," said El-Deiry, who is also the associate director for translational research with Penn State's Cancer Institute. The TRAIL receptors--death receptors--on the cancer cells respond to TRAIL by dying. The artificial antibodies act as surrogates of TRAIL by activating the same signaling pathway resulting in tumor cell death. The monoclonal antibodies have an advantage over TRAIL because they remain active in the body for a longer period of time. TRAIL receptor antibodies last for less than 30 minutes, while the artificial monoclonal antibodies last for about nine days. Although the antibodies can act similarly to TRAIL, they do not completely substitute for TRAIL, and ultimately which one gets used in what situation is still being tested in clinical trials. Lapatinib increases the amount of "death receptor" protein available for TRAIL to do its job--killing off cancerous cells in mice--El-Deiry and his colleagues report in a recent issue of Science Translational Medicine. Source: EurekAlert! 6/8/11

Vaccine First to Show Improved Survival Rates for Metastatic Melanoma

For patients with advanced melanoma, the results of a large clinical trial show that a vaccine combined with the immune-boosting drug Interleukin-2 can improve response rate and progression-free survival. The findings of the study were published in the June 2 issue of the New England Journal of Medicine. This marks the first vaccine study in the disease and one of the first in all cancers to show clinical benefit in a randomized Phase III clinical trial. According to Dr. Howard Kaufman, director for the Rush University Cancer Center and study coinvestigator, “If we can use the body’s own defense system to attack tumor cells, we provide a mechanism for ridding the body of cancer without destroying healthy tissue." Therapeutic cancer vaccines, unlike typical vaccines that prevent infections, are meant to jump-start the immune system to help it battle existing tumors. Rush was one of 21 centers in the U.S. participating in the trial. Researchers randomly assigned 185 patients with metastatic melanoma to either a combination of the peptide vaccine, which is a small portion of protein that is present on the surface of the melanoma cancer cells, and Interleukin-2, a drug that activates the immune system, or a high dose of Interleukin-2 alone. The peptide vaccine, known as gp100:209-217 (200M), works by stimulating the patient’s T-cells. The injection primes the immune system to recognize the protein and activates the body’s cytotoxic T-cells to recognize the antigens on the surface of the cancerous tumor. The T-cells then secrete enzymes to seek out and destroy the tumor cell’s membrane. The immune-boosting drug, Interleukin-2, enhances the vaccine's effectiveness by stimulating the production of lymphocytes. More circulating lymphocytes means there are more cells available to do the job the vaccine has educated them to do. Ninety-four patients in the Interleukin-2 arm of the study were enrolled and 93 were treated and evaluated for response. In the Interleukin-2 and peptide vaccine combination arm of the study, 91 were enrolled, 86 were treated, and 85 were evaluated for response. About 16 percent of study participants given the vaccine and Interleukin-2 combination saw tumors shrink by 50 percent or more, compared to 6 percent given Interleukin-2 alone. Those in the vaccine and drug combination group also had slightly longer progression-free survival rates of 2.2 months compared to 1.6 months. Patients given the combination also lived an average of nearly seven months longer than those only give Interleukin-2--17.8 months longer compared to 11.1 months. In order for this vaccine to work, patients had to have a particular tissue type, called HLA-A2, which is present in about half of whites. The study was partially funded by the National Cancer Institute. Source: Newswise 6/3/11

Researchers to Lead Pan-Canadian Trial on Early-Stage Oral Cancer

Researchers from the University of British Columbia's (UBC's) Faculties of Medicine, Science, and Dentistry are leading a $4.7-million pan-Canadian clinical trial aimed at improving outcomes for patients undergoing surgery for oral squamous cell cancers. Funded by the Terry Fox Research Institute, the Canadian Optically Guided Approach for Oral Lesions Surgical Trial, or COOLS Study, involves universities and hospitals in nine Canadian cities. The COOLS Study will investigate the effectiveness of a fluorescence visualization (FV), or "blue light," to distinguish between healthy tissues from tumors or precancerous cells in the mouth. Under the blue light, normal tissue generates a fluorescence which is absent in tumor or precancerous tissue. "In work we've conducted to date in Vancouver, there has been almost no recurrence where surgery followed the contour of the lesion shown by using FV-guided surgery. Working together with surgeons, pathologists, research staff, and scientists, this...study will enable us to test the approach on a broader cohort of patients at sites across the country and obtain the evidence required to change current practice," says principal investigator Dr. Catherine Poh, a senior scientist with BC Cancer Agency, an associate professor at UBC, and a consulting dentist at Vancouver General Hospital. Source: EurekAlert! 5/27/11

Clinical Trial of Malaria Vaccine Begins in Africa

A vaccine known as RTS,S, developed by GlaxoSmithKline Biologicals and the PATH Malaria Vaccine initiative, is currently in Phase III clinical trials and has previously reduced episodes of malaria in infants and young children by more than 50 percent. A University of Liverpool team, in collaboration with the University College of Medicine, Malawi, is working in Blantyre over the next three years to investigate how to maximize the vaccine's effectiveness when delivered through a childhood immunization program. The team will examine the performance of the vaccine as it is administered to infants at different stages between birth and nine months of age, alongside the standard set of immunizations used in national programs for young children. Studies have so far suggested that the vaccine could be safely integrated with other vaccines in the World Health Organization's Expanded Program for Immunization schedule. The research is funded by GlaxoSmithKline Biologicals, Belgium. Source: EurekAlert! 5/25/11

NIH Grant Supports Progress Toward Trials on Lysosomal Storage Disease

Investigators at Nationwide Children's have received a grant from the National Institutes of Health (NIH) to help move a therapy for mucopolysaccharidosis (MPS) IIIB that has been shown effective in mice toward clinical trials in humans. MPS IIIB, also known as Sanfilippo syndrome B, is a lysososmal storage disease caused by deficiency in the essential enzyme NAGLU. Children with MPS IIIB appear normal at birth, but develop severe, progressive developmental delay and neurological disorders by 2 years of age. MPS IIIB is a fatal disease and there is currently no treatment available. "To date, the greatest challenge in developing therapies for MPS IIIB has been the presence of the blood-brain barrier, which prevents therapeutics from entering the central nervous system," said Haiyan Fu, PhD, the project's lead investigator. For more than a decade, Dr. Fu's team in the Center for Gene Therapy in the Research Institute at Nationwide Children's Hospital has been focusing on developing gene delivery approaches to efficiently restore the central nervous system NAGLU activity, which is missing in MPS IIIB patients. Using a single intravenous injection of a recently characterized viral vector, AAV9, which has the unique ability to cross the blood-brain barrier, Dr. Fu's team has achieved the best long-term therapeutic benefits to date in adult MPS IIIB mice. This strategy has resulted in correction of cognitive and motor function and extended survival in these mice, which, like humans with MPS IIIB, lack the NAGLU enzyme. The NIH funding, awarded to Dr. Fu and coinvestigator Kevin Flanigan, MD, will allow the team to complete necessary preclinical studies and to submit an investigational new drug application to the U.S. Food and Drug Administration for a Phase I/II AAV9 gene therapy clinical trial in patients with MPS IIIB. "Importantly, the intravenous rAAV9 gene delivery procedure is minimally invasive and is therefore a clinically relevant approach," said Dr. Fu. A research grant through Ben's Dream – The Sanfilippo Research Foundation was critical in supporting Dr. Fu's team while it was pursuing the NIH grant application. Source: EurekAlert! 5/25/11

Heart Drugs Ineffective in Treating Pulmonary Arterial Hypertension

Despite their beneficial effects in treating heart disease, neither aspirin nor simvastatin appear to offer benefit to patients suffering from pulmonary artery hypertension (PAH), according to a National Institutes of Health (NIH)-funded study conducted at four U.S. medical centers. This was the first NIH-funded randomized clinical trial in PAH. The results of the study were presented at the ATS 2011 International Conference in Denver, Colo. PAH is a progressive, incurable disease characterized by increased blood pressure in the arteries of the lungs, which causes shortness of breath, dizziness, and fatigue, and can lead to heart failure and death. PAH can occur on its own or be associated with other conditions, such as connective tissue diseases and congenital heart disease. Although both aspirin and simvastatin are effective in many types of cardiovascular disease, these drugs have not been well-studied in the treatment of PAH, said Steven Kawut, MD, MS, lead author and associate professor of medicine and epidemiology at the University of Pennsylvania School of Medicine. The study was designed to determine if the drugs could be effective in the treatment of patients with PAH. "Surprisingly, we found no evidence that aspirin or simvastatin had beneficial clinical effects in this population, and the study was terminated early by the National Heart Lung and Blood Institute upon the recommendation of the data and safety monitoring board," said Dr. Kawut, who is also director of the university's Pulmonary Vascular Disease Program. "The results of this study do not support the routine treatment of PAH with these medications." Researchers enrolled 65 patients in this placebo-controlled trial and randomized them into four groups: one in which patients received aspirin, one in which patients received simvastatin, one in which patients received both drugs, and one in which patients received neither drug. The main outcome, six-minute walk distance (6MWD), tended to be lower in the group taking simvastatin at six months. There was no significant difference in the 6MWD between the group taking aspirin and the group taking placebo. According to Kawut, "The findings show the importance of subjecting traditional cardiovascular therapies and drugs which appear effective in the laboratory to placebo-controlled, [randomized clinical trials] in humans before recommending their use." Source: EurekAlert! 5/18/11

Existing Drug Treatment Reduces Pain in Young Sickle Cell Anemia Patients

A cancer drug already used to treat adults and school-age children with sickle cell anemia is safe and significantly reduces pain and other complications of the disease in children as young as 9 months, according to a national study involving a University of Texas (UT) Southwestern Medical Center researcher. Pediatric researchers at UT Southwestern and 13 other academic medical centers say hydroxyurea should be offered to all young children with sickle cell anemia, regardless of disease severity and clinical symptoms. The findings of the Pediatric Hydroxyurea in Sickle Cell Anemia, or BABY HUG, trial appear online and in the May 14 edition of the Lancet. "We've offered hydroxyurea at Children's since 1992 to severely involved patients with frequent or severe complication down to age 3. On the basis of the BABY HUG study's findings, our sickle cell team has made a conscious decision to now offer hydroxyurea to all sickle cell anemia patients in the first year of life," said study coauthor Dr. Zora Rogers, professor of pediatrics at UT Southwestern and clinical director of the general hematology and bone marrow failure program at Children's Medical Center Dallas. The findings, Dr. Rogers said, likely will change how all medical professionals treat very young children with sickle cell anemia. In BABY HUG, researchers wanted to determine whether hydroxyurea therapy would prevent early organ damage in very young children with sickle cell anemia. From October 2003 to September 2007, researchers enrolled 193 children between the ages of 9 and 19 months and randomly assigned each to receive hydroxyurea or the placebo for two years. A total of 167 (including 12 at Children's) completed the trial--the first randomized, double-blind trial to examine the drug's effect in very young children with sickle cell anemia. "We found a decrease in chest syndrome and hospitalization among trial participants who received hydroxyurea," said Dr. Rogers, adding that the findings represent the culmination of 15 years of work at UT Southwestern and Children's. "We used to offer hydroxyurea as secondary prevention, but with these findings, it could become the primary preventive measure." In addition to UT Southwestern's participation, researchers from St. Jude's Children's Research Hospital; SUNY Downstate Medical Center; University of Mississippi Medical Center; Johns Hopkins University School of Medicine; Children's National Medical Center; Howard University College of Medicine; Duke University Medical Center; Medical University of South Carolina; University of Miami; Emory University School of Medicine; Children's Hospital of Michigan; University of Alabama at Birmingham; Medical College of Georgia; the National Heart, Lung and Blood Institute; and the Clinical Trials & Surveys Consortium contributed to the study. The National Heart, Lung and Blood Institute, and the National Institute of Child Health and Human Development supported the study. Source: EurekAlert! 5/12/11

Lessons From Major Heart Trial Need Implementation

A NewYork-Presbyterian Hospital/Weill Cornell Medical Center review of almost 500,000 cardiac cases nationally shows that the clinically indicated medical therapy reported in a widely publicized study was lost in translation to real-world heart care after its publication. The researchers report in the May 11 issue of the Journal of the American Medical Association that medical therapy given to patients who received a heart stent improved less than 3 percent as a result of the Clinical Outcomes Utilizing Revascularization and Aggressive Drug Evaluation (COURAGE) trial. Overall, fewer than half of all patients received appropriate treatment with the combination of common cardiac drugs used in the COURAGE trial, such as aspirin, before their stenting procedure, and almost one-third did not receive these drugs afterward. "We find that an expensive and highly publicized clinical trial had a very limited effect on the clinical practice of providing optimal medical therapy, and this snapshot of what is happening in the real world should be a call for physicians, as well as policy makers, to look at how patient care can be improved," says the study's lead author, Dr. William Borden, assistant professor of medicine and of public health at Weill Cornell Medical College, and a cardiologist at the Ronald O. Perelman Heart Institute of NewYork-Presbyterian Hospital/Weill Cornell Medical Center. In this study, the research team, which includes investigators from the University of California, San Francisco, Duke Clinical Research Institute, and the University of Missouri, sought to see if heart care changed after publication of the $33.5 million COURAGE clinical trial in the New England Journal of Medicine in 2007. While previous investigators have shown that translation of clinical trials into patient practice has been "suboptimal," no one had looked at whether this is true in patients who receive a stent through percutaneous coronary intervention (PCI). To find out, the researchers studied 1,013 U.S. hospitals in what they believe to be the largest PCI registry in the United States. They analyzed clinical data on 467,211 patients who had received a stent between 2005 and 2009 to examine changes in the use of optimal medical therapy before PCI and at the time of discharge, both before and after the March 26, 2007, publication of the COURAGE trial. They determined that the use of optimal medical therapy in all patients between 2005 and 2009 was 44.2 percent before PCI, and 65 percent upon discharge after patients received their stent. Before results of COURAGE were known, medical therapy was used before PCI in 43.5 percent of patients, and after COURAGE was published, the rate rose to 44.7 percent. After PCI, at discharge, medical therapy was prescribed to 63.5 percent of patients before COURAGE was published, and 66 percent after. That means that the wide publicity that followed publication of the study resulted in a net benefit of 1.2 percent increase in use of optimal medical therapy before a stent was inserted, and 2.5 percent after the procedure, says Dr. Borden. "While this is a statistically significant result given that thousands of patients were included in the study, it is not clinically significant," he says. The study was funded by the National Cardiovascular Data Registry, an outcomes-based quality improvement initiative of the American College of Cardiology Foundation. Source: EurekAlert! 5/10/11

Researchers Find New Treatment for Constipation

Constipation is definitely not a glamorous topic. In reality, it affects nearly 30 million Americans and costs more than $1 billion annually to evaluate and treat. While not often life-threatening, the pain, bloating, discomfort, and straining associated with constipation lead sufferers to focus on one thing--relief. Mayo Clinic researchers recently had success in the clinical trial of a new medication shown to provide relief from constipation in a way that capitalizes on the body's natural processes. The drug, called A3309, targets bile acid recycling in the body. Bile acids, created in the liver and released into the digestive system, aid in breaking down fats and absorbing them into the body. Bile acids also are natural laxatives that promote bowel movements by softening stool and speeding up how fast stool moves through the colon. During digestion, most bile acids are absorbed back into the blood in the lower small intestines for recycling, letting very little bile acids to leak into the colon to help facilitate bowel movements. A3309 works by inhibiting bile acid absorption in the small intestines, allowing more bile acids to enter the colon to stimulate bowel movements. "The new medication is a novel approach which allows the delivery to the colon of normal substances produced by the patient's own liver to induce a laxative effect," says Michael Camilleri, MD, a gastroenterologist and Atherton and Winifred W. Bean Professor at Mayo Clinic and the study's lead author. In a Phase II, randomized, double-blind, placebo-controlled study, the Mayo Clinic research team tested the effectiveness of A3309 for two weeks in patients with constipation. The study yielded promising results. The drug sped up the movement of stool through the colon. Patients with constipation who took A3309, compared to those who received placebo, reported significantly less straining and softer stool during bowel movements, says researcher Banny Wong, MD. The next step in the drug's development will be Phase III studies that will involve more people and a longer treatment duration. Source: EurekAlert! 5/10/11

