JDRF Research In the News July 2012 JDRF Cure Research Halting the autoimmune process and restoring normal insulin production. JDRF Immune Therapy Research Antibodies Reverse T1D in New Immunotherapy Animal Study – Science Daily T1D is an autoimmune disease in which the body's own immune T cells target and destroy insulin-producing beta cells in the pancreas. JDRF-funded scientists, led by Dr. Roland Tisch at the University of North Carolina School of Medicine have used injections of antibodies to rapidly reverse the onset of T1D in mice genetically bred to develop T1D. Within 48 hours, blood glucose levels in these mice came to normal levels and in 5 days they had a remission of diabetes. In this study, just two injections of the antibodies maintained disease remission indefinitely without harming the immune system. The findings suggest for the first time that using a short course of immunotherapy may someday be of value for reversing the onset of T1D in recently diagnosed people. The researchers are now planning to test human version antibodies. Novo Nordisk Weaves Itself Into Seattle Biotech, With Diabetes R&D - Xconomy Novo Nordisk, which spends $1.7 billion a year on diabetes R&D, has signaled its intent to broaden its commitment to T1D R&D through collaboration with JDRF. In this alliance, Novo will seek to pick up the baton on some of the more interesting projects JDRF has supported at academic groups and biotech companies. This represents a key step in translating research discoveries into new products for patients. Doug Ringler, the former CEO of Cambridge, MA-based ToleRx, a T1D drug developer, says he's impressed with the collaboration between Novo and JDRF. It's the kind of partnership that could help answer important questions that biologists want to know, like whether inhibiting cells with a marker called CD-3 might be an effective way to fight T1D. The center will run sideby-side comparisons of technologies being developed through JDRF’s sponsored projects as well as other opportunities either party identifies. A joint committee will select the most promising projects for further development. For more information, please see JDRF’s Press Release. 1 JDRF Beta Cell Replacement Research Human Stem Cell Transplants Successfully Reversed Diabetes in Mice – Medical Daily JDRF-supported scientists led by Dr. Timothy Kieffer, at the University of British Columbia in Canada, and scientists from the New Jersey-based BetaLogics, successfully reversed diabetes in mice by transplanting human stem cells into mice in a discovery that may lead to finding a cure for T1D. Researchers say that the latest study, published in the journal Diabetes, was the first to show that human stem cell transplants can successfully restore insulin production and reverse T1D in mice. In an experiment designed to mimic human clinical conditions, researchers were able to wean diabetic mice off of insulin four months after the rodents were transplanted with human pancreatic stem cells. The studies were performed in diabetic mice that lacked a properly functioning immune system that would otherwise have rejected the cells. The next step is to identify a suitable way of protecting the cells from immune attack so that the transplant can ultimately be performed in the absence of any immunosuppression. Caspase Inhibitor Enters Clinical Trial In Islet Transplantation - MarketWatch JDRF-supported researchers at the University of Alberta announced today the treatment of the first patient in an early phase islet cell transplant trial of emricasan, a pan-caspase inhibitor. This compound is being developed by Conatus Pharmaceuticals Inc. The objective of the trial is to determine its safety and the ability of treated patients to achieve and maintain insulin independence following their transplant. The study will also collect preliminary data on the efficacy of emricasan in maintaining adequate immunological protection against autoimmunity of islet cell transplant recipients. For more information, please see JDRF’s Press Release. JDRF Beta Cell Regeneration Research CureBeta Enters Strategic Alliance With Janssen Pharmaceuticals - MarketWatch The CureBeta initiative was established by Harvard, the Howard Hughes Medical Institute (HHMI), and Evotec in 2011 to leverage the assets and expertise in industry and academia to identify and develop T1D modifying therapeutic targets. Long-time, JDRF-supported investigator and recognized leader in the field of beta cell research at Harvard University helped launch this unique international collaboration. JDRF’s Chief Scientific Officer, Dr. Richard Insel, stated, "We are delighted to see Evotec, Dr. Doug Melton, and Janssen launch this collaborative partnership and commitment to develop innovative regenerative medicine therapies for T1D. JDRF has been