JDRF Research In the News June 2013 JDRF is the only global organization with a strategic plan to deliver a continuous flow of life-changing therapies and, ultimately, a cure for T1D. Past JDRF investments have yielded significant advances and taught us that progress towards our goals will be made incrementally over time. These advances and breakthroughs are what move scientific discoveries from the laboratory to clinical trials and ultimately into the hands of patients. As the leader in global T1D research and development, JDRF-supported T1D research progress is routinely reported in the media. Here is a summary of JDRF-supported T1D research progress reported by the media in June 2013. JDRF Research Portfolio Patient Venture Philanthropies: Catalyzing the Development and Delivery of Therapeutic Breakthroughs How are patient-focused organizations making tangible advances in creating life-saving and life-enhancing therapeutic innovations? The experiences of at least two foundations show that collaboration with stakeholders across industries – private, government, academia, insurers and clinicians – is a good place to start. One example of success is JDRF, the only global organization with a strategic plan to systematically eradicate the effects of T1D from people’s lives. “The challenge is bigger than we anticipated,” according to Jeffrey Brewer, President & CEO of JDRF. “We remain committed to a cure, but are also focused on helping patients live safely and well until a cure comes, through preventive and treatment strategies.” Brewer says that JDRF has started working more with industry, noting that historically, the organization focused on academic-based research. “JDRF is willing to take on more risk at an earlier stage than pharmaceutical companies,” adds Dr. Richard A. Insel, M.D., Chief Scientific Officer, JDRF. “We also are not driven by profit margins or market size. We act as a sort of virtual pharma or biotech company in the early stages of research, and then our industry partners step in to bring therapies and devices to market.” JDRF Beta Cell Encapsulation Research Hurdle Potentially Cleared to Diabetes Transplants JDRF-funded researchers at Washington University School of Medicine in St. Louis have identified a way to trigger the reproduction in the laboratory of clusters of human cells that make insulin, potentially removing a significant obstacle to transplanting the cells as a treatment for patients with T1D. Efforts to make this treatment possible have been limited by a dearth of insulin-producing beta cells that can be removed from donors after death, and by the stubborn refusal of human beta cells to proliferate in the laboratory after harvesting. The new technique uses a cell conditioning solution originally developed to trigger reproduction of cells from the lining of the intestine. Such cells could also be a source of insulinproducing cells for use in encapsulation products that would be implanted in individuals with T1D after encapsulated by a protective barrier. JDRF Beta Cell Restoration Research Stem Cell Therapy Cures T1D in Mice Using an immune-suppressing medication and adult stem cells from healthy donors, JDRF-supported researchers say they were able to cure T1D in mice. The researchers expected that the adult stem cells would turn into functioning beta cells (cells that produce insulin). Instead, the stem cells turned into endothelial cells that generated the development of new blood vessels to supply existing beta cells with the nourishment they needed to regenerate and thrive. "This is a theme we've seen a few times recently. Beta cells are plastic and can respond and expand when the environment is right," said Dr. Andrew Rakeman, a senior scientist in beta cell regeneration at JDRF. "But, there's some work still to be done. How do we get from this biological mechanism to a more conventional therapy?" Translating these findings into potential therapies is the important next step. 1 JDRF Immune Therapy Research Newly Identified Immune Protein Could Stop Diabetes An immune protein that has the potential to stop or reverse the development of T1D in its early stages, before insulinproducing cells have been destroyed, has been identified by JDRF-supported researchers in Melbourne, Australia. The discovery has wider impact, as the protein is responsible for protecting the body against excessive immune responses, and could be used to treat, or even prevent, other immune disorders such as multiple sclerosis and rheumatoid arthritis. Professor Len Harrison, Dr Esther Bandala-Sanchez and Dr Yuxia Zhang led the research team from the Walter and Eliza Hall Institute's Molecular Medicine division that identified the immune protein CD52 as responsible for suppressing the immune response, and its potential for protecting against autoimmune diseases. Clinical Trial Underway for Potential T1D Treatment Human trials have begun on a new T1D treatment that could improve the lives of future sufferers of the disease. Developed by JDRF-funded scientists at the University of Cambridge, the treatment could reduce a patient's