New Research Suggests Dramatic Shift in Understanding of Personalized Medicine

Researchers at Mount Sinai School of Medicine, in collaboration with researchers at Loyola University Chicago Stritch School of Medicine, have made a critical discovery that may lead scientists to abandon the use of broad conventional ethnic labels—African-American, Hispanic, and Caucasian—to estimate a patient's genetic risk for disease. This first-of-its kind study, conducted with diverse patients receiving care at a single urban academic medical center, marks an important step in the clinical application of personalized medicine. The data are published online in the peer-reviewed journal PLoS ONE. The Mount Sinai Biobank, a program of the Charles R. Bronfman Institute for Personalized Medicine, enrolls consented patients representing the diverse communities surrounding the Mount Sinai Medical Center, who confidentially provide DNA and plasma samples to aid in genomic and personalized medicine research. Researchers used state-of-the-art genomic technology to determine the genetic make-up, or genotype, of nearly 1,000 local Biobank participants who self-identified as European American, African-American, or Hispanic. They found that there was a continuum in ancestral genetic heritage at the individual level of African-American and Hispanic patients receiving care at Mount Sinai—meaning considerable fractions of their genome came from mixed European or African ancestry, respectively—and with it genetic variants that indicate risk for developing disease. "Our data indicate that historical population labels may not be helpful in predicting disease risk or guiding how a patient will respond to certain medications," said Erwin Bottinger, MD, director of the Bronfman Institute. "Rather, a spectrum of mixed ancestry is emerging in the largest U.S. minority groups. These findings further validate the importance of considering the unique genotype of the individual patient rather than grouping patients by self-reported ethnicity." On the basis of their findings, researchers at the Bronfman Institute are evaluating the clinical use potential of hundreds of genetic markers for major diseases, such as heart disease, kidney disease, liver disease, and diabetes, and various drug responses, in all Biobank samples. The goal is to identify those genetic markers that may be useful to predict disease risk in local people with mixed genetic backgrounds. The project will be completed with all 20,000 Biobank samples by the end of 2011. This study was supported by The Andrea and Charles Bronfman Philanthropies, Inc. Source: EurekAlert! 5/4/11

New Trial Aims to Help Pregnant Women Stop Smoking

A clinical trial to test the safety and efficacy of a medication that could help pregnant women stop smoking has begun enrollment at the University of Texas Health Science Center at Houston (UTHealth). The study targets pregnant women in their second and third trimesters when smoking can be quite harmful to the fetus, said Angela Stotts, PhD, associate professor in the Department of Family and Community Medicine at the UTHealth Medical School. "Pregnant women receive counseling or self-help materials, but they may need something more powerful to stop smoking. Nicotine is one of the hardest substances to quit due to its action in the brain," Stotts said. The medication, bupropion, is known under the brand name Zyban for smoking cessation and as the antidepressant Wellbutrin in a stronger formula. "In nonpregnant smokers, bupropion has been shown to almost double the success rate of smoking cessation, and [it] is a relatively safe medication," Stotts said. "Some obstetricians already prescribe it during pregnancy for depression." The randomized, placebo-controlled trial, funded by the Gilstrap Center and the UTHealth Center for Clinical and Translational Sciences, will recruit 50 women over a two-year period from the UT Physicians Obstetrics and Gynecology Clinic and the UT Physicians Women's Health Center. Source: EurekAlert! 5/4/11

Glass Fibers Appear to Speed Healing in Wound Trial

Imagine a battlefield medic or emergency medical technician providing first aid with a special wad of cottony glass fibers that simultaneously slows bleeding, fights bacteria (and other sources of infection), stimulates the body's natural healing mechanisms, resists scarring, and—because it is quickly absorbed by surrounding tissue—may never have to be removed in follow-up care. Or, imagine diabetics with hard-to-heal wounds finding a source of relief from the battle against infections and limb amputation. Those scenarios are the hope of the developers of a revolutionary borate glass nanofiber material, which appears have sped and helped the final of healing long-term wounds in eight out of 12 venous stasis wound sufferers in a recent clinical trial held at a medical center in Rolla, Mo. Details about the trials and the glass fiber material were published in the May issue of the American Ceramic Society's Bulletin magazine. The story reports on the discovery of the fibers and on an empirical study that began late in the fall of 2010 supervised by the internal review board of the Phelps County Regional Medical Center. According to Peggy Taylor, the registered nurse who administered the treatments, all of the volunteers who took part in the trial are enthusiastic about the use of the glass fiber product, which she says "looks like cotton candy." All of the participants had diabetes, and several of them had wounds that had been unhealed for more than a year. One patient had the same wound for three years. After using the glass fiber product to provide a healing "scaffold" for skin reconstruction for a few months, Taylor and other specialists were able to repair the skin in eight of the patients. "Remarkably," she says, "the other four have made a lot of progress and all of their wounds should be healed soon, too." The goal of the trial was to provide an initial evaluation of the effects of the novel fibrous glass material produced by the Mo-Sci Corp., a Rolla company already known for creating glass-based materials for medical applications. The next step is expanded human trials, which will be conducted in partnership with the Center for Wound Healing and Tissue Regeneration at the University of Illinois at Chicago this summer. Source: EurekAlert! 5/3/11

Gene Therapy Shows Promise Against Age-Related Macular Degeneration

A gene therapy approach using a protein called CD59, or protectin, shows promise in slowing the signs of age-related macular degeneration (AMD), according to a new in vivo study by researchers at Tufts University School of Medicine. Led by senior author Rajendra Kumar-Singh, PhD, the researchers demonstrated for the first time that CD59 delivered by a gene therapy approach significantly reduced the uncontrolled blood vessel growth and cell death typical of AMD, the most common cause of blindness in the elderly. The study was published on April 28 in PLoS ONE. Activation of the complement system, a part of the immune system, is responsible for slowly killing cells in the back of the eye, leading to AMD. Activation of this system leads to the generation of pores or holes known as 'membrane attack complex' or MAC in cell membranes. CD59 is known to block the formation of MAC. "CD59 is unstable and hence previous studies using CD59 have had limited success. The gene therapy approach that we developed continuously produces CD59 in the eye and overcomes these barriers, giving us renewed hope that it can be used to fight the progression of AMD and potentially other diseases," said Kumar-Singh. Kumar-Singh and colleagues delivered CD59 to the eye using a deactivated virus similar to one previously shown to be safe in humans. Using an established mouse model of age-related macular degeneration, they found that eyes treated with CD59 had 62 percent less uncontrolled blood vessel growth and 52 percent less MAC than controls. The current standard treatment for some forms of AMD requires an injection directly into the eye approximately every four weeks. According to Kumar-Singh, gene therapy approaches to treat AMD are especially attractive because they will allow patients to be treated less frequently, reducing patient discomfort and lowering chances of infection and other side effects associated with frequent injections into the eye. "Prior to initiating human clinical trials, we will need to perform extensive preclinical toxicology studies. In order to advance this study to Phase I clinical trials, we have formed a partnership with Hemera Biosciences Inc. to raise private venture capital," said Kumar-Singh. Source: EurekAlert! 4/29/11

Tierney Named 2011 Clinical and Translational Research Distinguished Investigator

William M. Tierney, MD, president and CEO of the Regenstrief Institute and associate dean for clinical effectiveness research at the Indiana University School of Medicine, received a 2011 Distinguished Investigator Award presented at the Clinical and Translational Research and Education Meeting on April 28 in Washington, D.C. Tierney received the National Award for Career Achievement and Contribution to Clinical and Translational Science. He is being honored for his "contributions to the field from clinical use into public benefit and policy." The award is presented to a senior investigator who has had "national impact by virtue of contributions to clinical and translational science." Source: EurekAlert! 4/29/11

Clinical Trial Recommends New Antibiotic for Treating Typhoid in Low Income Countries

A large clinical trial comparing treatments for typhoid has recommended the use of gatifloxacin, a new generation and affordable antibiotic. The results of the trial in Kathmandu, Nepal, funded by the Wellcome Trust and the Li Ka Shing Foundation, are published in Lancet Infectious Diseases. The standard treatment for enteric fever since the 1950s was the drug chloramphenicol. The spread of multidrug resistant forms of the Salmonella typhi and Salmonella paratyphi bacteria, which cause the disease, saw a move toward a new generation of antibiotics known as fluoroquinolones, but now there is evidence that the bacteria are becoming resistant to even these drugs. Gatifloxacin--a new type of fluoroquinolone--was released in North America in 1999 under the brand name Tequin by the pharmaceutical company Bristol-Myers Squibb, but was subsequently withdrawn following the publication in 2006 of a retrospective Canadian study in the New England Journal of Medicine claiming that the drug can cause serious side effects including very high and low blood sugars. In the new study, researchers from the Wellcome Trust Major Overseas Programme at the Oxford University Clinical Research Unit, Ho Chi Minh City, Vietnam, together with researchers at the Oxford University Clinical Research Unit–Patan Academy of Health Sciences, Kathmandu, Nepal, conducted an open-label, randomized, controlled trial to compare gatifloxacin to chloramphenicol. Their study, which enrolled 844 children and adults, is the largest randomized controlled trial for enteric fever carried out to date. The researchers found both drugs to be equally effective. They found no difference between the two drugs in terms of treatment failure and the time to clear the fever. However, the side effects, including anorexia, nausea, diarrhea, and dizziness, were significantly worse in patients being treated with chloramphenicol. Because of concerns of the side effects of gatifloxacin in an elderly North American population, the researchers closely monitored the patients' blood sugar levels. Although they found a higher number of patients with elevated blood sugar levels during the first week of treatment, these levels had returned to normal once the treatment course had ended and no change in treatment was required. The researchers have submitted evidence to the World Health Organization (WHO) arguing that gatifloxacin should be retained in young populations not at risk of diabetes. It is understood that the WHO will consider this evidence later this year. The drug is also in Phase III trials for treatment of tuberculosis. Source: EurekAlert! 4/28/11

Trial Will Evaluate Whether Gene Expression Test Can Drive Chemotherapy Choice

Cancer researchers at hundreds of U.S. sites are about to launch a SWOG-led clinical trial that could keep thousands of breast cancer patients from getting chemotherapy that is unlikely to do them any good. Each year in the United States, more than 60,000 women are diagnosed with hormone receptor-positive breast cancer that has spread to their lymph nodes. Almost all get chemotherapy in addition to endocrine therapy, but analyses of earlier studies have suggested that the two-thirds of these women with the lowest Oncotype DX® recurrence scores may see little or no benefit from that chemotherapy. The RxPONDER (Rx for Positive Node, Endocrine Responsive Breast Cancer) trial will reveal whether chemotherapy benefits patients with node positive breast cancer who have low to intermediate Oncotype DX recurrence scores. The trial also seeks to determine whether there is an optimal recurrence score cutpoint for these patients, above which chemotherapy should be recommended. "If the RxPONDER trial confirms findings from earlier studies," says study coordinator Ana M. Gonzalez-Angulo, MD, of the MD Anderson Cancer Center, "it will mean that we know more precisely how to use a genomic-based test to spare thousands of women whose breast cancer has spread to as many as three lymph nodes the grueling side effects and staggering costs of chemotherapy they don't need." The Oncotype DX assay costs about $4,000 per patient, yet the chemotherapy treatment now routinely given to these patients costs more than $20,000 per course, meaning that the results of the RxPONDER study could also save hundreds of millions of dollars in healthcare costs each year. Researchers plan to enroll 4,000 women with recurrence scores of 25 or less who have early stage, hormone receptor-positive, HER2-negative breast cancer that has been found to involve one to three lymph nodes. They expect to screen about 9,000 breast cancer patients in order to register these 4,000, who will be randomized to receive either chemotherapy with endocrine therapy or endocrine therapy alone. The Phase III trial is being led by SWOG, one of the largest of the National Cancer Institute supported clinical trial cooperative groups, but is expected to be opened by the other major cooperative groups as well, giving patients almost anywhere in the United States access to the trial. Patients can see a list of sites where the trial is currently open by going to swog.org/RxPONDER or can ask their doctor about the trial. The Oncotype DX test measures the expression, or activity, level of 21 specific genes within a tumor sample and, based on that pattern, assigns a recurrence score of anywhere from 0 to 100. Earlier trials have shown that the higher the recurrence score, the more likely the patient's cancer will recur. The assay is performed by Genomic Health, Inc., based in Redwood City, Calif. RxPONDER researchers plan to evaluate other tests as well, including the PAM50 test, which measures the expression level of a set of 50 genes to determine a patient's "risk of recurrence" score. Source: EurkeAlert! 4/26/11

First Trial of Gene Therapy for Pain Reported

In the first clinical trial of gene therapy for treatment of intractable pain, researchers from the University of Michigan Department of Neurology observed that the treatment appears to provide substantial pain relief. In a study published online in the Annals of Neurology, the researchers showed that the novel agent NP2 is safe and well tolerated. In addition, measures of pain in the treated patients suggested that NP2 may provide a substantial analgesic effect. NP2 is a gene transfer vector that expresses the naturally occurring opioid peptide enkephalin. In preclinical work in animals, David Fink, MD, the Robert Brear Professor and chair of the Department of Neurology, and his coworkers had demonstrated that injection of NP2 into the skin reduces pain in models of pain caused by nerve damage, inflammation, or cancer. In the clinical trial, 10 patients with unrelenting pain caused by cancer were injected with the gene transfer agent in the area of skin related to the location of pain. Patients who received the low dose of vector showed little reduction in pain; patients receiving the higher doses showed a greater than 80 percent reduction in pain over the course of four weeks following treatment. There was no placebo control in the Phase I study, but the apparent dose-dependent pain relief was encouraging to the researchers. A Phase II trial to compare NP2 to a placebo control has already been initiated under sponsorship from Diamyd. Source: EurekAlert! 4/11/11

Researchers Show Drug is Effective in Prevention of Type 2 Diabetes

Playing a leadership role in a nationwide clinical study, Keck School of Medicine of University of Southern California (USC) researchers have found that pioglitazone, a drug that reverses some of the bad effects of obesity, reduced the risk of type 2 diabetes by 72 percent in pre-diabetic patients. The researchers also found that the drug slowed the development of an early marker of cardiovascular disease in the carotid arteries. Findings of the study were reported in the March 24 issue of the New England Journal of Medicine. Thomas A. Buchanan, professor of medicine, obstetrics and gynecology, and physiology and biophysics at the Keck School, was a co-investigator of the study, along with two other of the school’s researchers: Howard N. Hodis, the Harry Bauer & Dorothy Bauer Rawlings Professor of Cardiology, and Wendy J. Mack, associate professor of preventive medicine. The lead investigator was Ralph A. DeFronzo of the University of Texas at San Antonio. In the study of 602 patients, 2.1 percent of participants who received pioglitazone developed type 2 diabetes, compared to 7.6 percent of participants given a placebo. In addition, 48 percent of drug-treated participants saw their glucose tolerance levels return to normal, compared to 28 percent of those who received a placebo. In examining pioglitazone’s effect on the development of cardiovascular disease, the researchers found that it slowed the development of atherosclerosis, or thickening, of the carotid artery. The carotid thickness for those in the study who received the drug was 31 percent less than the placebo group. The glucose and cardiovascular results of the study confirmed the findings of research on thiazolidinediones by Buchanan and his USC team between 1995 and 2000. They conducted the first U.S. studies of troglitazone, finding that it delayed or prevented the development of diabetes by 55 percent. Sold under the brand name of Rezulin, troglitazone was taken off the market because it was found to cause liver damage in rare cases. Two other thiazolidinediones then were developed--rosiglitazone and pioglitazone. Also contributing to the study were researchers at the University of California, San Diego, the University of Tennessee, Georgetown University, the Pennington Biomedical Research Center in Baton Rouge, the Phoenix VA Health Care System, and the State University of New York Health Science Center. Source: USC News 4/5/11

