Less Until None
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Presentation by Julia Greenstein PhD, JDRF's Vice President of Cure Therapies, at JDRF New England chapter's 2nd Annual "Living Well with T1D" Symposium on March 9, 2013.
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Less Until None
- 1. Less Until None: A Plan for a World without T1D
- 2. 2 LESS UNTIL NONE 2 JDRF’s Vision for the Future
- 3. 3 Therapies CureDelivery of Continuous Therapeutic Improvements Side-Effects Complications Patient Burden Glycemic Control Safety Normal Physiology Restored Better Safety, Fewer Side-Effects, Less Exposure to Complications
- 4. 4 JDRF Research Priorities Regenerating new beta cells Encapsulation of alternative beta cell sources Immune therapies Diagnostic, measurement and imaging tools Artificial pancreas systems Novel insulins and other blood sugar control drugs Diabetic eye disease Complications prevention Preventing the autoimmune process from ever starting Stopping and reversing the autoimmune process early to maintain insulin independence
- 5. 5 Artificial Pancreas Automated technologies to help maintain better control of blood- glucose levels Integrated smart technology to automate routine elements of insulin management FDA released final guidance on pathway for commercialization of devices
- 6. 6 Glucose Responsive Insulin (GRI) Form of insulin that activated when blood-glucose levels start to rise Simplifies life for and improves overall blood-glucose controls JDRF competition underway to accelerate progress on GRI
- 7. 7 Encapsulation Easily implantable Beta cells or islets surrounded by a protective shield/capsule Able to sense glucose levels and produce insulin as needed Hidden from the immune system to sustain normal function Eliminating the need for immunosuppressive drug therapy
- 8. 8 Regeneration Ability to create new insulin producing cells destroyed by autoimmune attack Creating abundant supply of Beta cells through stem cells and regeneration Immune therapy for suspected “triggers” including viruses
- 9. 9 Secondary Prevention Preserve beta cell function in these newly diagnosed individuals Approaches include: Reducing inflammation Boosting beta cell survival Suppressing the autoimmune attack
- 10. 10 Primary Prevention Prevent the autoimmune attack on beta cells before it starts Approaches include: Viral vaccines Beta Cell Autoantigen Vaccines
- 12. 12 Make a Difference and Get Involved! Give generously to speed JDRF research to cure, better treat and prevent T1D. Join a JDRF Walk, Ride, Gala or other fundraising event. Volunteer at the local JDRF chapter. Participate in clinical research.
- 13. 13 Thank you!
Editor's Notes
- The vision for JDRF is simple: a world without type 1 diabetes (T1D). This is a world where normal physiology is restored and no one suffers from the terrible impact of T1D and its complications, burdens, and damages. JDRF-funded research efforts are creating the outlines of this world and helping it coming into view. We know it is possible, we just can’t say exactly when. But we know we can get there faster by accelerating our fundraising to continue building momentum in T1D research. Until we get to the world without T1D, we want Less -- Less T1D until none. JDRF’s strategic plan for its research funding is focused on ensuring there are an ongoing stream of meaningful efforts underway to develop and commercialize therapies that lessen the impact of T1D -- keeping patients healthy and safe now, until a cure is achieved.
- Our past investments have yielded significant advances. But they have also taught us that there will be no single “eureka” moment that will cause T1D to vanish all at once. Instead, our progress will be a series of breakthroughs across different therapeutic approaches occurring on different timelines. Therefore the path to cures for T1D is actually continuum of therapies that lead to cures. As therapies move across the continuum towards cures, new treatments will progressively remove the activities that represent daily patient burden, side effects and complications. As we move from left to right on this continuum and deliver a flow of therapies, glycemic control is restored on the way to our ultimate goal of restoring normal physiology.JDRF is the only global organization with a strategic plan to bring a continuous flow of life-changing therapies and, ultimately, a cure for T1D.JDRF Research and Advocacy works in partnership with public, private, academic and corporate partners to expedite the discovery, development and delivery of T1D therapeutics. We are already making important advancements…
- Each year, JDRF research management takes a hard look at where we can have the biggest impact toward our mission and objectives. Like any organization, JDRF can’t do everything. So, JDRF carefully considers where we can make the biggest impact and then focuses our efforts in those particular areas.JDRF research works across three key areas: Cure, Treat, and Prevent. Within each area, JDRF has developed a strategic plan (or roadmap) that identifies the top research priorities areas within each category, seen here.
