The video below titled “Beta Cells From Stem Cells Nearing a Cure for Type 1 Diabetes” features Dr. Anne L. Peters discussing the results of clinical trials involving beta cells derived from stem cells in patients with type 1 diabetes. The trials were conducted by a company named Vertex, for which Dr. Peters serves on the steering committee.
The trials involved the transplantation of beta cells derived from stem cells into patients. The dosage was determined based on previous experiences with beta-cell transplants from cadaveric donors. Initially, only half a dose was given due to caution from the US Food and Drug Administration.
Two patients were initially treated. The first patient responded incredibly well to the treatment, becoming insulin independent and essentially free of type 1 diabetes after over a year. The second patient showed some activity of the beta cells but not enough to achieve insulin independence. She received a second dose but decided to withdraw from the trial shortly after.
The trials then moved on to four additional patients who received a full dose of the beta cells. One of these patients has been followed for a year and, like the first patient, is off insulin. The remaining three patients are still being monitored, but early results show that the beta cells are working, producing insulin and showing positive C-peptide levels.
Dr. Peters emphasizes that this is a small sample size and there was variation in the response to treatment. As the trials progress, different doses and responses are expected. The side effects observed were related to immunosuppression, but there were no unexpected outcomes.
In summary, Dr. Peters expresses hope for this development, stating that the beta cells derived from stem cells seem to work in human beings as native beta cells might. This could potentially lead to newer therapies in the treatment of people with type 1 diabetes.