Drug Used to Treat Alcoholism May Help in Fragile X/Autism Cases

In small, early clinical trials, adults and children with autism and fragile X syndrome have shown improved communication and social behavior when treated with acamprosate, according to Craig Erickson, MD, assistant professor of psychiatry at the Indiana University School of Medicine and chief of the Riley Hospital for Children Christian Sarkine Autism Treatment Center at Indiana University Health. Acamprosate, which affects chemicals in the brain by blocking certain receptors associated with mental health, has approval from the Food and Drug Administration for the treatment of alcoholism in adults. Erickson is the inventor on a pending utility patent for the use of acamprosate as a therapeutic agent for fragile X syndrome, the most common inherited form of intellectual disability and the most frequent single gene cause of autism. "We have been treating small numbers of both adults and children," said Erickson. "We have observed improvements in eye contact, social interaction and speech. This is very early work, but it appears promising." Source: EurekAlert! 4/4/11

University and Dietary Supplement Company Start Clinical Trial in Autism

Nova Southeastern University and the dietary supplement company Immunotec announced in late March that they will conduct a new research study to determine the effects of a supplement to improve behaviors in children with autism spectrum disorder. The two-year study will investigate the effects of a cysteine-rich whey protein isolate in children diagnosed with autism. The primary function of this research is to establish whether taking a 90-day diet of cysteine-rich whey proteins (which comes in a powder form), will have a positive effect on autistic behaviors. Improvements in affected behaviors may result in an enhanced quality of life for both children and their families. During the clinical study, there will be an analysis of the children’s exhibited behaviors. Source: Newswise 3/30/11

New Cancer Drug Heads to Clinical Trials

Researchers at the University of Michigan Comprehensive Cancer Center have developed a new drug called AT-406 with potential to treat multiple types of cancer. A recent study in the Journal of Medicinal Chemistry showed that AT-406 effectively targets proteins that block normal cell death from occurring. Blocking these proteins caused tumor cells to die, while not harming normal cells. The researchers believe the drug could potentially be used alone or in combination with other treatments. In animal models, the drug shrank tumors but caused few side effects. The drug is designed to be taken by mouth, which researchers say will make it easier than traditional intravenous chemotherapies to administer. Patent applications covering the drug are exclusively licensed to Ascenta Therapeutics, a privately held, clinical stage biopharmaceutical company. After extensive testing, Ascenta began the first clinical trial in 2010 testing AT-406 for cancer treatment in all solid tumors. Ascenta has also recently opened a second trial of AT-406 in high-risk acute myeloid leukemia, and several more trials are planned. Source: Newswise 3/29/11

College Seeks 25,000 Volunteers for Breast Cancer Research

The College of Notre Dame of Maryland is supporting the Love/Avon Army of Women, a nationwide initiative to recruit 1 million volunteers to enroll in research to help uncover the causes of breast cancer. A program of the Dr. Susan Love Research Foundation funded by a grant from the Avon Foundation, the Army of Women seeks women and men across the U.S., of all ages (over 18) and ethnicities, with or without breast cancer, to be counted as “one in a million” by signing up at armyofwomen.org. To raise awareness for the campaign, College of Notre Dame is hosting a recruitment rally on May 13 featuring Dr. Susan Love, founder of the Army of Women, president of the Dr. Susan Love Research Foundation, and a leader in the breast cancer field for more than 30 years. Notre Dame is working toward an ambitious goal—adding 25,000 more names to the Army of Women—and hopes to bring women and men from the entire region to sign up and be counted. After signing up at armyofwomen.org, members will receive regular e-mails notifying them of research studies that they can participate in if willing and if they are eligible. Participation can involve anything from providing a blood sample to something as simple as filling out an online questionnaire. Source: Newswise 3/28/11

Cancer Drug Shows Promise for Treating Scleroderma

A drug approved to treat certain types of cancer has shown promising results in the treatment of patients with scleroderma, according to results from an open-label Phase II trial. While the drug's efficacy must be demonstrated in a Phase III trial, the gold standard for testing a drug, researchers are optimistic that Gleevec™ (imatinib) could potentially be a weapon against the chronic connective tissue disease for which a treatment has remained elusive. "This trial showed Gleevec has acceptable safety and tolerability, and there are hints of efficacy or suggestions the drug may work," said Robert Spiera, MD, an associate attending rheumatologist at Hospital for Special Surgery who led the study. "This study strongly suggests that a randomized placebo-controlled trial is warranted." The study appears online in advance of print in Annals of the Rheumatic Diseases. To date, there has never been a drug that has been shown to be effective for scleroderma. For the study, investigators at Hospital for Special Surgery enrolled 30 patients with diffuse scleroderma, a widespread severe form of the disease, and gave them 400 mg of Gleevec per day. Patients were evaluated monthly for 12 months during treatment and were seen for follow-up three months after discontinuing the drug. Source: EurekAlert! 3/28/11

Predicting Serious Drug Side Effects Before They Occur

All medications have side effects, from common aspirin to herbal remedies and from standard anticancer drugs to experimental immunosuppressants. Although predicting important side effects, or serious adverse drug reactions (ADRs), is almost impossible with current understanding, a neural network technology trained with past data could give drug companies and healthcare workers a new tool to spot the potential for ADRs with any given medication. Writing in the International Journal of Medical Engineering and Informatics, a team from the University of Medicine and Dentistry of New Jersey has developed a new model that tests show is 99.87 percent accurate in predicting ADRs among 10,000 observations and 100 percent for nonserious ADRs. The team's artificial neural network (ANN) is a mathematical model of the biologic neural network embedded in computer software. It is trained by feeding in structural and physical data associated with known pharmaceutical products and any ADRs. A feedback loop discards those connections where a wrong prediction of a known outcome is made and, as data are added, the ANN builds up a network of correct "predictions." After sufficient training, the ANN can then be tested on another set of pharmaceuticals and outcomes checked against known ADRs. If confidence is sufficiently high, the ANN can be used to predict ADRs for new drugs. Source: EurekAlert! 3/28/11

Interventional Radiology Using Y-90 Liver Cancer-Busting Treatment Deemed Safe

Interventional radiologists have been the leaders in the use of intra-arterial yttrium-90 (Y-90) radioembolization, since its introduction in 2000, to treat liver cancer. Now, new results from a large multi-institutional study show that treating liver tumors with higher doses of Y-90 than previously tried is safe, provides results when chemotherapies have failed, preserves the patient's quality of life, and can be done on an outpatient basis. This study, presented by researchers at the Society of Interventional Radiology's 36th Annual Scientific Meeting in Chicago, Ill., further validates previous findings on the safety and efficacy of liver cancer treatments using Y-90. "We knew that this unique interventional radiology treatment, done on an outpatient basis, which combines the radioactive isotope Y-90 into microspheres (small beads about the width of five red blood cells) that deliver radiation directly to a tumor, was one of the best ways to give patients a treatment that doesn't harm healthy cells," explained Riad Salem, MD, MBA, FSIR, professor of radiology, medicine, and surgery and director of interventional oncology at Northwestern University in Chicago. "Now we know that patients can actually tolerate much higher doses of radiation than previously thought, which provides results in patients progressing on standard chemotherapy. While patients aren't cured, their lives are being extended with less down time and their quality of life is improving." The four-year prospective study looked at 151 patients with liver metastases from colorectal, neuroendocrine, and other cancers. Source: PRNewswire 3/28/11

Institute Launches Lung Cancer Research Initiative

The Addario Lung Cancer Medical Institute (ALCMI) in late March announced the enrollment of the initial subjects into its inaugural clinical trial, known as CASTLE, targeting 250 subjects over two years among academic and community medical centers in the United States. CASTLE stands for Collaborative Advanced Stage Tissue Lung Cancer study. ALCMI is an innovative, patient-founded, nonprofit research consortium that facilitates and drives comprehensively contracted collaborations among researchers in academic and community medical centers with the mission of significantly increasing the currently poor survival rates of patients diagnosed with lung cancer. Recognizing the imperative for a paradigm shift in the battle against lung cancer, the Bonnie J. Addario Lung Cancer Foundation and leading lung cancer scientists in the U.S. and Europe cofounded ALCMI in 2008 as a standalone, results-focused research consortium. ALCMI pools knowledge, biospecimens, and data to facilitate and accelerate the development and delivery of much more effective diagnostics and treatments tailored to the individual patient, leading as soon as possible to personalized therapies and vastly improved patient survival rates. Source: PRNewswire 3/28/11

Experimental Radioprotective Drug Safe for Lung Cancer Patients

Patients with advanced non-small cell lung cancer can safely take an experimental oral drug intended to protect healthy tissue from the effects of radiation, according to a study led by researchers at the University of Pittsburgh Cancer Institute (UPCI) in Pennsylvania and published in the March issue of Human Gene Therapy. The findings support further clinical testing of the agent, called manganese superoxide dismutase (MnSOD) plasmid liposome, to determine if giving it alongside chemotherapy and radiation will prevent damage to normal cells that is the typical cause of side effects in cancer treatment, said senior investigator Joel S. Greenberger, MD, professor and chair of the Department of Radiation Oncology in Pitt's School of Medicine and codirector of the lung and esophageal cancer program at UPCI. For the safety study, 10 patients with inoperable stage III non-small cell lung cancer took oral doses of MnSOD plasmid liposome twice weekly for a total of 14 doses during seven weeks of conventional chemotherapy and radiation treatment. The agent, which boosts levels of an antioxidant the body makes naturally, is made of fat droplets containing the gene that produces MnSOD. When swallowed, it is absorbed by cells in the esophagus, which is a common site for severe side effects during radiation treatment for lung cancer. "The results of this initial trial indicate that MnSOD plasmid liposome can be safely administered," Greenberger said. "It did not linger in normal cells after treatment, nor did it protect cancer cells from radiation treatment. The next study, which is under way at UPCI, is to determine whether it protects normal tissue, particularly the esophagus, from radiation exposure." He and his team plan to investigate the use of MnSOD plasmid liposome for other cancers, such as protecting the rectum from radiotherapy for prostate cancer and protecting the bladder during ovarian or endometrial cancer treatment. The research team includes colead author Ahmad A. Tarhini, MD, James D. Luketich, MD, and others from UPCI; colead author Chandra P. Belani, MD, of Penn State Hershey Cancer Institute; and others from Emory University, University of Washington School of Medicine, and PharmaReg Consultants, of San Leandro, Calif. The study was funded by the UPCI Specialized Program of Research Excellence in lung cancer. Source: EurekAlert! 3/22/11

Trial Asks Whether Platelet-Rich Plasma Can Ease Osteoarthritis Pain

For years, doctors have used platelet-rich plasma (PRP) to promote healing after surgery. Now, Rush University Medical Center in Chicago, Ill., is studying whether PRP can help relieve knee pain in patients with mild to moderate osteoarthritis. PRP contains growth factors that promote cell proliferation and is prepared from the patient's own blood tissue. It has received popular attention recently because of its use in treating sports injuries in professional athletes, but the jury is still out on whether it is effective. "There have been few controlled clinical trials, and results are inconsistent, but data so far suggest that it could be a promising treatment for healing in a variety of tissues," said Dr. Brian Cole, orthopedic surgeon and head of the cartilage restoration center at Rush. "The therapy will not be a cure for osteoarthritis, but it could help put off the day when a patient will need to get a knee implant." In the double-blind, randomized, controlled study, 100 patients will receive either hyaluronic acid or PRP. The PRP is prepared from 10 millimeters of the patient's own blood. The blood is spun in a centrifuge to separate the platelets from the red and white blood cells. The platelets are then injected into the knee joint using ultrasound imaging to guide placement. Patients will receive three injections over three weeks and will be monitored for two years. In periodic clinical exams, the physician will assess pain and knee function. In addition, a teaspoon-size sample will be taken of the synovial fluid around the knee joint to test for molecular changes that may indicate a shift in the balance of anabolic factors that increase the buildup of tissue and catabolic factors that break it down. An imbalance in these factors has been implicated in the deterioration of cartilage that leads to osteoarthritis. Source: EurekAlert! 3/22/11

Can a Cholesterol Drug Prevent Colon Cancer?

Thomas Jefferson University in Philadelphia, Pa., has started recruiting patients for a new National Cancer Institute-sponsored clinical trial to test whether the cholesterol-reducing drug rosuvastatin is effective in the prevention of recurrent colon cancer. Previous laboratory research and population studies have shown that patients taking statins, the class of drugs that lowers cholesterol, had fewer colon polyps, which can lead to cancer if left untreated. However, those findings come largely from retrospective, observational studies originally designed to investigate lipid-lowering or cardiovascular endpoints in the short term rather than tumor endpoints. "The jury is still out, and we need to get definitive answers," said Bruce Boman, MD, PhD, professor of medical oncology at Thomas Jefferson University and principal investigator for the national clinical trial. "This prospective design comparing a statin against a placebo is what is needed to address the question: Are statins effective chemoprevention agents or not?" This five-year, nationwide study will be the first randomized, prospective, placebo-controlled, double-blind study to evaluate the drug's role in preventing colon cancer and will involve 1,740 patients in total. Conducted by a network of cancer research professionals from the National Surgical Adjuvant Breast and Bowel Project at 400 medical centers across North America, including Jefferson, the study involves patients who have recently been diagnosed with early stage colon cancer, and who were not already taking statins for high cholesterol. Those recruited have been surgically treated for stage I and II colon cancers previously. Patients will be randomly assigned to one of two groups. Each group will take one pill a day for five years. One group will receive rosuvastatin, while the other group will receive a placebo. The principal investigator for the trial at Jefferson is Scott Goldstein, M.D, director of the Division of Colon and Rectal Surgery at Thomas Jefferson University. Source: EurekAlert! 3/22/11

Hearing the Patient’s Voice in Clinical Trials

An upcoming presentation in the Current Challenges Facing Health Professions series at Regis College in Weston, Mass., invites attendees to focus on "Hearing the Patient's Voice in Clinical Trials." The event, featuring clinical research and measurement authority Barbara Marino of PHT Corporation as speaker, will be held at 6 p.m. Thursday, April 7, in the Upper Student Union, Alumnae Hall, at the college. Marino asks you to imagine that you are a subject in a clinical trial of a new compound. The drug is intended to treat a disease that has very personal symptoms. You have been given a paper diary to record all those very personal symptoms every day. You are embarrassed to learn that your teenaged son has found your diary. What’s more, the nurse at your doctor’s office goes through the completed diary page by page with you when you come in for a visit. What would you do? Would you skip the very personal questions, or maybe wait until the last minute before your doctor’s visit to complete the diary? If you complete lots of diaries in one sitting, would you remember your symptoms from last week, or just have to guess? These are some of the issues that have plagued researchers who want to capture accurately patient experience in a clinical trial. Other issues to be explored include whether electronic capture of patient-reported clinical information improves data quality; how it affects clinical trial design and costs; how the products that allow it can/will be regulated; and whether the information can/will be integrated with healthcare informatics. This event is appropriate for the general public, as well as for students and professionals in the health field. For more information, contact Marjorie Arons-Barron at aronsbarron@barronaw.com.

