Pluripotent stem cells as a therapy for type 1 diabetes.

Abstract

The current most pragmatic therapy for type 1 diabetes (T1D) is the administration of insulin through a pump, with continuous monitoring of glucose levels. Replacing the pancreatic ß cells that were destroyed by an autoimmune process will achieve more physiological control of blood glucose levels. However, this treatment is only available to patients with hypoglycaemic unawareness if the benefit outweighs the risk due to the restricted supply of donor pancreatic islets and the requirement that recipients take toxic anti-rejection medicines. Pluripotent stem cell derived ß cells are now a reality, thanks to progress in developmental biology over the past decade. It takes 4 weeks in a laboratory to differentiate ß cells from pluripotent stem cells. The administration of these cells in the clinic requires a bioengineered device that can immunoisolate the ß cells and avoid their rejection by the recipient. The first human trial with ß cell precursors in such a device commenced in 2014 in California, USA, showing safety but not efficacy because of the host reaction to the implant. In the USA, Europe and Australia, different devices have progressed to phase 1b/2a clinical trials. It is reasonable to believe that with such forces in play and the continuing involvement of large pharmaceutical companies, a practical solution to the administration of pluripotent stem cell derived ß cells is near at hand.