Stem cell and islet transplant for children with genetic diabetes and immune problems

This trial is testing a new treatment approach called sequential transplantation of umbilical cord blood stem cells and islet cells for children and adolescents with a rare genetic form of type 1 diabetes that also affects the immune system. The treatment involves two transplant procedures: first, stem cells from umbilical cord blood are transplanted to help restore immune function; then, islet cells (which produce insulin) are transplanted to help control blood sugar. Because this genetic form of diabetes can cause serious infections and damage to multiple organs, this new treatment may offer hope where traditional diabetes management alone is not enough.

Children with this rare genetic form of diabetes face a difficult situation—their weak immune system makes them prone to serious infections and complications, while their damaged pancreas cannot produce enough insulin. This trial exists because early treatment with this new transplant approach may be able to prevent organ damage and infections that currently lead to severe complications and early death in these children.