Institute for Pediatric Innovation and Children's Medical Center Dallas Team Up

Children's Medical Center Dallas is the latest hospital to join the Institute for Pediatric Innovation's Consortium of Pediatric Hospitals. These progressive children's hospitals work closely with the institute to identify new medical devices and drug formulations that specifically address the health needs of babies and children. The institute has formalized a model based on a structured assessment of needs and feasibility in different pediatric settings and the facilitation of partnerships with stakeholders (including nonprofit organizations, academic institutions, industry, government, foundations, and advocacy groups) for product development. With this partnership, Children's Medical Center Dallas joins seven additional children's hospitals, including Phoenix Children's Hospital, Children's Hospital of Wisconsin, the Children's Hospital of Denver, Children's Mercy Hospital and Clinics, Lucile Packard Children's Hospital, Rainbow Babies & Children's Hospital, and Children's National Medical Center. Source: Marketwire 3/7/11

Cancer Institute of New Jersey Welcomes System Partner

Continuing to build on their collective strength in the areas of oncology research, teaching, and clinical care, the Cancer Institute of New Jersey (CINJ) and Meridian Health have announced a new level of collaboration benefitting patients across the state through increased access to clinical trials. Several of Meridian Health's hospitals have longstanding affiliations with CINJ, a Center of Excellence of UMDNJ-Robert Wood Johnson Medical School and the state’s only National Cancer Institute-designated Comprehensive Cancer Center. The high quality programs at Meridian, whose specialists treat more than 4,000 newly diagnosed cancer patients each year, will now have additional support from CINJ’s clinical and academic researchers. Meridian Health includes Jersey Shore University Medical Center, Ocean Medical Center, Riverview Medical Center, Southern Ocean Medical Center, and Bayshore Community Hospital. Each of Meridian’s hospitals has a Commission on Cancer-accredited cancer program. Source: Newswise 3/3/11

A North American First Could Help Treat Thousands of Canadians

The interventional cardiology team at the Montreal Heart Institute (MHI) recently began patient enrollment for a new device, the Neovasc Reducer™, designed to treat patients suffering from refractory angina. The treatment method is a first in North America and is being conducted as part of an international study, the COSIRA trial. This innovative treatment is promising for thousands of Canadians disabled by refractory angina and who lack alternatives for relieving their symptoms and improving their quality of life. Developed in Canada by Neovasc Inc., the Reducer is implanted in the coronary sinus vein using minimally invasive techniques. Placement of the device is performed using a procedure that is similar to implanting a coronary stent and takes approximately 20 minutes. The Reducer is designed to establish a permanent and controlled narrowing of the coronary sinus, which is a new technique to provide relief of refractory angina symptoms by altering blood flow in the coronary sinus and thereby increasing perfusion of oxygenated blood to certain areas of the heart muscle that receive an inadequate supply of oxygen. Patients are discharged within 24 hours after the intervention. This landmark procedure was carried out by a MHI multidisciplinary team composed of Drs. Marc Jolicoeur, Serge Doucet, and Jean-François Tanguay, interventional cardiologists, as well as Dr. Raymond Cartier, heart surgeon. The COSIRA (Coronary Sinus Reducer for Treatment of Refractory Angina) trial is a multicenter, sham-controlled, randomized, double-blinded study of the Reducer that is expected to enroll up to 124 patients. The primary endpoint is efficacy in reducing angina symptoms after six months. In addition to the Montreal Heart Institute, the COSIRA trial is enrolling patients at the University of Ottawa Heart Institute; at the Antwerp Cardiovascular Institute and Ziekenhuis Oost-Linburg Hospital in Belgium; and at Ultrecht Medical Center in the Netherlands. Additional sites are expected to join the trial in the coming months. Results from the initial first-in-human trial for the device showed that three years after implantation of the Reducer, it remained safe and the majority of the 15 patients treated continued to show measurable improvement in angina symptoms. MHI is affiliated with the Université de Montréal. Neovasc Inc. is a specialty vascular device company that develops, manufactures, and markets medical devices for the rapidly growing vascular and surgical marketplace. Source: EurekAlert! 2/24/11

Deep Brain Stimulation Helps Severe OCD; Pioneer Advises Caution and Compassion

When obsessive-compulsive disorder (OCD) is of crippling severity and drugs and behavior therapy can’t help, there has been for just over a year a thread—or rather a wire—of hope. By inserting a thin electrode deep into the brain, doctors can precisely deliver an electrical current to a cord of the brain’s wiring and soften the severity of the symptoms. Deep brain stimulation (DBS) therapy for OCD won Food and Drug Administration approval in 2009 for extreme cases under its humanitarian device exemption. In February, at the annual meeting of the American Association for the Advancement of Science, Benjamin Greenberg, MD, a psychiatrist at Brown University and at Butler Hospital, discussed the longest-term results so far of the technique he’s helped pioneer during the last decade. Encouraged as he is with the progress, he addressed how to best apply it, and how basic and clinical researchers are working to understand the changes in brain networks and behaviors that occur when patients improve. “These techniques are promising but must be used with an abundance of caution,” said Greenberg, associate professor of psychiatry and human behavior at the Warren Alpert Medical School of Brown University and chief of outpatient services at Butler Hospital. “This is reserved for the small proportion of people who are severely disabled and have not benefitted anywhere near adequately from very aggressive use of conventional treatments.” Just more than 50 patients have had DBS for OCD in the United States since the work began in 2000. Some researchers have questioned whether granting the humanitarian device exemption for DBS for OCD was appropriate because the potential patient population was too large, but Greenberg said he disagrees. He noted that only about 15 patients nationally have received DBS in each of the last two years despite extensive outreach to clinicians for his current National Institute of Mental Health-funded DBS clinical trial, and the separate humanitarian device exemption approval. “In the real world, the group of patients meeting appropriate selection criteria is truly small,” he said. Greenberg also noted that OCD patients selected for this study have an excellent understanding of the risks, burdens, and potential benefits of DBS. “They clearly know exactly what they’re signing up for,” he said. It was not unusual for patients to have read his scientific papers on DBS, he added, and all have scored perfectly on a test of comprehension of study details after informed consent. Source: Brown University 2/16/11

Trial Will Test Whether Surgery is Best Option for Type 2 Diabetes

A new clinical trial at NewYork-Presbyterian Hospital/Weill Cornell Medical Center is among the first to test surgery specifically for type 2 diabetes. The aim of the study is to understand whether surgery can control diabetes as well as, or even better than, the best medical treatment available today. This is the first study of its kind open to patients who are overweight or mildly obese. Recent recommendations from the American Diabetes Association suggest that randomized clinical trials for the study of surgery in patients with Body Mass Index (BMI) below 35 are priority for diabetes research. The new study is enrolling 50 patients with type 2 diabetes who will be randomized to receive surgery--specifically, Roux-en-Y gastric bypass--or traditional medical therapy and intensive lifestyle modification. All patients will be counseled in lifestyle modification techniques like diet and exercise. Patients in the medical arm will also be offered the chance to switch study arms and have surgery free of charge after the study is complete, or earlier should their diabetes remain poorly controlled after medical and lifestyle therapy. Because insurers use BMI-based criteria, bariatric surgery is currently not covered for patients with a BMI less than 35, regardless of the severity of their disease. Consequently, the study is supported by a research grant from Covidien covering the cost of surgery for patients enrolled in the study. The research team will measure gut hormone responses to meal stimulation when an equivalent amount of weight loss has been achieved in both surgically and conventionally treated patients. This design may help uncover endocrine effects specific to gastric bypass surgery beyond those associated with nonsurgical weight loss, and could be a template for larger, international studies. Source: EurekAlert! 2/10/11

Public Sector Research Responsible for Many New Drug Discoveries

Researchers from Boston University's Schools of Medicine, Management, and Law, along with collaborators from the National Institutes of Health, believe that public sector research has had a more immediate effect on improving public health than was previously realized. The findings, which appear in the February 10 issue of the New England Journal of Medicine, have economic and policy implications. Historically, public sector research institutions have not participated in any major way in the downstream, applied phase of drug discovery, in which the actual products are discovered and patented. However, in the mid-1970s, the newly emerging tools of biotechnology allowed these institutions to create and patent biologic drug candidates and discover and patent small molecule drugs. At that time, all products created in academic institutions were owned by the government, which granted only nonexclusive licenses. In 1980, Congress passed two pieces of legislation that transformed the ownership, management, and transfer of intellectual property that is created by public sector research institutions. Under the new approach, inventions that arose from these institutions, in addition to being freely published in the scientific literature, could also be converted into intellectual property and transferred through license agreements to the private sector for commercialization and public use. In order to quantitate the contribution of public sector research to the applied research phase of drug discovery, the researchers identified new drugs and vaccines approved by the Food and Drug Administration (FDA) and classified them according to their therapeutic category and potential therapeutic effect. The researchers found that during the past 30 years, 153 new FDA-approved drugs, vaccines, or new indications for existing drugs were discovered through research carried out in public sector research institutions, including 93 small-molecule drugs, 36 biologic agents, 15 vaccines, eight in vivo diagnostic materials, and one over-the-counter drug. Source: EurekAlert! 2/9/11

Personalized Medicine Institute to Develop Targeted and Individualized Treatments

Indiana University has announced a major commitment to research in one of healthcare's most promising fields with the creation of the Indiana Institute for Personalized Medicine. The institute's members will be drawn from the university's medical, informatics, and nursing schools, with $11.25 million in funding provided by the School of Medicine, the school's Department of Medicine, Indiana University-Purdue University Indianapolis, the Indiana Physician Scientist Initiative, and the Indiana University Melvin and Bren Simon Cancer. The Indiana Physician Scientist Initiative is funded by a $60 million grant from the Lilly Endowment. "Much of the future of healthcare is in personalized medicine, meaning more precise targeting of the right medication to the right patient at the right time," said David Flockhart, MD, PhD, who has been named director of the institute. University scientists have been working on related research for at least a decade, but creating an institute "allows you to really jump start research and raise the level participation of an institution in both the laboratory and in the clinic, in a broad range of research interests," Flockhart said. The institute's mission is to conduct research, train new specialists in personalized medicine and promote the translation of scientific discoveries into new more precise therapeutics for patient care. To help move scientific discoveries to patient bedsides, the institute will have a panel of university scientists who will advise researchers on steps to take to move their research beyond the laboratory stages. Source: EurekAlert! 2/9/11

Technology in Trials Aims to Spot Cardiac Disease, Cancer, Drug Abuse

Heart disease is a silent killer, but new microchip technology from Rice University is expected to advance the art of diagnosis. During National Heart Health Month, Rice Professor John McDevitt will discuss the potential of this technology to detect cardiac disease early at the annual meeting of the American Association for the Advancement of Science in Washington, D.C., February 17-21. Cardiac disease is the focus of one of six ongoing major clinical trials of Rice's programmable bio-nano-chips (PBNCs). PBNCs combine microfluidics, nanotechnology, advanced optics, and electronics to enable quick, painless diagnostic tests for a wide range of diseases at minimal cost. Current clinical trials employ PBNCs to test more than 4,000 patients for signs of heart disease (by analyzing a patient's saliva for biomarkers associated with cardiovascular disease), ovarian cancer, prostate cancer, oral cancer, and drug abuse. Versions to test for HIV/AIDS and other diseases are also in development. A video with a short discussion and demonstration of the technology is available here. Source: EurekAlert! 2/9/11

Researchers Lead Search for Better Drug Addiction Treatments

University of Texas Southwestern Medical Center psychiatry researchers are leading the Texas arm of a national network that conducts clinical trials aimed at finding effective treatments for drug addiction. More than 100 community treatment providers and academic medical centers throughout the country are funded in part through the National Institute on Drug Abuse's Clinical Trials Network. The Texas component includes partnerships between academic and community treatment providers in Dallas, El Paso, Austin, and Houston. Each network study is conducted in multiple community treatment provider sites across the country, led by a network substance abuse researcher and supported by the researchers in the network academic institutions affiliated with each participating site. One such national study is the Stimulant Reduction Intervention Using Dose Exercise (STRIDE), which tests the short- and long-term effectiveness of adding either exercise or health education to treatment as usual in adults who abuse stimulants such as cocaine or methamphetamine. Sites participating in this study include Nexus Recovery Center and Memorial Hermann Prevention and Recovery Center in Texas, as well as multiple other sites across the country. Other studies being conducted in the network in Texas include a trial that tests whether an interactive web-based therapy added to usual treatment improves abstinence from drug use, and a trial that examines whether medication, counseling, and incentives to quit smoking added to usual treatment can do the same. University of Texas Southwestern is partnered with the addiction treatment providers Nexus Recovery in Dallas, Homeward Bound in Dallas and El Paso, Memorial Hermann in Houston, and the Center for Healthcare Services in San Antonio. Source: EurekAlert! 2/2/11

Dots that Light Up Cancer Cells Enter Clinical Trials

"Cornell Dots"--brightly glowing nanoparticles--may soon be used to light up cancer cells to aid in diagnosing and treating cancer. The U.S. Food and Drug Administration (FDA) has approved the first clinical trial in humans of the new technology. It is the first time the FDA has approved using an inorganic material in the same fashion as a drug in humans. "The FDA approval finally puts a federal approval stamp on all the assumptions we have been working under for years. This is really, really nice," said Ulrich Wiesner, the Spencer T. Olin Professor of Materials Science and Engineering at Cornell University, who has devoted eight years of research to developing the nanoparticles. "Cancer is a terrible disease, and my family has a long history of it. I, thus, have a particular personal motivation to work in this area." The trial with five melanoma patients at Memorial Sloan-Kettering Cancer Center in New York City will seek to verify that the dots, also known as C dots, are safe and effective in humans, and to provide data to guide future applications. "This is the first product of its kind. We want to make sure it does what we expect it to do," said Michelle Bradbury, MD, radiologist at the cancer center and assistant professor of radiology at Weill Cornell Medical College. C dots are silica spheres less than 8 nanometers in diameter that enclose several dye molecules. The silica shell, essentially glass, is chemically inert and small enough to pass through the body and out in the urine. For clinical applications, the dots are coated with polyethylene glycol so the body will not recognize them as foreign substances. To make the dots stick to tumor cells, organic molecules that bind to tumor surfaces or even specific locations within tumors can be attached to the shell. When exposed to near-infrared light, the dots fluoresce much brighter than unencapsulated dye to serve as a beacon to identify the target cells. The technology, the researchers say, can show the extent of a tumor's blood vessels, cell death, treatment response, and invasive or metastatic spread to lymph nodes and distant organs. For the human trials, the dots will be labeled with radioactive iodine, which makes them visible in PET scans to show how many dots are taken up by tumors and where else in the body they go and for how long. Bradbury says the dots also may serve as a carrier to deliver radioactivity or drugs to tumors. "This is step one to jump-start a process we think will do multiple things with one platform," she said. First-generation Cornell dots were developed in 2005 by Hooisweng Ow, then a graduate student working with Wiesner. Wiesner, Ow, and Kenneth Wang '77 have cofounded the company Hybrid Silica Technologies to commercialize the invention. Wiesner's original research was funded by the National Science Foundation, New York state, and Phillip Morris USA. Source: EurekAlert! 1/31/11 For more information, click here. 

