Memorandum

JDRF workshop proposes new classification system for early stages of type 1 diabetes – October 14, 2014

JDRF hosted a workshop last Friday in Bethesda, MD titled “Defining the Early Stages of Type 1 Diabetes.” The centerpiece of the meeting was a tentative staging process designed to break up the current “monolithic” (as the ADA’s Dr. Robert Ratner put it) definition of type 1 diabetes into a more refined, segmented system that would facilitate individualized therapy and catalyze the development of new therapies to slow or reverse the disease’s progression. Under the proposed paradigm for the earliest stages, Stage #1 would include patients with at least two islet autoantibodies, Stage #2 would be marked by the onset of dysglycemia, and Stage #3 would be equivalent to full-scale symptomatic diabetes. Until the development of proven interventions for disease prevention, the main clinical benefit of such a system would be the ability to identify at-risk patients earlier in disease process and generally smooth the transition into symptomatic type 1 diabetes (particularly by beginning insulin earlier and reducing DKA). From a drug development perspective, the staging system could also help facilitate more efficient clinical trials, as intermediate endpoints (such as the progression from one stage to another) could be used in lieu of symptomatic diabetes diagnoses (which, as an endpoint, requires trials that last many years). Additionally, the staging could facilitate better risk/benefit assessment of potential therapies, by clarifying the risk of “doing nothing” (the expected progression of the disease at each stage) and enabling more appropriate targeting of potential prevention therapies by stage. As a follow-up to this meeting, a Consensus Conference focused on research in pediatric diabetes patients will take place on January 14 in Alexandria, VA (contact spendleton@diabetes.org to register by December 1), and possible meeting(s) later in 2015 could extend the staging system beyond Stage #3, perhaps on the basis of complications. We were certainly impressed by the range of diabetes foundations, researchers, clinicians, industry representatives, and regulatory figures represented in the room – stay tuned for our full report from the packed day.