Evolving Concepts in Pathophysiology, Screening, and Prevention of Type 1 Diabetes: Report of Diabetes Mellitus Interagency Coordinating Committee Workshop

The approval of teplizumab to delay the onset of type 1 diabetes is an important inflection point in the decades long pursuit to treat the cause of the disease rather than its symptoms. NIDDK convened a workshop of the Diabetes Mellitus Interagency Coordinating Committee titled “Evolving Concepts in Pathophysiology, Screening, and Prevention of Type 1 Diabetes” to review this accomplishment and identify future goals. Speakers representing Diabetes TrialNet (TN) and the Immune tolerance Network (ITN) emphasized that the ability to robustly identify individuals destined to develop type 1 diabetes was essential for clinical trials. The presenter from the FDA described that regulatory approval relied upon data from TN's single clinical trial testing teplizumab for delay of clinical diagnosis, along with confirmatory evidence from studies in patients after diagnosis. The workshop reviewed the etiology of type 1 diabetes as a disease involving multiple immune pathways, highlighting the current understanding of prognostic markers, and proposing potential strategies to improve the therapeutic response of disease modifying therapies based on their mechanism of action. While celebrating these achievements funded by the congressionally appropriated Special Diabetes Program, panelists from professional organizations, non-profit advocacy/funding groups, and industry also identified significant hurdles in translating this research into clinical care.