The Special Diabetes Program

Transcription

1 The Special Diabetes Program Advancing Research & Improving Lives on the Path to a Cure Charlie was diagnosed with T1D at 10 months old. Some days, he wants to be a doctor when he grows up. Other days, he wants to be an artist, an engineer, a truck driver or a fireman. More than anything, he wants a cure.

2 The Burden of Diabetes Type 1 diabetes (T1D) is a devastating autoimmune disease for which there is no cure. T1D occurs when the body s immune system destroys insulin-producing cells in the pancreas. Unrelated to diet or lifestyle, T1D causes lifelong dependence on injected insulin. Type 2 diabetes (T2D) is not an autoimmune disease. With T2D, the body produces insulin but cannot use it effectively. While T1D and T2D are different, the resulting costly and burdensome complications are the same. $245 Billion Annual cost of diabetes to the U.S. economy in x Health costs of diabetes are predicted to nearly triple in the next 25 years 32% Percent of Medicare budget spent on people with diabetes 1 in 3 Number of adults in the U.S. who could have diabetes in 2050 if current trends continue 23% Increase in the prevalence of T1D in people under age 20 between #1 Diabetes is the leading cause of kidney disease, blindness in working age adults, and amputations unrelated to accidents. But there is hope People with diabetes are living longer, healthier lives with fewer complications because of research advances, including research supported by the Special Diabetes Program. We have a way to go to cure, treat, and prevent diabetes but continued funding of the Special Diabetes Program will help us get there sooner.

3 The Special Diabetes Program Strong, Bipartisan Support for the SDP The Special Diabetes Program (SDP) has led to groundbreaking discoveries and new treatments that are improving the lives of people with diabetes and demonstrating a strong return on the federal investment. SDP-funded research opportunities that are being explored and others on the horizon show great promise in relieving the growing burden on our nation s economy, the millions of Americans affected by diabetes, and other diseases that benefit from the SDP s work. Congress and the Administration created the SDP in 1997 to address the growing burden of type 1 diabetes (T1D) by providing more funding for T1D research. At the same time, Congress created a sister program, the Special Diabetes Program for Indians, focused on type 2 diabetes (T2D) treatment and prevention in American Indian and Alaska Native populations. Congress renews these programs together. Since the enactment of the SDP, the program has enjoyed strong, bipartisan support in Congress and the Administration. Recent examples include: Stand-alone legislation in 2010 (H.R & S. 3058) was cosponsored by 62 Senators and 296 Representatives. A two-year renewal at $150 million, for both parts of the program, was included as part of the Medicare and Medicaid Extenders Act of 2010 (P.L ). Letters circulated in 2012 garnered the support of 72 Senators and 272 Representatives. The SDP was renewed as part of the American Taxpayer Relief Act of 2012 (P.L ) for one year at $150 million, for each of its two parts. JDRF is grateful that the program was extended for one year. It is our hope that the program will be extended for multiple years, as Congress has done previously, to ensure critical clinical trials continue uninterrupted and resources are allocated most effectively. Diagnosed with T1D at age 4, Kerry experienced the thrill of not having to worry about her diabetes for a few days when she participated in an artificial pancreas trial. The artificial pancreas, which automatically controls blood sugar levels, has been tested in a hospital setting with great results. The next step is further testing in patients at home so the technology can one day be available to all people with T1D. The development of this technology would be the biggest breakthrough since the discovery of insulin.

4 SDP Research Advances Cure T1D By the time T1D is diagnosed, patients have already suffered an immune attack that has destroyed most of the insulinproducing cells in the pancreas. SDP research is supporting trials to halt and reverse the destruction of these cells and replace cells via transplantation. Treat T1D & Related Complications Extensive, multi-year clinical trials have demonstrated that tight blood glucose control plays a major role in preventing burdensome complications. New tools are being developed to achieve better control and new insight on complications Immune Therapies Immune therapy drugs have slowed the immune attack for approximately one year in patients newly diagnosed with T1D. Patients required less insulin and had improved blood glucose control for a period of time. Since these drugs can have serious side effects, further research will help find more targeted drugs and new protocols with the hope of halting the autoimmune attack permanently to prevent T1D and prolong the honeymoon period in new onset T1D. Replacement Transplantation of insulinproducing islet cells from cadaveric donor pancreases into patients with the most difficult to control T1D significantly reduced loss of consciousness from hypoglycemia and achieved insulin independence for over five years. Further research focused on developing alternative sources of islets could allow the approach to be extended to large numbers of individuals. Researchers could also encapsulate islets to help avoid the toxic effects of immune suppression and develop strategies to transform a patient s own cells into insulin producing beta cells. is leading to improved treatments and long-term cost savings. Artificial Pancreas Patients with T1D achieved tighter blood glucose control by using artificial pancreas (AP) technologies in clinical trials. A recent study estimates use of AP technology in working age adults who have T1D will result in nearly $1 billion in savings to Medicare over 25 years. Additional studies will test AP technologies in the outpatient setting and will focus on developing new generation systems with increased automation. Diabetic Retinopathy Treatment using a drug that reached the market last year in the United States preserves and even improves vision in people who have diabetic eye disease. This advance makes the difference between being able to see well enough to drive or hold a job or not. Further research is required to develop new therapies to treat the large population of individuals whose eye disease fails to respond to this newly approved drug. After Ryan s diagnosis in 2002 at the age of 8, his father, Scott, enrolled in a study for relatives of people with T1D. Scott tested positive for T1D antibodies and enrolled in another SDP-funded study to test a drug called rituximab (anti-cd20) to see if it would slow the disease s progression. Today, Scott has T1D but is only in need of small doses of insulin to control his diabetes and doesn t have high and low blood sugar levels like others with T1D.