insulin injections from several per day to potentially just a few times a week. The immune-targeted treatment slows the damage to the pancreas seen in T1D, meaning that newly diagnosed patients, in the future, may be able to retain more of their ability to produce insulin naturally requiring fewer injections. Vaccine May Stop Immune Attack in T1D Although an initial trial of the vaccine wasn't able to free anyone from their daily insulin injections, it did boost insulin production, which could help prevent some of the most devastating complications of T1D by improving blood glucose control. A new type of vaccine may stop the autoimmune attack that occurs in people with T1D, researchers report. Dr. Richard Insel, chief scientific officer at JDRF, said, "The encouraging results from this initial trial in established T1D not only demonstrated safety, evidence during the vaccine dosing period showed preservation of beta cell function, a decrease in detectable immune cells (that likely attack the beta cells), and a relationship between the two." He added that further clinical trials will be needed to figure out the optimal dose for vaccine efficacy and safety. JDRF provided funding for the early stage development of the vaccine. JDRF Artificial Pancreas Project Research JDRF Extends T1D Collaboration with BD to Develop Combined Infusion and Monitoring Products JDRF and BD are accelerating the development of new products that combine BD's proprietary insulin infusion and glucose sensing technologies through a new collaboration. Many patients with T1D not only take daily insulin via an insulin pump -requiring a catheter to be placed in the body for a number of days -- but are also checking their glucose levels via a continuous glucose monitor (CGM), which also requires a catheter-like sensor to be placed in the body for a number of days. While BD has been working to improve the products used for insulin infusion and CGM, the new collaboration with JDRF will enable additional research to develop a single optimized device that will perform both functions and eliminate the need and complexities of multiple in-dwelling catheters. The ability to perform these discrete metabolic actions automatically and accurately, with little patient intervention will be a key component of future artificial pancreas systems. For more information, please see JDRF’s Press Release. JDRF Announces Two Partnerships to Develop Stable, Pumpable Glucagon Xeris Pharmaceuticals, Inc., and LATITUDE Pharmaceuticals, Inc., will develop two different formulations of soluble glucagon for use in infusion pumps, and better determine their impact in regulating blood-glucose levels in people with T1D. JDRF announced partnerships with both Xeris Pharmaceuticals, Inc., and LATITUDE Pharmaceuticals, Inc., to support the development soluble glucagon formulations—an important step toward the advancement of future generation, fully automated and multi-hormonal artificial pancreas systems for people with T1D. Such systems are anticipated to help individuals with T1D better manage their blood glucose levels and relieve a significant portion of the burden of living with the disease. For more information, please see JDRF’s Press Release. 2 Study Gains Put Diabetics Nearer to Artificial Pancreas A decade of work by scientists to create an artificial pancreas system that will independently control insulin levels in people with T1D took a leap forward in research from Medtronic Inc. and Johnson & Johnson. Medical pumps developed by the two companies were shown in separate studies at a scientific meeting in Chicago to successfully sense changes in blood sugar levels and adjust insulin delivery accordingly. JDRF is supporting the program reported on by J&J. Smart Phone Can Track Blood Sugar The first wearable artificial pancreas system platform based on a smart phone has passed early feasibility tests in real-world settings, JDRF-supported researchers reported recently. "The investigational automated system worked correctly 98% of the total possible time from admission to discharge, which exceeded the initially set primary endpoint goal of 80%," said Boris Kovatchev, PhD, of the University of Virginia in Charlottesville, and colleagues. This is another sign of the accelerating progress being made on artificial pancreas systems, one of the key research priorities for JDRF. JDRF Prevention Research Multiple Islet Autoantibodies In Children Indicate Risk of Developing T1D A decade-long JDRF-funded study led by the Institute of Diabetes Research in Helmholtz Zentrum Munchen, Germany, is providing a deeper understanding of the link between autoantibodies and the risk of developing T1D, highlighting the importance of pre-diabetes research into possible preventions for the disease. The study was recently published in The Journal of the American Medical Association. These results heighten the need for research to find ways to prevent the disease in those with multiple antibodies who are at the greatest risk of developing T1D and becoming insulin dependent. For more information, please see JDRF’s Press Release. 3