Ohio State University to Lead New Pancreatic Cancer Trial

Dr. Tanios Bekaii-Saab, medical director of gastrointestinal oncology at the Ohio State University Comprehensive Cancer Center, is leading a new, two-arm, randomized Phase II pancreatic cancer clinical trial that will study a formulation of the human reovirus that is designed to kill cancer cells. Expected to enroll approximately 70 patients with pancreatic cancer, both at Ohio State and at other institutions, the study will compare the effects of two courses of treatment. One group of patients will be given the chemotherapy drugs carboplatin and paclitaxel. The other group will be given these two medications plus Reolysin, an engineered version of the human reovirus developed by Oncolytics Biotech Inc. of Calgary. Reolysin works by replicating inside of, and subsequently destroying, cancer cells that have a certain characteristic (called an activated RAS pathway) that is shared by about two-thirds of all cancers. The drug targets tumor cells and leaves healthy, normal cells unharmed. Reolysin appears to kill cancer cells by rupturing their walls. In the trial, patients will receive intravenous doses of medication every three weeks and undergo CT scans for tumor response assessment every eight weeks. Patients whose condition does not improve with carboplatin and paclitaxel alone will have Reolysin added to their treatment. The study is sponsored by the Cancer Therapy Evaluation Program, Division of Cancer Treatment and Diagnosis, U.S. National Cancer Institute, which is a part of the National Institutes of Health. Ohio State researchers are also conducting clinical trials with Reolysin in patients who have lung cancer and ovarian cancer. Source: Newswise 1/25/11

First Liver Transplant Patients Receive Experimental Drug to Prevent Hepatitis C Infection

Following a successful Phase I study for safety, researchers at MassBiologics of the University of Massachusetts Medical School in January announced the beginning of a Phase II clinical trial testing the ability of a human monoclonal antibody they developed to prevent hepatitis C virus (HCV) infection of a donor liver in transplant patients. The first patients were enrolled in the study in December. The primary goal of this randomized, double-blind, placebo-controlled study is to test if the monoclonal antibody, designated MBL-HCV1, prevents re-infection of patients chronically infected with HCV who are undergoing liver transplantation. MassBiologics plans to enroll 16 patients in the first part of the study. There are currently five hospitals participating in the trial--Massachusetts General Hospital and Beth Israel Deaconess Medical Center in Boston, Lahey Clinic in Burlington, Mass., Yale-New Haven Hospital in Connecticut, and Mount Sinai Hospital in New York City--and others may join in the coming months. Patients will be randomized to receive an infusion of either the antibody or placebo between one and four hours prior to surgery. Then, during the phase of surgery when the diseased liver is removed, but before the donor liver is implanted, patients will receive a second infusion of either the antibody or placebo. After the surgery is completed, the patients will receive a third infusion, and then daily infusions during the first week of recovery. A final infusion is administered on the 14th day after liver transplantation. After transplantation, patients' blood will be tested on a regular basis to screen for reemergence of HCV, which is usually detected within the first week after transplantation. The primary goal of the Phase II trial is to see if the patients who received the antibody are free of HCV at 42 days after transplantation. An interim analysis is planned after the first 16 patients have been enrolled in the trial, and a data safety and monitoring board overseeing the study will assess the effectiveness and safety of MBL-HCV1. Source: EurekAlert! 1/18/11

Clinical Trial Studies Safety of Malaria Drug for Melanoma Use

Building upon recent laboratory discoveries on resistance by cancer cells to therapies that attempt to starve cancer, scientists at the Cancer Institute of New Jersey (CINJ) are conducting a clinical trial that further explores how to prevent that action. The goal is to discover if an antimalaria drug approved by the Food and Drug Administration is able to block a cellular process that acts as a survival method for malignant cells in human melanoma, the most life threatening form of skin cancer. Hydroxychloroquine, which is commonly used to treat malaria and certain types of arthritis, has been shown to block autophagy, the process by which cells eat themselves in order to survive through times of stress. This process allows cells to become resistant to cancer therapies. Research by CINJ Associate Director for Basic Science Eileen White, PhD, adjunct professor of surgery at UMDNJ-Robert Wood Johnson Medical School and professor of molecular biology and biochemistry at Rutgers, the State University of New Jersey, and other laboratories at CINJ indicates that drugs such as hydroxychloroquine may help cancer treatments work more effectively by providing an easier path for the cancer drugs to enter the cell. In this trial, investigators want to learn how to detect the process of autophagy in humans with melanoma, a goal lead researcher Janice Mehnert, MD, medical oncologist at CINJ and assistant professor of medicine at UMDNJ-Robert Wood Johnson Medical School, says is key. “By further examining the process of autophagy and learning if hydroxychloroquine is able to interrupt this process in human blood and tumor samples, we may be able to find a way to maximize the benefit of hydroxychloroquine in order to improve clinical outcomes,” she said. Adults with stage III or stage IV melanoma tumor or tumors (cancer that has spread beyond where it first occurred) who can be scheduled for surgery to either cure the cancer or lessen the symptoms of cancer are eligible to take part in the trial, although other criteria must be met. Patients with newly diagnosed melanoma tumors or who have disease that has returned also are eligible to participate. The trial is supported by grants from the National Cancer Institute and the Harry Lloyd Foundation. Source: Newswise 1/13/11

Sanofi-Aventis Enters Research and Development Collaboration with UCSF

Sanofi-aventis and the University of California, San Francisco (UCSF) have formed two research and development collaborations that join together leaders from academia and industry to more rapidly advance groundbreaking innovation from the lab to the patient. The first is an oncology partnership that will focus on project-based collaboration to accelerate research progress through the clinical proof-of-concept stage. The second collaboration promotes innovative research in pharmacological science and in multiple therapeutic areas, such as oncology, aging, diabetes, and inflammation. Sanofi-aventis will be the first industry partner for UCSF's Program for Breakthrough Biomedical Research (PBBR). PBBR awards funding to projects of potentially high impact and greater creativity, and which offer innovative approaches to scientific discovery. The partnership with UCSF enhances sanofi-aventis' approach of setting up a unique structure revolving around networks of creativity spread across regions, technologies, and scientific areas of excellence. Source: EurekAlert! 1/12/11

Bipolar Trial Site Launched with AHRQ Grant

The Mood Disorders Program at Case Western Reserve University School of Medicine and University Hospitals Case Medical Center, along with the Bipolar Trials Network, is launching Bipolar CHOICE (Clinical Health Outcomes Initiative in Comparative Effectiveness). The 10-site nationwide trial evaluating the real-world advantages and disadvantages of quetiapine, a widely prescribed second-generation antipsychotic mood-stabilizing medication, compared to lithium, the gold standard mood stabilizer, for the treatment of outpatients with bipolar disorder. The study is funded by a $600,000 grant to Case Western Reserve University School of Medicine from the Agency for Healthcare Research Quality (AHRQ), and began enrolling patients on January 11. “Studies that compare treatments for bipolar disorder usually don’t allow participants to take other medications, and most patients need several medications to feel and stay well," says Joseph R. Calabrese, MD, professor of psychiatry at Case Western Reserve School of Medicine, director of the Mood Disorders Program at University Hospitals Case Medical Center, and principal investigator of Bipolar CHOICE. “Bipolar CHOICE is unique because participants can be treated, not only with quetiapine or lithium, but with almost any other medication excluding other antipsychotics. ...Future trials need to focus on effectiveness, not just on whether one treatment appears better than placebo. We need studies that inform the day-to-day practice of psychiatry in a meaningful way. With real-world studies in psychiatry viewed as being unusual, this study has the potential of doing just that.” The AHRQ grant is part of an investments made under the American Recovery and Reinvestment Act of 2009, which designated $1.1. billion to support patient-centered outcomes research. This research is designed to inform healthcare decisions by providing evidence and information on the effectiveness, benefits, and harms of different treatment options. Ten grants totaling $100 million were awarded across the country, and this particular grant was the only one awarded to study mental illness. Source: Newswise 1/11/11

Study Shows Promise for New Drug to Treat Fragile X

The first drug to treat the underlying disorder instead of the symptoms of Fragile X, the most common cause of inherited intellectual disability, shows some promise, according to a new study published in the January issue of Science Translational Medicine. Researchers from Rush University Medical Center helped design the study and are now participating in the larger follow-up clinical trial. The data from the early trial of 30 Fragile X patients found the drug, called AFQ056 (Novartis Pharmaceuticals), helped improve symptoms in some patients. Patients who had the best response have a kind of “fingerprint” in their DNA that could act as a marker to determine who should get treatment. “This is an exciting development. It is the first time we have a treatment targeted to the underlying disorder, as opposed to supportive treatment of the behavioral symptoms, in a developmental brain disorder causing intellectual disability. This drug could be a model for treatment of other disorders such as autism,” said pediatric neurologist Dr. Elizabeth Berry-Kravis, a study author and director of the Fragile X Clinic and Research Program and the Fragile X-Associated Disorders Program at Rush. The drug is designed to block the activity of mGluR5, a receptor protein on brain cells that is involved in most aspects of normal brain function, including regulation of the strength of brain connections, a key process required for learning and memory. Fragile X patients have a mutation in a single gene, known as Fragile X Mental Retardation-1 or FMR1. The mutation prevents FMR1 from making its protein, called FMRP, such that FMRP is missing in the brain. FMRP normally acts as a blocker or “brake” for brain cell pathways activated by mGluR5. When FMRP is missing, mGluR5 pathways are overactive, resulting in abnormal connections in the brain and the behavioral and cognitive impairments associated with Fragile X. The research team, led by Sebastien Jacquemont of Vaudois University in Switzerland in collaboration with Baltazar Gomez-Mancilla of Novartis, found no significant effects of treatment when the entire group of 30 patients was analyzed. However, in a subsequent analysis, seven patients who had a fully methylated gene, a gene that was fully shut down, presumably resulting in no FMR protein in the blood or brain, showed significant improvement in behavior, hyperactivity, and inappropriate speech with the treatment compared to placebo. A larger study of the drug is now under way that will recruit 160 patients worldwide and test the effects of a longer period of treatment. Rush University Medical Center is one of the participating sites. Source: Newswise 1/7/11

Clinical Trials Under Way to Test Anti-Obesity Compound

Clinical trials started in December 2010 to determine how well an anti-obesity compound derived from a Chinese herb works in humans. South Dakota State University (SDSU) researcher Gareth Davies, the scientific director for the Avera Institute for Human Behavioral Genetics in Sioux Falls, said collaborators from SDSU and Avera have published several articles about the compound, which they call AIHBG-10. Davies, formerly an associate professor in SDSU’s Department of Pharmaceutical Sciences, left his university post as of January 1 to concentrate solely on his work with Avera. He remains an adjunct associate professor at South Dakota State. “We studied a natural product used extensively in Chinese medicine to treat obesity,” Davies said. “We showed that we can use this compound to stop the development of fat cells and to change the gene expression in these cells and basically prevent them developing from a pre-fat cell to a fat cell. We studied the response in cell lines and in animals, and now Avera is beginning a clinical trial in humans.” A second clinical trial beginning in early 2011 will use the anti-obesity compound in individuals who are being treated with anti-psychotic drugs in order to determine how well it reduces the weight gains associated with such treatments. Source: Newswise 12/30/10

Clinical Trial Launched for Rett Syndrome

Researchers at Children's Hospital Boston have begun a randomized, placebo-controlled trial to test a potential drug treatment for Rett syndrome, the leading known genetic cause of autism in girls. The drug, mecasermin, a synthetic form of insulin-like growth factor-1 (IGF-1), is already approved by the Food and Drug Administration for children with short stature due to IGF-1 deficiency. The trial, now enrolling patients, marks the beginning of a trend toward drug treatments seeking to modify the underlying causes of autism spectrum disorders, rather than just behavioral symptoms such as anxiety or aggression. The clinical trial is funded by the International Rett Syndrome Foundation, Autism Speaks, and Harvard University's Catalyst Pilot Awards for Clinical Translational Research. Source: EurekAlert! 12/16/10

It's Time for a New Approach to Alzheimer's Disease

Karl Herrup thinks that the national research effort to understand Alzheimer’s disease has gone about as far as it can go with its current theories. And that’s not far enough. Researchers have spent many years and many millions of dollars looking for ways to prevent plaques as a way of treating, curing, or preventing Alzheimer’s. In recent years, however, dozens of human clinical trials based on this theory have failed. Herrup, the chair of the Department of Cell Biology and Neuroscience at Rutgers University, suggests an alternative perspective, which he has set forth in a paper published in the Journal of Neuroscience. Pointing out that age is the most important risk factor in the disease, he suggests a new hypothesis with age as the starting point. Herrup believes three three key steps that are needed for an individual to progress from the natural path of age-related changes in the brain to the full spectrum of Alzheimer’s clinical symptoms: an initiating injury that is probably vascular in nature; an inflammatory response that is both chronic and unique to Alzheimer’s; and a cellular change of state, a one-way cell biological door that permanently alters the physiology of neurons and several other cell types in the Alzheimer’s disease brain. “My hypothesis implies that beta-amyloid aggregation is not a central part of the biology of Alzheimer’s disease,” Herrup says. “It predicts that one can have plaques without having Alzheimer’s and that one can have Alzheimer’s without having plaques.” Source: EurekAlert! 12/14/10

Drugs are Safe, Active in Patients Normally Ineligible for Clinical Trial

A two-drug combination is safe and active in newly diagnosed acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) patients who are usually excluded from clinical trials because they have other illnesses or poor performance status, researchers reported at the 52nd American Society of Hematology Annual Meeting. "Our findings suggest current eligibility standards that prevent participation by these patients in Phase I and Phase II clinical trials might be inadequate," said Guillermo Garcia-Manero, MD, professor in the University of Texas MD Anderson Cancer Center's Department of Leukemia, who presented early results of an ongoing Phase II clinical trial. Such patients also are not eligible for standard therapies. The drug combination of 5-azacitidine and vorinostat is known to be safe and active against AML and MDS among patients without the complications that participants in this trial have, Garcia-Manero said. So far, 27 patients have been treated, nine with AML, 18 with MDS. At a median follow up of 3.6 months, 20 of 24 survived past 60 days (83 percent). One patient died during therapy, two died of disease progression after going off the study. Fifteen could be evaluated for response to the combination, seven (46 percent) had complete responses. Stable disease was noted in two patients. "This combination is safe and active for these poor-prognosis patients at similar levels of safety and activity seen among patients who are eligible for clinical trials," Garcia-Manero said. The clinical trial is supported by Merck & Co., makers of vorinostat, and Pharmion Corp., which makes azacitidine. Source: EurekAlert! 12/10/10

Research Fuels Hope for Hard-To-Treat Hepatitis C Patients

The outlook for patients with hepatitis C continues to improve, as results from a clinical trial led by a Saint Louis University researcher found that the drug boceprevir helped cure hard-to-treat patients. The findings were reported at the 61st annual meeting of the American Association for the Study of Liver Diseases in November. Bruce R. Bacon, MD, professor of internal medicine at university's School of Medicine and co-principal investigator of the HCV RESPOND-2 study, studied the protease inhibitor, boceprevir, and found that it significantly increased the number of patients whose blood had undetectable levels of the virus. The study looked at 403 patients with chronic hepatitis C infections with genotype one, the most difficult strain of the virus to treat, who still had significant levels of the virus after being treated with peginterferon and ribavirin, the standard hepatitis C treatment. "These results are very exciting," Bacon said. "In this study, boceprevir helped cure significantly more patients in 36 weeks of therapy than did treatment with peginterferon and ribavirin alone." A second study, HCV SPRINT-2, examined patients with hepatitis C with genotype one who had not yet been treated with the standard treatment. They, too, responded well to the drug. The clinical trial was funded by Merck, which expects to begin seeking Food and Drug Administration approval this year. Source: Saint Louis University 12/8/10

