As one financial year comes to a close, JDRF is full steam ahead on work to fund the next stage of type 1 diabetes (T1D) research. At the global internal research strategy meeting this month, 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. JDRF Australia’s own Dr Dorota Pawlak, Head of Research Development and Director of the T1DCRN, attended.
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 ways to cure, treat and prevent 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:
We now know that children with two or more autoantibodies — the markers of the disease — have a nearly 100 percent risk of developing T1D. This means diagnosis is a question of not if, but when. 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. Pilot screening studies have greatly reduced DKA. Universal screening would also allow people with autoantibodies to go onto clinical trials looking to delay the onset of T1D.
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 crucial to 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 financial year, we will focus on clinical testing of repositioned drugs— which are drugs already available and approved for other diseases—that have the potential to improve beta cell survival. We’ll also 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 surrounds the beta cells to protect them from immune attack, while letting insulin out and oxygen and other nutrients in. As we explore new ways to take these therapies to the next level, we’re considering additional technologies and gene therapy as possible investments.
Just imagine if you could mix and match features of an insulin pump, CGM and algorithm together into a safe and effective closed loop system. It’s not that far away. The T1DCRN-funded hybrid closed loop trial is getting us there, and globally, studies to improve this technology are accelerating this field forward.
A key question we ask at JDRF is how we can improve ‘non-device-based’ therapies, like insulin and other drugs, that will restore glucose control and reduce complications. Strategic discussion focused particularly on building on the successes of potential therapies used together with insulin, where JDRF has been leading the way. We’re collaborating across our research areas to improve health outcomes and the amount of time spent within the target blood glucose level range for people with T1D.
Despite advances in treatments, it is a huge burden to meet glycaemic targets. Even people with optimal control of their T1D may develop complications, and often the damage can occur before any symptoms and signs. This can make identifying the right people at the right time for treatment or clinical trials somewhat of a challenge. 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 led 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 financial year, there’s much more work to be done, and we’re looking forward to sharing our progress. Stay tuned!