Stem-Cell Islets for Type 1 Diabetes
In 2025 Vertex's stem-cell-derived islet cells left 10 of 12 people with type 1 diabetes free of insulin injections after a year, but they need lifelong immunosuppression.
Open in the interactive tree →Type 1 diabetes destroys the pancreas cells that make insulin. Donor islet transplants work, but a transplant needs islets from about two pancreases and donors are scarce. Vertex's zimislecel, islet cells grown from stem cells and infused into the liver's portal vein, left 10 of 12 full-dose recipients free of insulin injections after a year in a trial published in the New England Journal of Medicine in June 2025. All recipients took drugs that suppress the immune system.
As of now
In the 2025 trial all 12 full-dose recipients reached a healthy blood-sugar range; two of 14 participants died, deaths Vertex said were unrelated to the cells. Vertex had planned regulatory filings for 2026, but paused dosing in the Phase 3 portion for an internal manufacturing analysis; it resumed dosing by spring 2026 and said in its mid-2026 quarterly report that updated timelines would follow later in 2026. Its device-encased version (VX-264) failed its efficacy goal in March 2025. In 2025 Sana Biotechnology reported gene-edited donor islets in one patient that made insulin for at least 12 weeks without any immunosuppression, a single-patient company result.
Where today's AI could help
Medium AI leverage. AI can forecast which stem-cell batches become good islet cells, but safety, rejection and durability can only be tested in patients.
- Predict from early microscope images whether a stem-cell batch will turn into insulin-making cells
- Screen gene edits that let transplanted cells hide from the immune system
Shown so far
- A 2025 bioRxiv preprint (not peer reviewed) trains a deep-learning model to forecast stem-cell-to-beta-cell differentiation efficiency from early phase-contrast images. source
Prerequisites
- Insulin1921Injected insulin keeps patients alive but cannot match natural sugar control.
- Ciclosporin and Anti-Rejection Drugs1978-1983Transplanted cells are still attacked without anti-rejection drugs.
- Induced Pluripotent Stem Cells2006Stem cells are the unlimited source of insulin-making cells.