Therapeutic Cooling Studied to Reduce Brain Injury after Stroke

The largest clinical trial of therapeutic brain cooling (hypothermia) after stroke has launched, led by researchers at the University of California, San Diego (UCSD), the University of Texas (UT) Health Science Center at Houston, and at Cedars-Sinai Medical Center in Los Angeles. This study looks at whether hypothermia can safely be used in elderly stroke patients. In earlier studies, brain cooling decreased brain swelling after an acute stroke. It also saved lives and prevented neurological damage after cardiac arrest and after oxygen deprivation in newborns. “We know hypothermia works, but is it safe when you consider age and other conditions such as diabetes or hypertension?” said Patrick D. Lyden, MD, former director of the UCSD Stroke Center who now serves as the chairman of the Department of Neurology at Cedars-Sinai. He is the study’s overall principal investigator. Thomas Hemmen, MD, PhD, director of the UCSD Stroke Center, and James C. Grotta, MD, chairman of the Department of Neurology at UT Health, are the principal investigators at their sites. The study employs an advanced temperature modulation system that provides quick and controlled cooling. A metallic cooling catheter is inserted into the body’s largest vein, the inferior vena cava. No fluid enters the patient, but fluid circulating inside the catheter transfers heat out. Study participants are covered with warming blankets to trick the body into feeling warm, which together with a mild sedative helps suppress shivering. In this study, body temperature will be cooled to 33 degrees C (about 91 degrees F) and maintained at that level for 24 hours. Participants then will be gradually rewarmed over 12 hours. Philips Healthcare, the InnerCool system’s developer, is providing the equipment and catheters. The study will enroll 400 patients at up to 26 sites in the United States and Europe and is sponsored by the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health. It is a single-blind, randomized, controlled trial. Source: Newswise 12/7/10

Personalized Vaccine for Lymphoma Patients Extends Disease-Free Survival

A personalized vaccine is a powerful therapy to prevent recurrence among certain follicular lymphoma patients, according to the latest results of ongoing research led by the University of Pennsylvania School of Medicine. The new findings show that when these patients--whose tumors are marked by a specific protein that may be present in up to half of people with this type of cancer--receive a vaccine made from their own tumor cells, disease-free survival is improved by nearly two years, compared to patients who receive a placebo. “The treatment effect of the personalized vaccine is stunning in our trial,” says Stephen J. Schuster, MD, an associate professor in the division of hematology-oncology and director of the lymphoma program at Penn’s Abramson Cancer Center. Schuster presented data from a randomized, double-blind, Phase III multicenter clinical trial in early December at the annual meeting of the American Society of Hematology. In the current study, individuals who responded to initial chemotherapy and remained in remission for at least six months were eligible to continue in the trial, and received either a personalized idiotype vaccine plus an immune-stimulating agent called GM-CSF, or placebo vaccine plus GM-CSF. When researchers analyzed the patients who received at least one dose of personalized vaccine, they saw a 14-month improvement in disease-free survival, compared to those who received the placebo. Following a hunch and some earlier observations in the literature, Schuster and colleagues reanalyzed the data after dividing the patients based on the type of isotype protein on their tumor cells’ surface, and found that patients whose tumors had an IgM-idiotype protein had a robust response to the vaccine, with an increase in median disease-free survival from 28.7 months in placebo-treated patients to 52.9 months in vaccine-treated patients. To confirm their novel results, Schuster’s team plans to launch a new trial next year using the same vaccine approach, but this time they will divide patients at the outset, according to whether their idiotype protein is an IgM- or IgG-type. The trial was supported by Biovest International, Inc. and the National Cancer Institute. Source: Newswise 12/6/10

Drug Development Partnership Capitalizes on University's Success in Nanomedicine Research

University of Missouri leaders celebrated a nanomedicine milestone in early December as they announced the creation of a new drug development company, Shasun Nanoparticle Biochem Inc. The new company forms a partnership between university researchers and an international pharmaceutical firm to advance testing of a potential cancer treatment created at the university. Shasun Pharmaceuticals Ltd., one of the world's leading suppliers of ibuprofen, formed the new company in partnership with nanomedicine experts Kattesh Katti, PhD, and Raghuraman Kannan, PhD. The scientists are developing a promising prostate cancer treatment that uses gold nanoparticles they created at the university. The new company will continue research and testing that is required for the treatment to receive approval for use in humans. Katti and Kannan have studied their new therapy for treating late-stage prostate cancer for more than five years. Their efficacy studies in mice with prostate tumors have demonstrated an unprecedented 85 percent reduction in tumor volume after administering a single dose of their radioactive gold nanoparticle. They have observed little or no toxic side effects as a result of the injected dose. "This is probably the most promising result we could expect," said Andrew Kurtz, PhD, a program manager at the National Cancer Institute's Small Business Innovation Research Development Center. "The next stage would be to hopefully move things into clinical trials to see if we find the same results in humans." The therapy will advance toward human testing with support from a $1.5 million initial investment by Shasun. The money will help the university hire scientists and conduct further laboratory studies. If the therapy is approved for humans, patients at the university's hospitals and clinics could be among the first to benefit from the cancer treatment. Shasun was incorporated in 1976 and is headquartered in Chennai, India. It manufactures active pharmaceutical ingredients and exports pharmaceutical products to countries across Europe, North America, and elsewhere. Source: EurekAlert! 12/2/10

International Clinical Trial Tests Targeted Drug for Melanoma

Rush University Medical Center in Chicago has just enrolled the first U.S. patient in an international clinical trial testing a novel drug to treat certain kinds of melanoma, a deadly skin cancer that in its advanced stages currently has few effective treatments. Rather than blocking or killing all rapidly dividing cells, whether malignant or not, the drug, called nilotnib, is one of a new class of agents that have been designed to sabotage aberrant molecules characteristic of individual cancers--in this case, the c-kit protein. Dr. Howard Kaufman, director of the Rush University Cancer Center and lead investigator of the study at Rush, said that this kind of "targeted" therapy holds out hope of transforming cancer from a lethal disease into a chronic, but manageable disease. "For advanced melanoma, there are currently few satisfactory treatments," Kaufman said. "But new targeted therapies, including vaccines, antibodies and small molecules like nilotnib are in clinical trials now, adding to an arsenal of treatments that appear to be promising. This trial is especially significant since the c-kit mutation is found more commonly in melanoma arising from the mucosa and foot, which are historically very difficult types of melanoma to treat." Nilotnib, marketed by Novartis under the brand name Tasigna, is an oral drug approved by the Food and Drug Administration for the treatment of chronic myelogenous leukemia, but is now being tested for the first time for the treatment of melanomas that express the c-kit gene. As a small molecule, nilotnib is able to slip across the cell's membrane and into the machinery inside. There it targets, and turns off, the abnormal c-kit protein, created by a mutated c-kit gene, shutting it down and thus disrupting the relay team of molecular signals the protein participates in that ultimately spur cell growth and cause melanoma lesions to proliferate. Patients with melanomas expressing a mutated c-kit gene are eligible to participate in the study. These types of melanomas, which typically occur in mucosal tissue, the eye or the foot, are extremely aggressive. The aim of the trial is to determine whether nilotnib can block the growth and spread of this kind of melanoma and extend life. Participants will receive either nilotnib or dacarbazine, a chemotherapy drug commonly used to treat advanced melanoma. Those in the latter group will be able to begin receiving nilotnib if their cancer progresses after dacarbzaine treatment. Source: EurekAlert! 11/30/10

Implanted Devices as Effective in "Real World" as in Clinical Trial Settings

Implanted devices that treat cardiac dysfunction in heart failure patients are as successful in "real world" use as they are in controlled clinical trial settings, according to a large new study reported in Circulation: Journal of the American Heart Association. In the study, researchers focused implantable cardioverter defibrillators (ICDs), cardiac resynchronization therapy (CRT), and CRT-Ds (defibrillators with a CRT device). Researchers examined data on 185,778 ICD and CRT patients (average age 67). After one year, 92 percent of ICD recipients and 88 percent of CRT-D patients were alive. Survival rates after five years were 68 percent for ICD and 54 percent for CRT-D. Among 8,228 CRT-only patients, the survival rate was 82 percent at one year and 48 percent at five years. "I'm very encouraged that survival after defibrillator implant is as good as it is for as long as it is," said Leslie A. Saxon, MD, co-author of the study. The data complement previous results of clinical trials that evaluated ICDs for primary prevention of sudden death. Real world patients were expected to fare worse because they could also be using an ICD for secondary prevention (recurrent event), which puts them at higher risk. The study also found that patients whose devices were subject to ongoing remote monitoring on a network were about half as likely to have died as were those receiving traditional in-clinic monitoring only, which typically occurs quarterly. Cardiologists decide whether to enroll patients in a remote monitoring network, which is available only for ICD and CRT-D devices and is free. Enrolled patients are mailed a communicator unit that connects to their telephone line and queries the implant daily about how it is functioning and whether cardiac events occurred. The data are delivered to a website the physician monitors. The communicator can also help monitor other health indicators, dispatching data on factors such as fatigue, weight or blood pressure. Changes in weight and blood pressure can predict worsening status. Patients with CRT-Ds whose communicators regularly transmitted weight and blood pressure data had a lower risk of death than did other networked CRT-D patients. Because the network gives a continuous view of patients' status--and provides alerts when something is wrong--physicians can more effectively triage their patients and intervene immediately. The new study is part of ongoing research called the ALTITUDE project, involving physicians from across the United States who are learning how to optimize ICD, CRT and CRT-D devices. Boston Scientific, a manufacturer of the devices, funded the research. Source: EurekAlert! 11/19/ 10

Cancer Center Named Center of Excellence for Phase I Clinical Trials

The University of Southern California (USC) Norris Comprehensive Cancer Center has been designated a Phase I Clinical Trial Center of Excellence by Bristol-Myers Squibb Co. The collaboration will bring a number of new Phase I clinical trials to USC Norris, and will enable the cancer center’s clinical and basic scientists to provide guidance in early phase drug development and trial design. USC Norris is one of only 39 centers in the United States designated as "comprehensive" by the National Cancer Institute. The center currently enrolls approximately 150 patients per year in Phase I clinical trials, which test the safety and efficacy of promising new drugs and therapies. USC Norris is among a handful of global sites and one of two in the United States to be selected for this designation from the pharmaceutical company. The selection was based on several factors, including the quality of the investigators and research staff, the clinical research infrastructure, and the ability of the clinician-scientists to be active partners in the design of the clinical and translational research studies. The clinical studies conducted at USC Norris will cover a range of cancers, including blood cancers and solid tumors. Source: Newswise 11/18/10

Medical Center Joins Idiopathic Pulmonary Fibrosis Clinical Research Network

Beth Israel Deaconess Medical Center will join the Idiopathic Pulmonary Fibrosis Clinical Research Network (IPFnet), a clinical research network sponsored by the National Heart Lung and Blood Institute to conduct randomized clinical trials to evaluate new and existing medications for the treatment of IPF. The network is made up of healthcare institutions from around the United States. Other than lung transplantation, there are no proven therapies currently in existence to treat IPF, a respiratory disorder characterized by cough and shortness of breath that affects between 80,000 and 100,000 individuals in the U.S. While pulmonary fibrosis can, in some cases, be linked to a particular cause--such as environmental exposures, chemotherapy, radiation therapy, infection, or autoimmune diseases--in many instances, no cause it is established. It is these cases that are referred to as “idiopathic pulmonary fibrosis.” The IPFnet was created in 2005 to find effective therapies for patients with both early and advanced stages of the disease. Source: Newswise 11/15/10

Children's Hospital Leading Landmark Batten Study

Oregon Health & Science University (OHSU) Doernbecher Children's Hospital will lead the next phase of a landmark clinical trial to further assess the safety and preliminary effectiveness of purified human neural stem cells (HuCNS-SC®) as a potential treatment for infantile or late-infantile neuronal ceroid lipofuscinosis (NCL), a rare and currently fatal neurodegenerative disorder that affects infants and children. The Phase Ib trial, sponsored by StemCells, Inc., is designed to further assess the safety and preliminary effectiveness of StemCells' HuCNS-SC as a potential treatment for NCL, also referred to as Batten disease. This second trial is expected to enroll six children whose NCL is less advanced than those enrolled in the initial Phase I trial, also carried out at OHSU Doernbecher, and, in addition to safety, is designed to evaluate the impact of HuCNS-SC on disease progression. Nathan Selden, MD, PhD, FACS, FAAP, a professor of pediatric neurosurgery and head of the Division of Pediatric Neurological Surgery at the hospital, was co-principal investigator on initial Phase I trial and will lead the Ib study. The initial trial in six children was the first-ever Food and Drug Administration-authorized clinical trial of purified human neural stem cells as a potential therapeutic agent; it found that high doses of HuCNS-SC transplanted directly into multiple sites within the brain followed by 12 months of immunosuppression were well tolerated. The data also included evidence of engraftment and long-term survival of the donor cells. Participants in the new study will be transplanted with HuCNS-SC via a neurosurgical procedure and their immune systems will be suppressed for nine months. Following transplantation, the children will be monitored and regularly evaluated for 12 months to assess the safety and tolerability of the HuCNS-SC, the surgery, and the immunosuppression. In addition, participants will be evaluated and assessed at regular intervals using a comprehensive range of clinical and X-ray tests, both before transplantation to establish a baseline, and over the course of 12 months following transplantation. StemCells, Inc. will initiate a separate four-year observational study at the conclusion of this trial. Source: EurekAlert! 10/29/10 

Medical School Receives $45 Million Donation for Celiac Research

With a new $45 million private gift from the family of a grateful patient, the University of Maryland School of Medicine is planning to establish the nation's only major research enterprise devoted to the study of autoimmune and inflammatory diseases such as celiac disease, multiple sclerosis, chronic obstructive pulmonary disease, asthma, and type 1 diabetes. The gift, from Indiana couple Ken and Shelia Cafferty, is the largest private donation in the history of the University System of Maryland. The planned research enterprise will be a full-fledged, multidisciplinary academic organization that includes and expands upon two of the school's research centers, the Mucosal Biology Research Center and the Center for Celiac Research, which are directed by Alessio Fasano, MD, a celiac disease researcher and professor of pediatrics, medicine, and physiology at the School of Medicine who will lead the new research enterprise. The enterprise will initially include 13 faculty members, with more to be recruited in the future. Fasano envisions it employing as many as 200 people once it is up and running. "We are assembling a critical mass of multidisciplinary expertise, building the best infrastructure that we can in order to investigate inflammation and autoimmunity from every possible medical and scientific perspective," says Fasano. "Our effort will involve both basic and clinical scientists, and will capitalize on the complementary expertise of our institutes and organized research centers on campus. This thorough, multidisciplinary approach will help us find answers to our questions as quickly and efficiently as possible. There is simply no other way to do it, and this incredibly generous gift makes it possible. We are grateful to the Caffertys for recognizing the potential of this science and putting their faith in our world-class researchers." Source: EurekAlert! 10/28/10 

