Stem cell therapies for Type 1 diabetes: current status and proposed road map to guide successful clinical trials

Many people with Type 1 diabetes struggle with the burden of self-management and are unable to achieve optimal glycaemic control without risk of hypoglycaemia. Future therapies with the potential to reduce the risk for short- and long-term complications while simultaneously reducing the burden of diabetes are therefore attractive. β-cell replacement is one strategy which might achieve this. Islet transplantation is limited by organ supply and the risks of long-term immunosuppression. Encapsulated stem-cell-derived β cells have the potential to address both of these issues and phase I/II clinical trials of encapsulated pancreatic progenitors have begun. A significant risk associated with the translation of stem-cell science to the clinical management of Type 1 diabetes is an underestimation of the complexity of the process and a mismatch between the hype and the expectations of both people with Type 1 diabetes and the public. We provide an update on progress in clinical trials of encapsulated stem-cell-derived β cells and propose a road map for the design and conduct of future trials to facilitate the translation of this exciting science to clinical care.