The discovery of insulin, over 100 years ago, was a major milestone offering the possibility to treat the previously lethal disease. However, our understanding of the pathogenesis of type1 diabetes (T1DM) is still limited and a cure has not yet been identified. Life expectancy is still reduced.¹

A diversity of interventions has been studied to arrest progression of beta cell destruction shortly after the onset of the symptomatic disease, however without lasting effect. Over the last decades a wealth of data has been collected on the progression to clinical diabetes in first degree family members with high risk HLA genotype and/or with multiple auto antibodies, with or without medicinal or nutritional interventions. This has led to the scientific statement on the pre-symptomatic stages of T1DM.²

Studying the disease in the early stages will lead to the Identification of better (surrogate) biomarkers for progression, improved understanding of potential disease pathways, and, targeted interventions to arrest T1DM. So far one product is approved by FDA to delay progression,³ confirming the importance to study these pre-symptomatic stages.

Initially, studies were limited to first degree family members. Most children with stage 3 T1DM have no T1D family members and too many present in diabetic ketoacidosis (DKA). Therefore several countries have started to screen the general population

This can reduce the frequency of DKA at onset, but it will create other challenges for families and health care professionals (HCPs). These need to be identified and addressed. Consensus Guidelines to support families, patients and (HCPs) in this new era are a high priority to manage the persons after screening until we can delay or cure T1DM.

References

1. Rawshani, et al. Lancet Diabetes and Endocrinol 2018.

2. Insel, et al. Diabetes Care 2015.

3. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-can-delay-onset-type-1-diabetes