- Few people, regardless of age, can focus intensely on the unrelenting balancing act T1D demands every moment of the day. But technology can. JDRF launched the Artificial Pancreas Project seven years ago with the goal of driving the development and approval of automated technologies to help people with T1D maintain better control of their blood-glucose levels. When JDRF stepped in, there was minimal direct investment being made in using integrated smart technology to automate the most routine elements of insulin management. JDRF’s investments, leadership and collaboration have radically shifted that landscape. New technology and new interest are bringing to market faster-acting insulin, faster mechanisms for delivering insulin, better CGM sensors, and new pump designs. JDRF’s active presence in this environment has led to a renaissance in the diabetes technology world and important new opportunities to help people with T1D manage more effectively.Artificial pancreas systems will be the most revolutionary advance in diabetes care since the discovery of insulin. Like the body’s pancreas, AP systems will react to rising blood-glucose levels by combining monitoring technology with insulin pumps to provide the right amount of insulin at the right time. Not only will an AP system result in much tighter control, lowering the risk of health complications later in life, but it will also reduce the constant worry about blood-sugar levels and what must be done to manage them.JDRF is committed to advancing progress on future, enhanced AP systems with initiatives to develop faster-acting insulin and faster insulin delivery, needed to boost the overall performance and efficacy of the AP system; to improve blood-glucose sensing technology to achieve greater accuracy, ease of use, and more accurate performance; and to develop systems adding hormones, which would allow for the most precise control imaginable.
- Glucose responsive insulin (GRI) is a form of insulin that only works when the body’s blood-glucose levels start to rise. When blood sugar levels are in proper range, the insulin is inactive. GRI would profoundly and positively change the lives of people with T1D. Their need for injections to lower blood-sugar levels would largely disappear. In short, GRI would not only make life much easier and simpler for T1D patients, it would also improve overall blood-glucose controls with all the positive benefits associated with that outcome. GRI is an example of JDRF’s investment strategy to accelerate the pace of science leading to cures and treatments for people with T1D by promoting highly innovative approaches to new therapy development.A very exciting opportunity in the GRI field is “smart insulin.” In 2003, an MIT researcher named Todd Zion founded a company called SmartCells, Inc. The company developed a form of GRI named Smart Insulin. JDRF invested in the company at an early, high-risk moment to help with early development efforts and to support preclinical safety and efficacy testing. Following JDRF’s early investment, the pharmaceutical company Merck announced that it had acquired SmartCells with the intent of supporting the continued development of Smart Insulin—a validation of JDRF’s strategy to support early stage, high-risk, therapy development to establish proof of concept to draw major drug manufacturers to the T1D field. JDRF is also stimulating progress in the GRI field through its Glucose Responsive Insulin Grand Challenge Prize. As promising as “smart insulin” is, JDRF believes that it is always prudent to seed or support multiple approaches to the same goal. While still years away from reaching patients, GRI will, with continued JDRF investment, become another life-changing therapy for those with T1D.
- Encapsulated cell therapy has the potential to virtually eliminate the relentless daily management burden for those living with T1D. No need for multiple daily insulin injections or pump therapy, no more constant blood testing and no more carb counting. In short, people with T1D would be liberated to go about their daily lives for extended periods of time as if they didn’t even have the disease at all. JDRF researchers are working to develop easily implanted products composed of beta cells or islets surrounded by a protective shield, or capsule. These encapsulated beta cells sense a person’s glucose levels and produce insulin as needed, while the barrier protects them from the autoimmune attack that initially triggered the onset of T1D. Encapsulation keeps the newly implanted cells alive by hiding them from the immune system, providing a safe environment where they can function normally. The cells can constantly assess the amount of glucose in the blood and release exactly the correct amount of insulin. This therapy overcomes one of the major obstacles that has slowed progress in other beta cell transplant fields: the need for potentially toxic immunosuppressive drugs to tame the autoimmune attack.JDRF’s goal is to accelerate progress on a number of encapsulation approaches, moving them into human trials as quickly as possible to determine which are the most promising. In fact, JDRF has already supported some of the first human trials to evaluate select encapsulation strategies. For example, through our industry partnerships, JDRF is testing the ability of encapsulated pig islets to reverse severe hypoglycemic unawareness in humans. And we’re helping to advance encapsulated stem cell precursors to beta cells into human testing.
