The American Diabetes Association’s 84th Scientific Sessions is here! Until June 24, scientists will present the latest type 1 diabetes (T1D) research, from beta cell replacement to regeneration to complications, all with the goal of improving lives for the T1D community.
1. We have a therapy, Tzield™ (teplizumab-mzwv), that can prevent the clinical diagnosis for up to 3 years. But first, we need to screen for autoantibodies. Secondly, we need to predict when the beta cells show signs of decline, moving from Stage 2 to Stage 3. In a Breakthrough T1D-funded study, Peter Calhoun, Ph.D., gave a fascinating talk on how continuous glucose monitors (CGMs) can predict who will move onto Stage 3 T1D earlier. He took a dataset from five screening programs—TrialNet, ASK (Autoimmunity Screening for Kids), DAISY (Diabetes Autoimmunity Study in the Young), DIPP (Type 1 Diabetes Prediction and Prevention), and BDR (Belgium Diabetes Registry)—and was able to show that incorporating CGM metrics into individual characteristics had the best predictive performance to classify individuals of the impending clinical onset of T1D.
At-risk, or Stage 2 T1D, means that a person exhibited 2+ T1D-related autoantibodies—antibodies against one’s own self—and their blood glucose is starting to be abnormal, but they are not yet insulin dependent. When someone becomes insulin dependent, they are in stage 3 T1D.
2. Kevan Herold, M.D., presented a secondary analysis of the phase III PROTECT clinical trial. In October 2023, Tzield™ (teplizumab-mzwv) showed that it can slow the loss of beta cells and preserve beta cell function in newly diagnosed (stage 3 T1D) children and adolescents ages 8-17. This analysis demonstrated that, in addition to slowing down the loss of beta cells in new-onset T1D, Tzield can decrease insulin dose and improve time-in-range. This reinforces our commitment to supporting therapies that preserve beta cells at onset, which is important for the prevention of complications and improvement in new-onset clinical factors.
Dr. Herold has been supported by Breakthrough T1D since the late 1980s. In his research, he showed that he could prevent autoimmune diabetes with an immune-modifying antibody (which, later, became a humanized version, Tzield) and was the lead on the clinical trial that demonstrated that Tzield could delay the onset of T1D in people almost certain to develop the disease. In November 2022, Tzield was approved by the FDA to delay the onset of the disease (Stage 3) in at-risk (Stage 2) individuals ages 8+.
3. Vertex Pharmaceuticals gave a highly anticipated update on the phase I/II clinical trial of VX-880, their stem cell-derived islet cell therapy. 12 individuals in the study have received the full, target dose of cells, and the results are remarkable. 11 of the 12 participants have reduced or eliminated the need for external insulin, and all 3 individuals who received the therapy at least 12 months ago have eliminated low blood sugar events and are fully off external insulin—meeting the primary and secondary endpoints.
This research was made possible by years of funding by Breakthrough T1D and the T1D Fund to Doug Melton, Ph.D., and Semma Therapeutics—a biotech company founded by Melton to develop a stem cell-derived islet therapy for T1D—which was acquired by Vertex Pharmaceuticals.