Clinical Trials Demonstrate Effective Weight Loss Strategies

Lifestyle interventions, including physical activity and structured weight loss programs, can result in significant weight loss for overweight, obese, and severely obese adults, according to two reports in the Journal of the American Medical Association. In a one-year intensive lifestyle intervention study of diet and physical activity, Bret H. Goodpaster, PhD, from the University of Pittsburgh School of Medicine, and colleagues randomized a group of 130 severely obese adult individuals without diabetes in two groups to assess weight loss for a period of one year. One group was randomized to diet and physical activity for the entire 12 months, while the other group had the identical dietary intervention, but with physical activity delayed for six months. The study was conducted from February 2007 with follow-up through April 2010. "Of the 130 participants randomized, 101 (78 percent) completed the 12-month follow-up assessments," the authors state. The group that started with the diet and physical activity lost more weight in the first six months than the delayed-activity group (about 24 pounds as compared to 18 pounds). However, the authors report that weight loss at 12 months was about the same in the two groups (almost 27 pounds versus about 22 pounds). "Waist circumference, visceral abdominal fat, hepatic (liver) fat content, blood pressure, and insulin resistance were all reduced in both groups," according to the authors. This study was funded by the Commonwealth of Pennsylvania Department of Health. Coauthor Jolene Brown, MD, was supported by a National Institutes of Health/National Institute of Diabetes and Digestive and Kidney Diseases grant. Source: EurekAlert! 10/26/10 

Chemotherapy Plus Radiation Prevents Bladder Cancer Recurrences

Adding chemotherapy to radiation therapy for muscle invasive bladder cancer allows 67 percent of people to be free of disease in their bladders two years after treatment. This compares to 54 percent of people who receive radiation alone, according to the largest randomized study of its kind presented at the plenary session on November 1 at the 52nd Annual Meeting of the American Society for Radiation Oncology. "The trial shows that this treatment offers improved control of cancer within the bladder with acceptable long-term side effects and is therefore a viable alternative to radical surgery in patients with muscle invasive bladder cancer," said Nicholas James, MD, an oncologist at University of Birmingham in the United Kingdom, who jointly led the study with Robert Huddart, MD, from the Institute of Cancer Research in the United Kingdom. "This may shift the balance from surgery to chemo-radiotherapy as the primary treatment for many patients with invasive bladder cancer." One common treatment for advanced bladder cancer is complete removal of the bladder, which compromises patients' normal urinary function. Radiotherapy has been used as an alternative for some time. Doctors have been making advances in combining radiation therapy and chemotherapy as a way to treat bladder cancer while allowing patients an opportunity to maintain normal bladder function. This multicenter randomized trial, conducted at 45 institutions in the United Kingdom, examined whether adding chemotherapy to radiation treatment is safe and more effective than giving radiation alone in preventing bladder cancer from returning. The trial also compared two ways of giving radiation therapy. From August 2001 to April 2008, 458 invasive bladder cancer patients entered the trial. Results of the study show that the combination of chemotherapy and radiation treatment reduced the long-term risk of recurrence of cancer within the bladder, while also preserving bladder function. This work was supported by Cancer Research U.K. Source: EurekAlert! 10/25/10 

Researchers Develop First Implanted Device to Treat Balance Disorder

A University of Washington Medical Center patient on October 21 was slated to be the world's first recipient of a device that aims to quell the disabling vertigo associated with Meniere's disease. The clinicians who developed the implantable device hope that success in a 10-person surgical trial of Meniere's patients will lead to exploration of its usefulness against other common balance disorders that torment millions of people worldwide. The device being tested--a cochlear implant and processor with re-engineered software and electrode arrays--represents four-plus years of work by Drs. Jay Rubinstein and James Phillips of the university's Department of Otolaryngology-Head and Neck Surgery. They worked with Drs. Steven Bierer, Albert Fuchs, Chris Kaneko, Leo Ling, and Kaibao Nie, specialists in signal processing, brainstem physiology, and vestibular neural coding. The disease affects hearing and balance with varying intensity and frequency but can be extremely debilitating. Its episodic attacks are thought to stem from the rupture of an inner-ear membrane. Endolymphatic fluid leaks out of the vestibular system, causing havoc to the brain's perception of balance. To stave off nausea, afflicted people must lie still, typically for several hours and sometimes up to half a day while the membrane self-repairs and equilibrium is restored. With their device, Phillips and Rubinstein aim to restore the patient's balance during attacks while leaving natural hearing and residual balance function intact. A patient wears a processor behind the affected ear and activates it as an attack starts. The processor wirelessly signals the device, which is implanted almost directly underneath in a small well created in the temporal bone. The device in turn transmits electrical impulses through three electrodes inserted into the canals of the inner ear's bony labyrinth. "It's an override," Phillips said. "It doesn't change what's happening in the ear, but it eliminates the symptoms while replacing the function of that ear until it recovers." A National Institutes of Health grant funded the development of the device and its initial testing at the Washington National Primate Research Center. The promising results from those tests led the U.S. Food and Drug Administration, in June, to approve the device and the proposed surgical implantation procedure. Shortly thereafter, the limited surgical trial in humans won approval from the Western Institutional Review Board, an independent body charged with protecting the safety of research subjects. Source: EurekAlert! 10/21/10

Researchers Help Mothers Cope with Child's Cancer

Mothers who have children diagnosed with cancer now have a better approach to address and cope with stresses associated with their child's disease. A new certified intervention has proven to be more effective long term compared to other psychological methods, as reported in October at the 42nd Congress of the International Society of Pediatric Oncology. In a joint oral presentation, researchers from the University of Texas MD Anderson Children's Cancer Hospital and Jonathan Jaques Children's Cancer Center of Miller Children's Hospital Long Beach reported that mothers of newly diagnosed patients were able to decrease their stress level sooner and sustain that level longer with an intervention known as Problem-Solving Skills Training (PSST). The multi-institutional randomized trial, conducted through the Psychosocial Adaptation to Childhood Cancer Research Consortium, also showed that Spanish-speaking mothers had the most significant response to the training compared to English-speaking and Arabic-speaking mothers. Three months after their child's initial diagnosis, the stress levels of mothers receiving PSST had decreased twice as much as mothers who had no intervention. In the consortium's 2009 study, researchers also evaluated PSST in comparison to reflective listening, a form of one-on-one counseling. Although both therapies decreased the stress level significantly, mothers counseled only with reflective listening eventually returned to higher stress levels after three months, unlike those who had PSST. In addition, the study evaluated the efficacy and feasibility of using a personal digital assistant (PDA) to supplement the training. Although there was no significant benefit reported from using a PDA in coordination with PSST, participants rated the PDA-based program favorably, which could lead to more technology-based interventions in the future, said the co-presenters. The PSST intervention was developed through the consortium. The training consists of eight, one-hour individual sessions between mother and therapist, in which they identify the mother's primary stressors, brainstorm solutions, weigh the benefits and costs associated with each solution, implement one of the solutions, and evaluate its effectiveness. Funding for the study was provided through the National Institutes of Health. Other study collaborators include researchers from Golisano Children's Hospital at Strong, the University of Colorado Health Sciences Center, Oregon Health & Science University, Children's Hospital Los Angeles, Schneider Children's Medical Center, Texas A&M University, Children's Hospital of Pittsburgh, and St. Jude Children's Research Hospital. Source: EurekAlert! 10/21/10

New Clinical Trial Explores Role of Vitamin D in Preventing Esophageal Cancer

In a first-of-its-kind clinical trial, physicians at University Hospitals Case Medical Center who are Case Western Reserve University School of Medicine researchers are exploring the role of Vitamin D in preventing esophageal cancer. Principal investigator Linda Cummings, MD, along with Amitabh Chak, MD, and Gregory Cooper, MD, from the Digestive Health Institute, is recruiting patients with Barrett's esophagus to measure the effects of Vitamin D on protein levels that may influence the risk of developing esophageal cancer. "Vitamin D is being studied for its role in possibly reducing the risk of developing several types of cancer, such as colon, breast and prostate," says Dr. Cummings, a gastroenterologist with the institute and assistant professor at the medical school. According to coinvestigator Sanford Markowitz, MD, Ingalls Professor of Cancer Genetics at the medical school and oncologist with the medical center, the study "has the potential to make a highly important contribution to the medical management of Barrett's esophagus, which is becoming an ever increasing challenge." The study is funded by the National Institutes of Health and American College of Gastroenterology. In this study, researchers are examining if Vitamin D intake is linked to an increase in a protein called 15-PGDH (15-prostaglandin dehydrogenase), which may help prevent Barrett's from turning into cancer. Patients with Barrett's esophagus will take Vitamin D weekly (50,000 IU) for up for four months, and their levels of 15-PGDH will be measured. Source: EurekAlert! 10/19/10

Clinical Trial to Test Early Detection Technique for Eye Disease in Premature Infants

A new clinical trial at the Children's Hospital at Oklahoma University Medical Center will focus on ways to catch a debilitating eye disease before it's too late. The study will look at a disease known as retinopathy of prematurity (ROP), which can affect infants born before 32 weeks of gestation. If severe disease is not detected and treated within a few days, the retinopathy can lead to blindness. "The timing is critical because the window we have to treat the disease is fairly tight. If you miss that window, you may miss your chance to prevent patients from losing their sight," said R. Michael Siatkowski, MD, a leading researcher in the clinical trial and an ophthalmologist at Dean McGee Eye Institute. "This study will evaluate a new technology aimed at preventing bad outcomes for these children, and can also save $1 million per child, the cost to society for lifetime support of a blind child." One of the largest barriers to detecting retinopathy in infants is the lack of trained pediatric ophthalmologists outside of large medical centers. The problem is especially acute in rural areas and third-world countries. During the clinical trial, nurses in the Neonatal Intensive Care Unit will learn how to perform a test to detect ROP. The study will compare the effectiveness and results from the technique administered by nurses to the standard exams by ophthalmologists. If researchers find both methods to be similar, it is hoped that by training nurses they will catch more disease in more infants around the state and globally. The ROP research and clinical trial are funded by grants from the National Institutes of Health and the Inoveon Corp. Source: EurekAlert! 10/15/10

Robot to Have First Clinical Trial Test in Prostate Cancer Patients

In the first-of-a-kind clinical trial, a robot will be used to place therapeutic radioactive seeds in prostate cancer patients. The National Cancer Institute-supported study, which will enroll 14 patients, has just opened at Thomas Jefferson University Hospital in Philadelphia, Pa. The robot has been designed by university scientists to provide the steadiest and most precise method possible to implant scores of the seeds directly at the site of a cancerous tumor in the prostate gland, eliminating the possibility of human error, says Adam Dicker, MD, PhD, professor and chairman of the Department of Radiation Oncology. Prostate brachytherapy, which requires accurate insertion of some 60 to 120 radioactive seeds in very specific places in the prostate, involves a high degree of clinical skill and attention to detail. When performed with good quality, brachytherapy offers excellent cure rates compared to surgery, external radiation, and proton therapy. However, poorly placed seeds may lead to urinary and rectal toxicities. Currently, physicians use a plastic or metal template with holes in which to insert 15 to 20 needles that contain radioactive seeds into the prostate gland, but because this grid is thin, it is difficult for a person guiding it to push it smoothly and straightly through glandular tissue. Yan Yu, PhD, professor and director of the Medical Physics Division in the Department of Radiation Oncology, led a team of medical physicists, engineers, radiation oncologists, radiologists, and urologists who spent seven years developing the robotic system, which is called EUCLIDIAN. It incorporates high resolution ultrasound image processing, dose planning using genetic algorithms, 3D visualization, smart needle rotation for reducing tissue deformation and prostate displacement, and force feedback from nano-sensors installed at various points on the robot. Needle insertion and seed delivery are fully automatic. Dicker is the principal investigator of the EUCLIDIAN trial, which is only available at Thomas Jefferson University Hospital. Source: Newswise 10/14/10

Cancer Vaccine May Help Prevent Malignant Brain Tumors from Recurring

Mary Lee’s primary care physician is also a family friend. When he walked into the exam room in October 2006 with tears in his eyes, she knew something serious was wrong, but she was shocked to learn she had a brain tumor--and later found out it was the most aggressive malignant type, a glioblastoma multiforme. The doctor faxed a note to neurosurgeon Keith L. Black, MD, chairman of the Department of Neurosurgery at Cedars-Sinai Medical Center. In less than two weeks, Black and neurosurgeon Ray Chu, MD, performed surgery to remove the golf ball-size tumor, and at the beginning of 2007, Lee, a San Dimas, Calif. resident, underwent a course of radiation therapy and chemotherapy. About a year after the tumor was removed, Lee enrolled in a clinical trial of an experimental vaccine developed at Cedars-Sinai that is designed to help prevent malignant brain tumors from recurring. It is one of several approaches being studied by research scientists at Cedars-Sinai’s Maxine Dunitz Neurosurgical Institute. Glioblastoma can make itself invisible to the immune system and it can turn on genes that render most chemotherapy useless. Cedars-Sinai’s brain tumor immunotherapy centers on dendritic cells, specialized antigen-presenting cells. These cells clear debris when other cells die, and when they encounter foreign cells (antigens) they present the proteins to killer cells called T lymphocytes. These specialized white blood cells swarm to the newly identified invaders and attack. Each vaccine provides an individualized, patient-specific, and tumor-specific therapy. When the patient undergoes tumor-removal surgery or a needle biopsy, specimens of tumor cells are saved for this purpose. Meanwhile, immature white blood cells called monocytes are filtered from the patient’s bloodstream to be developed into dendritic cells. When these millions of dendritic cells are later cultured in the laboratory with proteins from the tumor cells, they become alerted to recognize them as invaders. The newly “educated” dendritic cells are then injected under the skin of the armpit, an area rich with lymph nodes. This is usually done three times over six weeks. Once in the lymphatic system, the dendritic cells are expected to recruit millions of T lymphocytes to attack any residual brain tumor cells they encounter. The dendritic cell vaccine was first used as an experimental patient treatment in May 1998 and has been studied alone and in combination with other therapies. In a Phase I clinical trial evaluating the newest version of the vaccine--called ICT-107--the median progression-free survival time in 16 newly diagnosed patients is 17.7 months, or nearly 11 months longer than the historical progression-free survival time of 6.9 months. ICT-107 is being developed by ImmunoCellular Therapeutics, Ltd., a biotechnology company. Black is chairman of the scientific advisory board, and John S. Yu, MD, director of surgical neuro-oncology in the Department of Neurosurgery at Cedars-Sinai, serves as chief scientific officer and chairman of the board. Source: Newswise 10/13/10

Vaccine Extends Survival for Patients with Deadly Brain Cancers

A new vaccine added to standard therapy appears to offer a survival advantage for patients suffering from glioblastoma (GBM), the most deadly form of brain cancer, according to a study from researchers at Duke University Medical Center and the University of Texas MD Anderson Cancer Center. The vaccine also knocks out a troublesome growth factor that characterizes the most aggressive form of the disease. "About a third of all glioblastomas are fueled by a very aggressive cancer gene, called EGFRvIII; these tumors are the 'worst of the worst,'" said John Sampson, MD, PhD, the Robert H. and Gloria Wilkins Professor of Neurosurgery at Duke. "Our study showed that the vaccine eliminated all of the cancer cells carrying this marker in all but one of our study participants," said Darell D. Bigner, MD, PhD, director of the Preston Robert Tisch Brain Tumor Center and the senior author of the study. The study, appearing in the Journal of Clinical Oncology, involved 18 patients newly diagnosed with GBM from Duke and MD Anderson and a matched set of 17 patients who served as controls. Patients in both groups received surgery, radiation, and the chemotherapy drug temozolomide. Patients in the vaccine group began receiving injections one month after completing radiation and stayed on the vaccine as long as it appeared to be working. Adding the vaccine to standard therapy extended median survival time from an expected 15 months to 26 months. Patients in the vaccine group also experienced a much longer progression-free survival period, 14.2 months, compared to 6.3 months for those who did not receive the vaccine. Funding for the work came from the National Institutes of Health, the American Brain Tumor Association, Accelerate Brain Cancer Cure, the Brain Tumor Society, the Commonwealth Cancer Foundation, the Adam Singer Foundation, the Dr. Marnie Rose Foundation, and Golfers Against Cancer. Source: EurekAlert! 10/4/10 