- The growing field of regenerative medicine—which JDRF has helped to shape—continues to demonstrate the body’s amazing ability to heal itself. JDRF has funded dozens of human trials to test various immune interventions, and each one has added to our understanding of what is necessary to stop the immune attack. These areas of inquiry will yield a permanent cure for T1D. Ultimate success, however, requires overcoming a huge challenge: giving the body the ability to create new insulin producing beta cells to replace those destroyed in the autoimmune attack, in an environment where that attack can never happen again. JDRF is leading the way in funding research aimed at overcoming these obstacles. First, JDRF is engaged in a range of research to create an abundant supply of new beta cells. These include stem cell research focused on reprogramming cells to become beta cells. But one of the most promising areas of research today is in beta cell regeneration. We are working to learn how the pancreas can become its own source of new beta cells. JDRF researchers have already demonstrated that the body has the ability to grow new beta cells in certain situations, such as pregnancy. Now, JDRF investment strategies are employing a diverse set of tools and advancing a number of approaches to triggering the body’s ability to do the same in those with T1D.The second area of focus is in the immune therapy space. Our rapidly advancing understanding of the immune system, gleaned in part from JDRF funded research over the last decade, has led to discovery of a number of suspected “triggers” associated with the onset of T1D including certain viruses. This, in turn, opens the door to development of new approaches that would prevent the hallmark immune system attack in T1D. This includes research into developing a therapy, similar to an allergy shot, which would train the body’s immune system not to initiate an attack on the beta cells
- Preventing people from developing T1D is the ultimate answer. Polio has never been cured, but an effective vaccine has largely eradicated this disease. That’s why JDRF’s commitment to ending T1D includes finding ways to insure that new generations of children and adults never face the threat of T1D. JDRF is pursuing both primary and secondary prevention strategies. Primary prevention means literally preventing the autoimmune attack entirely so people never develop T1D at all. Secondary prevention is focused on finding ways to prevent insulin dependence in individuals where the autoimmune attack on beta cells has begun. The number of Americans who are diagnosed with T1D each year is growing at a rate of approximately 2.6%. The goal of secondary prevention is to preserve beta cell function in these newly diagnosed individuals. Approaches include:Reducing inflammation: Because inflammation of the beta cells is now seen as one of the causes of beta cell death, strategies that alleviate this inflammation could lead to the survival of remaining beta cells.Boosting beta cell survival: There are a number of possible drug-based therapies that could help beta cells survive attack.Suppressing the autoimmune attack: The use of autoantigen vaccines and highly targeted immunotherapies that impact only the part of the immune system associated with destroying beta cells.
- The goal of primary prevention is to prevent the autoimmune attack on beta cells from starting. Approaches include:Viral Vaccines: JDRF has identified a number of suspected triggers associated with the onset of T1D, including certain viruses. The development of new vaccines that stop these triggers may be able to prevent the subsequent autommune system attack.Beta Cell Autoantigen Vaccines:Similar to allergy shots, diabetes vaccines that train the body’s immune system not to initiate an attack on the beta cells would be an effective form of primary or secondary prevention. This approach may also benefit regeneration approaches to cure established T1D.Both primary and secondary prevention approaches show considerable promise. But critical knowledge gaps remain. We need to make progress to screen for the onset of the autoimmune attack. We need to better understand how the disease progresses to allow us to develop tailored ways to intervene at various early stages to prevent insulin dependence. And we need to identify biomarkers that yield insight into risk and susceptibility and can speed up prevention clinical trials.
- No other disease focused non-profit organization brings together JDRF’s combination of skills and resources that truly enable it to make a meaningful impact in T1D research advances. That is why JDRF is uniquely positioned to create a future without T1D. Our research expertise, fueled by a dedicated internal team of 24 Ph.D. and M.D. scientists with experience in translating research in academia and industry, is driving progress toward our strategic goals . This high level of expertise allows us to effectively partner with academia, foundations, industry, governments, regulators, and insurers -- putting us at the center of a globally coordinated effort to move research forward.JDRF advocacy is a powerful mix of grassroots and national efforts to engage law makers in supporting legislation that encourages T1D research funding. A key effort for this group is the multi-year renewals of the Special Diabetes Program (SDP) . Since its inception in 1997, the SDP has provided $1.9 billion through the NIH for type 1 research, resulting in treatments and management used every day to help people live better lives with type 1. JDRF advocacy has led the way in securing this vital funding for T1D research programs. We will persevere in our efforts to let key lawmakers know the importance of continued, uninterrupted funding for this critical research program.Our advocacy power allows us to have influence -- beyond funding -- in other key areas of government and industry that impacts T1D research and product commercialization. This unique combination of skills, expertise and influence allow us to make important strides in the regulatory arena and help break down barriers to innovation and research progress in T1D. We were very successful in our efforts with the FDA and the Artificial Pancreas program and we will continue to use this model as other products and devices approach commercial viability
- Thank you for attending today’s program and for your support of JDRF! I’ll turn it back to Amy who will be sharing some housekeeping items.