5 & Future Opportunities Prevent T1D Genetic and environmental factors are Diabetic Kidney Disease Researchers have found that intensive blood glucose control over 6.5 years can cut in half the onset of impaired kidney function. Diabetes is the leading cause of end-stage renal disease (ESRD), which cost $29 billion to Medicare in By lowering ESRD rates by 50%, Medicare would save over $14 billion in 10 years and nearly $126 billion in 25 years. Further development of AP technologies will help patients achieve tighter blood glucose control. Also, other avenues need to be explored for the prevention and treatment of kidney disease, as this disease may still occur in T1D patients with reported good blood glucose control. Projected Range of Medicare Savings for T1D and T2D from Improved ESRD Rates 150B 120B 90B 60B 30B 0B $14.5 BILLION $66.5 BILLION $125.9 BILLION believed to influence if and when a person will develop T1D. SDP-supported research is working to identify and understand these components so that one day, T1D can be prevented altogether. Genetics Over 50 genes or genetic regions, up from 3 genes about a decade ago, have been identified that influence a person s risk of developing T1D. One gene region was identified with regulating blood glucose levels. Further research will help to pinpoint how genes function in T1D onset and glucose control, and enable the design of specific clinical trials to test personally-tailored interventions for patients who have similar risk profiles. In addition, further study could lead to the identification of new ways to prevent or reverse the disease. Environmental Triggers Researchers are more than midway through a 15-year study, with over 8,000 at- risk children enrolled at birth, to determine what environmental factors influence T1D onset. Information on diet, infections, and other exposures is being analyzed and is expected to make a major contribution to understanding the cause of T1D. Strategies could be developed to prevent T1D onset, ranging from a vaccine for those at risk of developing T1D to avoiding certain foods if diet plays a key role. The comprehensive collection of data from these trials will also benefit other autoimmune diseases. Nilia had no family history of type 1 diabetes (T1D), but newborn screening, as part of a Special Diabetes Program funded trial, found she is at risk for developing it. She is participating in the trial until age 15 or T1D onset to help determine if diet, illnesses, or other exposures during her childhood are environmental triggers of T1D onset. This critically important research could enable scientists to prevent T1D altogether.

6 Research Consortiums & Networks Supported by the Special Diabetes Program The renewal of the SDP will allow researchers to build on the program s advances by translating those discoveries into even better treatments and eventually a cure for people with diabetes. Without a renewal, SDP-supported trials will be disrupted or halted completely and private funders will not be able to fill the gap: T1D Genetics Consortium/Function of T1D Genes & The Environmental Determinants of Diabetes in the Young (TEDDY) 100% SDP SUPPORTED Large clinical studies that are answering the question of what causes T1D. The answers will continue to help lead the way to new and better treatments to prevent the disease. Type 1 Diabetes TrialNet 67% SDP SUPPORTED A national network conducting clinical trials of therapies to prevent T1D in people who are at risk of developing diabetes and to stop the disease from progressing in those who are newly diagnosed. Clinical Islet Transplantation Consortium 98% SDP SUPPORTED Multi-center have helped advance insulin-producing cell transplant therapy. Beta Cell Biology Consortium 75% SDP SUPPORTED An international collaboration focusing on the function of insulin-producing cells and on developing cell-based therapies to treat T1D. Diabetes Research in Children Network 67% SDP SUPPORTED A network of U.S. clinical centers testing and validating new diabetes management technologies in children. Diabetic Complications Consortium 59% SDP SUPPORTED An interdisciplinary consortium advancing the development of diabetes cures and treatments by creating animal models (60 to date) that closely mimic the human complications of diabetes. Diabetic Retinopathy Clinical Research Network 26% SDP SUPPORTED A collaborative, nationwide research network of clinicians and researchers at more than 165 sites in 43 states focused on diabetes-induced eye disorders such as diabetic retinopathy, diabetic macular edema and associated conditions. JDRF a Partner on the Path to a Cure JDRF s research mission is to discover, develop and deliver advances that cure, better treat and prevent T1D. Driven by passionate, grassroots volunteers, JDRF is the largest charitable $150 million SDP $110 million JDRF supporter of T1D research with more than 80 % of JDRF s expenditures directly supporting research and research-related education. Since its founding in 1970, JDRF has awarded more than $1.7 billion to T1D research, including $110 million in FY JDRF s research complements federally-supported research. We are closer to a cure than ever before, thanks to the combination of federal diabetes research funding and JDRF s private investment one of the world s most effective public-private partnerships focused on curing a disease. The renewal of the SDP will capitalize on these investments and help us achieve our mutual goal of better treatments and a cure for people with diabetes. Thank you for your support!