New ways for prevention of type 1 diabetes in children Possibilities and risks

There is considerable heterogeneity within the clinical phenotype of type 1 diabetes: the genetic susceptibility is complex and progression rates markedly differ, as does the efficacy of therapeutic measures. New prevention strategies should use biomarkers to lead to personalized combination treatment analogous to the approach used in pediatric oncology. It is well known that type 1 diabetes starts with autoimmunity and the detection of two or more persistent diabetes-associated antibodies (stage 1). An additional subclinical dysglycemia is termed stage 2 and manifest type 1 diabetes is termed stage 3. Both genetic and immunological parameters enable early detection in children and adolescents. Because 9 out of 10 affected children come from families without other family members having type 1 diabetes, population-based screening strategies are needed for prevention of type 1 diabetes. Studies on the psychological burden of screening procedures showed that the vast majority of families were able to live relatively unburdened by the knowledge of their child's increased diabetes risk after a short period of time. The Global Platform for Prevention of Autoimmune Diabetes (GPPAD) research network is investigating primary prevention with oral insulin (POInT study) or probiotics (S1NTIA study). The European research network "an innovative approach towards understanding and arresting type 1 diabetes" (INNODIA) is developing a standardized study protocol (master protocol) to accelerate the investigation of new single agents and combination therapies in patients over 5 years of age. Secondary prevention approaches are showing promising results in initial pilot studies to prolong the remission phase or delay disease onset in populations at risk.