Family Therapy for Anorexia Twice as Effective as Individual Therapy

Family-based therapy, in which parents of adolescents with anorexia nervosa are enlisted to interrupt their children's disordered behaviors, is twice as effective as individual psychotherapy at producing full remission of the disease, new research from the Stanford University School of Medicine, Lucile Packard Children's Hospital, and the University of Chicago shows. The study is the first head-to-head comparison of these two common treatment approaches for adolescents suffering from the eating disorder. "This research was desperately needed," said James Lock, MD, PhD, one of the study's two lead authors and a professor of psychiatry and behavioral sciences at Stanford. "Anorexia nervosa is a life-threatening illness, and it's really remarkable how little information we have about how to treat it. There are serious cons to not knowing what to do." The research was published October 4 in the Archives of General Psychiatry. Lock's team at Stanford collaborated with researchers at the University of Chicago to test family-based therapy against individual psychotherapy therapy in 121 male and female anorexia patients aged 12 to 18. In family-based therapy, the clinician trains the patient's parents to help ensure that their child eats enough and does not overexercise. Individual psychotherapy, in contrast, focuses on resolving the patient's underlying anxiety and emotional problems, with only minimal involvement from the family. In order to control for differences between clinicians, all therapists in the study had patients in both treatment groups. The researchers evaluated each patient's condition at the start and end of the one-year treatment period, and then again six and 12 months after treatment ended. Patients were considered in full remission if they reached 95 percent of normal body weight and had a normal score on a standardized psychiatric assessment of attitudes about eating. At the end of the study, 49.3 percent of family-based therapy patients were in full remission, whereas 23.2 percent of individual psychotherapy patients were in full remission. The two treatments were equally effective in helping patients achieve partial remission, characterized by reaching a body weight of 85 percent of normal. "For the first time, we can confidently present parents with a treatment we consider the gold standard for this patient population," added Daniel Le Grange, PhD, the other lead author of the study and a professor of psychiatry and behavioral neuroscience at the University of Chicago. Lock noted, however, that individual psychotherapy works better in some cases, and that he and his colleagues at Packard Children's routinely offer both types of therapy. The scientists are now further analyzing the data to see if they can figure out how to identify which types of patients should be directed toward each therapy. The research was funded by grants from the National Institutes of Health. Source: EurekAlert! 10/4/10 

Researchers Find No Difference in Drugs for Macular Degeneration

Researchers from Boston University School of Medicine and the VA Boston Healthcare System have conducted a study that failed to show a difference in efficacy between bevacizumab (Avastin) and ranibizumab (Lucentis) for the treatment of age-related macular degeneration (AMD). The study, which appears currently online in Eye, is believed to be the first to describe one-year outcomes of a prospective, double-masked, randomized clinical trial directly comparing bevacizumab to ranibizuamab. Last October, these same researchers published early, six-month outcomes of the same study, which also failed to show a difference in efficacy between these two drugs for treating AMD. In this study, patients were enrolled by a 2:1 ratio to receive either the Avastin or Lucentis. Patients were given eye injections of Avastin or Lucentis every month for the first three months, followed by monthly examination and testing. They received further injections on an as needed basis for one year. Fifteen patients received Avastin and seven patients received Lucentis. There was no significant difference in visual acuity and anatomic outcomes between the two groups. Both groups had an average improvement in vision of 1.5 lines on the vision testing chart, and only one patient (who was in the Lucentis group) lost a significant amount of vision (three lines or more). In addition, patients in the Avastin group underwent an average of eight injections over one year, while patients in the Lucentis group underwent an average of four injections. "With the exception that total injections given to subjects over one year were significantly different between the two treatment arms, visual and anatomic outcomes at one year failed to show a significant difference between both groups," said lead author and principal investigator Manju Subramanian, MD, an assistant professor in ophthalmology at Boston University. According to the authors, further studies with larger sample sizes are warranted. This study is the result of work supported with resources and the use of facilities at the Veterans Affairs Boston Healthcare System, Jamaica Plain, Mass. The VA Boston funded the cost of medications for this study. Source: EurekAlert! 10/1/10 

New TB Vaccine Enters Clinical Testing

At a recent international gathering of tuberculosis (TB) vaccine researchers in Tallinn, Estonia, the Aeras Global TB Vaccine Foundation announced it will initiate a clinical trial of an investigational live recombinant TB vaccine to be led by researchers at Saint Louis University in St. Louis, Mo. The announcement was made at the Second Global Forum on TB Vaccine Development. Building on more than a decade of global scientific research, Aeras scientists have engineered a new investigational vaccine, called AERAS-422, which will undergo clinical trials to evaluate its properties for interrupting TB at all stages of infection, including initial infection, latency, and reactivation. AERAS-422 is a modernized version of the currently used TB vaccine known as Bacille Calmette Guérin (BCG). BCG is widely viewed as insufficient in preventing pulmonary TB, and this trial is part of a wider global effort to develop safer and more immunogenic TB vaccines that would be effective against all forms of TB. The Phase I clinical trial to test the safety of AERAS-422 will be led by Daniel Hoft, MD, PhD, at Saint Louis University's Center for Vaccine Development. The trial will enroll healthy adults who have never received a vaccination against TB. In addition to AERAS-422, developed as a "prime," Aeras is supporting the clinical development of four TB vaccine candidates designed as "boosters" in the prime-boost vaccine scenario. Two have reached the Phase IIb proof of concept stage. Source: EurekAlert! 9/23/10

New Treatment for Rabies Advances After Successful Phase I Trial in India

With the potential to save tens of thousands of lives each year, a new cost-effective rabies therapy developed by MassBiologics at the University of Massachusetts and the Serum Institute of India has taken an important step forward with positive results from a Phase I study. The recently completed study showed that a new monoclonal antibody (RAB-1) resulted in protective antibody levels in the serum of treated subjects equal to the current standard of treatment, which is often not available in the areas of the world hit hardest by rabies. Details of the study were reported on September 14 at the American Society for Microbiology's 50th annual Interscience Conference on Antimicrobial Agents and Chemotherapy meeting in Boston, Mass. by researchers from MassBiologics; the Serum Institute of India in Pune, India; and King Edward Memorial Hospital in Mumbai, India. In the trial run at the hospital in India, 74 healthy volunteers were randomized into several groups that either received escalating doses of RAB-1 or of human rabies immune globulin (HRIG) combined with vaccine. Blood samples were then analyzed and showed the volunteers who received RAB-1 and vaccine at a dose of 0.150 mg/kg had levels of rabies antibodies equal to or higher than the levels from those volunteers who had received the standard does of HRIG and vaccine. The half life of RAB-1 was 18 to 19 days. Blood samples were also analyzed by the Kansas State Veterinary Diagnostic Laboratory to determine if antibodies present in the volunteers' bloodstream could neutralize rabies virus in a cell-based assay using two different strains of virus. That data showed that volunteers who received RAB-1 at 0.150 mg/kg with vaccine had similar or better protective serum levels when compared to those who received HRIG with vaccine. The Serum Institute of India and MassBiologics are moving ahead in a clinical trial in India to evaluate the efficacy of RAB-1 combined with vaccine compared to the standard of care for patients who have been exposed to potentially rabid animals. Source: EurekAlert! 9/14/10

First U.S. Trial of Bone Marrow Stem Cells for Heart Attack Patients Proves Safe

The first randomized, placebo-controlled U.S. clinical trial to assess the use of bone marrow-derived mononuclear cells in patients after a ST-elevation myocardial infarction (STEMI; severe heart attack) demonstrated a strong safety profile for this cell therapy, based on Phase I results published in the September issue of the American Heart Journal. "The use of adult stem cells, derived from the patient's own bone marrow, presents a potential new type of therapy to benefit individuals after they suffer a heart attack," says the study's principal investigator Jay H. Traverse, MD, cardiologist at the Minneapolis Heart Institute® at Abbott Northwestern Hospital in Minneapolis, Minn. "Also, these types of stem cells do not possess any of the ethical concerns of embryonic stem cell research." In this single-center trial, the researchers enrolled 40 patients with STEMI, randomizing them in a 3:1 ratio to 100 million autologous cells versus placebo, administered three to 10 days following successful primary angioplasty and stenting of the left anterior descending coronary artery. Importantly, the researchers elected to deliver cells by an intracoronary infusion, as opposed to the stop-flow technique that had been used in all preceding trials, and all patients received an identical number of cells. While the cell therapy was associated with a significant improvement in left ventricular ejection fraction at six months (49 percent to 55.2 percent), the study failed to demonstrate that the cell therapy was superior to placebo because of the similar improvement in the small placebo group (48.6 percent to 57 percent). However, the cell group experienced a significant improvement in left ventricular volumes at six months compared to the placebo group. Traverse and his colleagues at the institute are also involved with the TIME trial, sponsored by the National Heart, Lung, and Blood Institute's Cardiovascular Cell Therapy Research Network, which is randomizing patients to stem cell therapy at three or seven days post-STEMI to determine the most appropriate timing to administer the stem cells following a heart attack. Source: EurekAlert! 9/14/10

Medical Center Leads Study of First U.S. Portable Driver for Powering the Total Artificial Heart

The Virginia Commonwealth University (VCU) Medical Center is the lead institution in a national clinical trial of technology that will allow artificial heart patients to recuperate, rehabilitate, and wait in the comfort of their own homes until a donor heart becomes available for transplant. The VCU Pauley Heart Center is one of up to 30 centers that will investigate a portable, mechanical driver that can power patients’ artificial hearts and enable them to recover outside the hospital environment, including at home and at step-down facilities. The medical center received institutional review board approval in early September to participate in an Investigational Device Exemption clinical study of the Freedom driver, the first-ever U.S. portable driver designed to power SynCardia’s Total Artificial Heart both inside and outside the hospital. The clinical trial is approved to enroll 60 patients. Source: Newswise 9/9/10

Promising Treatment for Metastatic Melanoma "Fast Tracked" by FDA

Researchers from the John Theurer Cancer Center at Hackensack University Medical Center in New Jersey played an important role in a study that led to the Food and Drug Administration's (FDA's) recent fast tracking of ipilimumab, a promising treatment for metastatic melanoma. The FDA based its decision largely on the results of a pivotal study published in the New England Journal of Medicine on August 19--the same day the agency accepted Bristol-Myers Squibb's application for the drug's approval and granted the application priority review status. Ipilimumab is the first drug shown in randomized, placebo-controlled trials to improve survival in stage IV melanoma. In this Phase III study, 676 patients participated at 125 cancer centers. Those who received ipilimumab, both by itself and with the cancer vaccine gp 100, lived a median of about 10 months, while those who received only gp 100 lived about 6.4 months. After two years, approximately 23 percent of those who got ipilimumab were alive, while 14 percent of those who did not receive this drug survived. Ten to 15 percent of those who received ipilimumab suffered attacks on their bodies' immune systems (autoimmune reactions), and seven of the 540 patients who got this drug died from these attacks. Most adverse events suffered by study participants, however, were reversible with treatment. The FDA grants priority review status to drugs that offer major advances in treatment, or that provide treatment where no adequate therapy exists. The projected FDA action date for the ipilimumab application is December 25, 2010. Source: EurekAlert! 9/9/10

Five Cancer Researchers Named Outstanding Young Investigators

The Southwest Oncology Group (SWOG), one of the nation's largest cancer clinical trials networks, has selected five talented researchers who are early in their careers for its 2010 Young Investigator Training Course. These five attended a three-day workshop in September in Seattle, Wash., for intensive training in how to design and conduct cancer clinical trials. To date, this nationally acclaimed program has provided intensive mentorship and career support to 56 investigators. With detailed instruction in protocol development, trial management, and statistical analysis, the course is designed to build a cohort of trained clinical trial researchers with a thorough understanding of SWOG procedures and the ability to efficiently plan and execute high-priority studies, which in some cases might enroll thousands of patients at hundreds of treatment sites. Each investigator presents a research proposal as part of the application process, and those proposals are closely reviewed and refined during the workshop. Many of the proposals advanced in previous workshops have since been launched as studies with National Cancer Institute funding. Costs of the Young Investigator program are paid for with a gift from the Hope Foundation, a philanthropic arm of SWOG that raises funds for educational and research efforts. The new investigators are Sikander Ailawadhi, assistant professor of medicine, University of Southern California; Heather Greenlee, ND, PhD, assistant professor of epidemiology and medical oncology, Columbia University; Kevin Kalinsky, MD, assistant professor of medicine, Columbia University; Tina (Tianhong) Li, MD, PhD, assistant professor of medicine, University of California Davis Cancer Center; and Margarett Shnorhavorian, MD, MPH, assistant professor of urology, University of Washington. Source: EurekAlert! 9/8/10

NIH Expands Network Focused on How Genes Affect Drug Responses

The National Institutes of Health (NIH) plans to spend $161.3 million over the next five years to expand the Pharmacogenomics Research Network, a nationwide collaborative of scientists focused on understanding how genes affect a person's response to medicines. Spearheaded by the NIH's National Institute of General Medical Sciences and launched in 2000, the network has already identified gene variants linked to responses to medicines for different cancers, heart disease, asthma, nicotine addiction, and other conditions. The expanded network will continue research in these areas and move into new ones, including rheumatoid arthritis and bipolar disorder. Network scientists will also develop novel research methods and study the use of pharmacogenetics in rural and underserved populations. The new awards include 14 scientific research projects and seven network resources. Source: EurekAlert! 9/7/10

First Clinical Trials Successfully Completed on Potent New Hepatitis C Drug

The first clinical trials on a new investigational drug being developed to treat infections caused by hepatitis C virus have been successfully completed. Completion of Phase Ia of trials in healthy volunteers with INX-189, discovered and first prepared by researchers at Cardiff University's Welsh School of Pharmacy in 2008, means the chances of it becoming an approved medicine have significantly improved. Professor Chris McGuigan of the Welsh School of Pharmacy, academic lead on the project, said: "This is still a very early stage of the trials process, but nonetheless a significant development. ...We believe that INX-189 offers the possibility of more potency against hepatitis, more rapid action in the liver, and fewer side effects than existing treatments." U.S. pharmaceutical company Inhibitex, which owns the licence to INX-189 and has been working with the Cardiff team, has announced it is looking forward to a Phase Ib trial, which would evaluate the compound's effectiveness in hepatitis C patients. Source: EurekAlert! 9/3/10

Team Discovers New Type of Antimalarial Compound

An international team led by scientists from the Scripps Research Institute, the Swiss Tropical Institute, the Genomics Institute of the Novartis Research Foundation, and the Novartis Institute for Tropical Diseases has discovered a promising new drug candidate that represents a new class of drug to treat malaria. Clinical trials for the compound are planned for later this year. The research was published on September 3 in Science. While some 40 percent of the world's population lives in malaria-infected areas, little economic incentive for pharmaceutical companies to develop new treatments exists, since malaria-infected areas correspond with the some of the world's most impoverished nations. To help surmount this barrier, concerned individuals have formed public-private partnerships to help spur research on much-needed treatments. The current study is the result of one such partnership. In addition to in-kind contributions by the pharmaceutical company Novartis (including its decade-old Novartis Malaria Initiatives) and the scientific expertise of scientists in academic laboratories around the world, the research was made possible by the support of the nonprofit organizations Medicines for Malaria Venture, the Wellcome Trust, and the W. M. Keck Foundation, as well as funding from government agencies in the United States (the National Institutes of Health's National Institute of Allergy and Infectious Diseases) and Singapore (the Agency for Science, Technology, and Research). Source: EurekAlert! 9/2/10