Cynthia Rice, JDRF Senior Vice President for Advocacy and Policy, joined us at our Annual Mission Update on Monday, June 3rd, to give her talk entitled “Driving the Mission Forward: Research & Advocacy Progress. JDRF research is laser focused on curing T1D and mission-driven to improve lives today.

Two research areas, Beta Cell Therapies and Immunotherapies, are giving JDRF a line of sight to real breakthroughs in curing T1D. Beta cell therapies focus on rejuvenating beta cell and insulin production. Within this area, research is being done on replacement: developing a capsule containing insulin producing beta cells that can release insulin, but are protected from the autoimmune attack and regeneration: discovering and developing therapies that will enable the pancreas to produce beta cells once we stop the autoimmune attack in people currently living with T1D.

Beta Cell Replacement Research

JDRF is now actually supporting phase 1-2 safety and efficacy trials in humans of several beta cell replacement product candidates, including approaches developed by ViaCyte, Beta O2 and Sernova. But challenges remain: we need to determine the right device design to protect the cells; we need to develop improved biomaterials for the capsule so that they won’t trigger any immune rejection when the device is implanted. We also need to sustain progress in developing an inexhaustible supply of insulin producing cells for implantation and capitalize on newly emerging field of gene editing and cell engineering. We remain very optimistic about this field having overcame what seemed like daunting scientific hurdles in the early days of artificial pancreas systems. Working with our beta cell consortium of scientists from around the world, we believe beta cell replacement therapy holds tremendous possibilities for our community.

Beta Cell Regeneration Research

In the last decade, our knowledge about beta cells has been transformed, creating new pathways to a cure. The old belief was that beta cells are completely destroyed in T1D and cannot be recovered. Through research we now know that in many individuals, beta cells can persist long after T1D diagnosis and function can increase. Even after 50 years of T1D, some people have beta cells that are still producing insulin and it has been determined that pregnant woman with T1D increase their beta cell mass. These discoveries have inspired researchers to look for ways to stimulate beta cell replication, and several promising avenues are being explored.

Clinical trials show that Verapamil slows T1D progression. Verapamil, already used to treat high blood pressure, when administered in newly diagnosed adults with T1D, helps to slow progression of T1D and promote insulin production by preserving beta cell function.

Immunotherapy Research

JDRF partnered with Orion Healthcare and Equity Partners to advance immuno tolerance therapy for T1D. Through this partnership, AnTolRX was created, a new company to develop an immunotherapy with microscopic particles developed by JDRF-funded research. Pfizer showed interest, and now has licensed AnTolRx’s immune tolerance therapy for further development. This work addresses the urgent need for disease-modifying T1D immunotherapies to slow down the autoimmune process and delay or block progression to symptomatic insulin-dependent diabetes.

The JDRF T1D Fund has backed innovation breakthrough at Provention Bio, Inc., a clinical stage biopharmaceutical company dedicated to sourcing, developing and commercializing novel therapeutics aimed at intercepting and preventing immune-mediated diseases. They have shared the in-licensing of an enterovirus vaccine platform, which the company is initially developing to reduce the onset of T1D by vaccinating at-risk populations against coxsackievirus B (CVB) infection. Research suggests that CVB infection could be responsible for more than half of T1D cases worldwide.

JDRF is working with IBM, leveraging their big-data capabilities to comb through years and years of data from generations of children with T1D, seeking to understand what might trigger the onset of the disease, and thus help us prevent it. This is a highly innovative partnership bringing together the best in industry leadership with research and non-profit to find solutions none of us could do alone.

JDRF also funds TrialNet, an innovative organization that advocates for T1D screening, that can help people better manage the disease before it catches them by surprise – sometimes with devastating results – and also advocates for participation in clinical trials that are key to advancing research in technologies, drugs and therapies.

Visit our blog next week to read our 2019 Mission Update Recap – Part 4: Improving Lives