Islet cell transplant trial outcome ‘major milestone’ toward cure for diabetes

A clinical trial for Type 1 diabetes, involving the transplant of islet cells derived from embryonic stem cells (Zimislecel), has yielded 'spectacular' results, with 10 out of 12 participants achieving insulin independence one year post-transplant. Amanda Smith, from London, Ont., was the first Canadian to receive this transplant at Toronto General Hospital in February 2023, subsequently gaining blood sugar control similar to a non-diabetic. While patients must take anti-rejection drugs for life, the procedure effectively eliminated dangerous hypoglycemic episodes. The international trial, funded by Boston-based Vertex Pharmaceuticals, was temporarily paused last year following two participant deaths, though these were unrelated to the therapy itself. This breakthrough is considered a major step towards a cure, building on previous work like the 'Edmonton Protocol.'

1. 1 2000: Dr. Shapiro and colleagues published the 'Edmonton Protocol'.
2. 2 February 2023: Amanda Smith became the first Canadian to receive the transplant.
3. 3 Last year: Vertex Pharmaceuticals temporarily paused the trial after two participant deaths (unrelated to the therapy).
4. 4 Friday (June 20, 2025): Results of the clinical trial were published in the New England Journal of Medicine.
5. 5 Next year: Vertex expects to apply for regulatory approval of Zimislecel.
6. 6 Future: Scientists aim to find ways to genetically engineer or encapsulate islets to avoid the need for immunosuppressants.

• Patients can achieve insulin independence
• Elimination of dangerous hypoglycemic episodes
• Patients must commit to lifelong anti-rejection medication
• Potential for regulatory approval of Zimislecel starting next year
• Considered a major milestone towards a cure for Type 1 diabetes