As one fiscal year closes, JDRF is full steam ahead on work to fund the next stage of type 1 diabetes (T1D) research. At our internal research strategy meeting last week, JDRF’s team of scientists met with members of the JDRF Research Committee, as well as key opinion leaders from the research community, to help us review and plan our research roadmap.
This annual meeting allows us to challenge our thinking across our research portfolio to see where we can do more, and do it better, faster, more efficiently and more effectively. We search the globe to inform our decision making. We make very critical decisions, rely on and work with our partners in the scientific community and combine all of that with the passion we have at JDRF to find a cure for T1D.
Our goal at the research meeting was focused on how we should be moving the science as quickly as possible. Some of these discussions included:
Prevention. We now know that children with two or more autoantibodies—the markers of the disease—have a nearly 100 percent risk of developing T1D—making diagnosis a question of when, not if. Universal screening will allow us to identify those at high risk and, when combined with follow-up monitoring, has the potential to prevent diabetic ketoacidosis (DKA) at diagnosis, a serious, life-threatening condition. DKA occurs in about 35 percent of new diagnoses. But pilot screening studies have greatly reduced DKA. Universal screening would also allow people with autoantibodies to go onto clinical trials to evaluate ways to delay the onset of T1D.
Immunotherapies. Targeting and ultimately restoring proper immune balance is central to JDRF’s efforts to ultimately cure T1D. The immunotherapy field is seeing exciting advances right now in cancer, other autoimmune diseases and T1D, providing a golden opportunity to take advantage of this unique convergence of approaches and breakthroughs. JDRF plans include leveraging the progress in cancer and other autoimmune diseases to advance new therapeutic approaches for T1D.
Beta Cell Regeneration. Restoring and protecting the insulin-producing beta cells is central to JDRF’s goals of curing T1D. JDRF spearheaded efforts to establish a pipeline of beta cell regenerating and beta cell survival therapies and has seen rapid progress towards translation and clinical testing. In the next fiscal year, we will focus on clinical testing of repositioned drugs—drugs approved for other diseases—that have the potential to improve beta cell survival and to continue to advance the pipeline of novel therapies for survival and regeneration of beta cells, translating scientific discoveries into therapeutic opportunities.
Beta Cell Replacement. There are some amazing advances in the science of beta cell encapsulation. Encapsulation is an advanced form of transplantation in which a material designed to keep beta cells protected from immune attack, while letting insulin out and letting in oxygen and other nutrients, is implanted in the body. As we explore new ways to take these therapies to the next level, we’re considering additional technologies and gene therapy and editing as possible investments.
Artificial Pancreas: Thanks to JDRF leadership, the first artificial pancreas system, the Medtronic 670G, gained FDA approval in 2016. But what if an insulin pump, a continuous glucose monitor (CGM) and an algorithm could “talk” to each other, regardless of whether they have the same manufacturer. Just imagine if you could mix and match components together into a safe and effective artificial pancreas system. It’s not that far away. There is already one CGM system—the Dexcom G6—that has been approved under a new FDA pathway that enables such interoperability, and JDRF is working with partners to encourage more in the future.
Glucose/Metabolic Control. A key question we ask at JDRF is how we can improve non-device based therapies, both insulin and non-insulin drugs, that will restore glucose control and reduce complications. Strategic discussion focused particularly on building on the successes of non-insulin adjunctive therapies to complement insulin action where JDRF has been leading the way. We’re collaborating across our research areas to improve outcomes and time in-range for T1D.
Complications. Despite advances in treatments, most people are not meeting glycemic targets. And even people in good control of their T1D may develop complications, and often the damage can occur before any noticeable signs. This can make it a challenge to identify the right people at the right time for treatment or for clinical trials. By identifying the markers that are associated with early-stage kidney decline or vision loss, we can change the paradigm for the currently cost-prohibitive and unwieldy drug development processes that is significantly dampening the progress in the field. Also, JDRF is strategically removing the barrier of T1D exclusion in kidney trials lead by the private sector.
T1D is an immensely complex disease and requires a deliberate and proactive approach to combat it from a variety of research disciplines. As we approach the new fiscal year, there’s much more work to be done, and we’re looking forward to sharing our progress. Stay tuned!