actively supporting research in the field of beta cell regenerative therapies, including other collaborative partnering programs with Evotec and Janssen Pharmaceuticals. JDRF looks forward to supporting the CureBeta initiative." Scientists Discover Key Pathway for Development of Insulin-Producing Cells – Health News Digest JDRF-supported researcher Dr. Seung Kim at Stanford University School of Medicine has identified a molecular signaling pathway that drives the growth and maturation of young human beta cells - the insulin-producing cell type in the pancreas that malfunctions in T1D - in mice and humans. The pathway, called the Cn/NFAT pathway, has been shown to be important in the growth and development of many cell types, including immune cells and neurons. But this is the first time it's been shown to be involved in the development of human beta cells. The new findings from Kim’s team bring scientists still closer to being able to generate functional beta cells in a laboratory dish for transplant into a human patient, or to coax a diabetic’s non-functioning beta cells to begin dividing and producing insulin. 2 JDRF Treat Research Optimizing blood sugar control, reducing the burden of management, and preventing & treating complications. JDRF Artificial Pancreas Project Research Outpatient Artificial Pancreas Study Funded by JDRF – Santa Barbara Independent The Sansum Diabetes Research Institute recently conducted a groundbreaking 45-hour medical research study funded by JDRF on an artificial pancreas system (AP). This significant milestone study is the largest in the United States to date and included five individuals with T1D from the local area. For one of the first times, the patients, not the researchers, were in control of this T1D management system which runs automatically, reacting and delivering precise amounts of insulin when the body needs it, like a normal human pancreas. The clinical trial participants were able to move about freely outside the confines of the research setting for short periods of time. This research is part of a major collaborative effort funded by JDRF focused on developing artificial pancreas systems and making them broadly available. JDRF Complications Prevention and Treatment Research Panel Backs Wider Approval of Roche Eye Drug – Wall Street Journal A federal advisory panel recommended wider approval for Roche Holding AG's eye drug, Lucentis. Roche's Genentech unit is seeking approval from the U.S. Food and Drug Administration to market Lucentis to treat an eye condition called diabetic macular edema (DME), which is related to diabetes. The eye condition is the leading cause of blindness among working-age Americans. Lucentis was first approved in 2006 to treat a form of macular degeneration. The panel of medical experts unanimously voted that Lucentis, an injection, was effective at treating DME. JDRF testified at the hearing on the urgent need for new and better options for the treatment of DME. In addition, JDRF-funded researchers in the mid-1990s discovered the underlying causative mechanism of DME that became the target for development of Lucentis by Genentech. For more information, please see JDRF's Statement. Novel Therapy May Prevent Eye Damage in Diabetic Eye Diseases - Newswise JDRF-funded researcher Dr. David A. Antonetti’s team at the University of Michigan Kellogg Eye Center have identified a compound that could interrupt the chain of events that cause damage to the retina in diabetic eye disease. The finding is significant because it could lead to a novel therapy that targets two mechanisms at the root of the disease: inflammation and the weakening of the blood barrier that protects the retina. To date, treatments for diabetic retinopathy, the leading cause of blindness among working-age Americans, have been aimed largely at one of those mechanisms. 3 JDRF Prevent Research Preventing the autoimmune process from starting and stopping it before the onset of insulin dependence. Protective Hormone Potential Path To Prevent Diabetes – GP Online Magazine An anti-diabetic hormone that can stimulate insulin production and protect pancreatic cells could prevent people from developing the disease, scientists believe. JDRF-supported researcher Dr. Christopher Newgard and colleagues from Duke University Medical Centre in North Carolina believe the discovery could lead to new preventive therapies for T1D. The novel hormone is manufactured in the pancreas and researchers found it could stimulate new insulin production in rats and protect key insulin-producing cells from destruction. Because diabetes starts to take hold when the number of beta cells dwindles and insulin production drops, finding the best way to produce more of this protective hormone could be valuable. Researchers now plan to test the hormone in T1D. 4