DRI’s Dr. Jay Skyler Outlines the 4 Ideal Therapeutic Goals in T1D

Making science accessible is a top priority at the Diabetes Research Institute Foundation so that we can stay connected to our community and keep you updated on the latest advancements happening at the Diabetes Research Institute. Although we are unable to host tours of the labs right now, we have been bringing the researchers to our supporters through our Meet a DRI Scientist virtual series. At the most recent, interactive session, DRI Deputy Director Dr. Jay Skyler gave an overview of what’s happening in type 1 diabetes (T1D) research, particularly at the DRI.

Type 1 diabetes evolves from autoimmune beta cell destruction, which causes patients to become dependent on lifelong insulin therapy. According to Dr. Skyler, there are four ideal therapeutic goals in T1D:

  • Prevention of Immune Destruction
  • Preservation of Beta Cell Mass or Function
  • Replacement or Regeneration of Beta Cells
  • Automated Insulin Delivery

Addressing the first two goals, Dr. Skyler shared findings and statistical data from three recent and continuing studies being conducted at the DRI and through TrialNet.

  • DRI’s Dr. Allison Bayer together with Dr. Francesco Vendrame in the Division of Endocrinology have been investigating a drug that the FDA approved in 2013 to treat multiple sclerosis, called dimethyl fumarate (DMF). Ongoing experiments are showing dramatic effects on study models with 100% diabetes remission.
  • DRI’s Dr. Norma Kenyon is working with another agent, called anti-CD154, which is profoundly altering the progression of the disease in new onset study models and appears extremely promising in preventing the progression of islet transplant rejection without the need for harsh anti-rejection drugs.
  • The TrialNet Study explored the potential of low-dose anti-thymocyte globulin (ATG) in preserving beta cell function and showed remarkable results.

Dr. Skyler outlined the history of beta cell replacement and the DRI’s pioneering islet transplantation clinical trials, which have resulted in a number of patients living insulin free – some for more than 10 years. The DRI is urgently trying to overcome the challenges associated with cellular replacement strategies, including availability of insulin-producing cells, rejection of transplanted cells, and the recurrent autoimmune response.

Stem cells could be the key to solving the supply issue. Dr. Skyler spoke about approaches in reprogramming and transdifferentiation (transforming other cell types into insulin-producing cells). He also talked about immune protection strategies being pursued at the DRI, including encapsulation and the use of immunosuppressive or immunomodulatory drugs.

As he summed up his presentation, Dr. Skyler left supporters with an optimistic view toward the future…

“I think there’s a lot of hope,” he said. “And with that hope, I look forward to a future where we will have a world without diabetes. And I hope that indeed comes to pass.”

Jay S. Skyler, MD, MACP, is currently a Professor of Medicine, Pediatrics, & Psychology, in the Division of Endocrinology Diabetes & Metabolism, at the University of Miami Miller School of Medicine. He is also Deputy Director for Clinical Research and Academic Programs at the Diabetes Research Institute. Prevention of immune destruction has been a major focus of his work throughout his impressive career, which spans five decades. Beginning in 1993, he designed and served as Study Chairman for the nationwide multicenter NIH-sponsored Diabetes Prevention Trial for Type 1 Diabetes, and also served as Study Chairman of its successor, the NIH Type 1 Diabetes TrialNet Clinical Trials Study Group.

Interested in attending a future Meet a DRI Scientist virtual event? Send us an email.

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