A stem cell trial on a dozen Type 1 diabetes patients suffering from a severe form of the disease has provided at least a temporary cure for 10 participants. The remaining two also dramatically reduced the amount of insulin they require daily.
The treatment, with an infusion called zimislecel, was created by Vertex Pharmaceuticals and has been the life’s work of Boston-based researcher Dr. Douglas Melton, whose stem cell research began at Harvard University. He now works in the stem cell and gene therapy program at Vertex. Melton’s interest in “curing” diabetes began more than 25 years ago when his infant son and then adolescent daughter were both diagnosed with Type 1 diabetes.
The recent study was published in the online New England Journal of Medicine and presented at the annual meeting of the American Diabetes Association. Though the study included only 12 patients who suffer from a subset of Type 1 diabetes known as hypoglycemic unawareness, characterized by unexpected and dangerous drops in blood sugar, Melton said that even he was surprised by the success of the infusions, which have allowed most of those patients to live what they call “normal lives,” free of insulin and debilitating sugar fluctuations, for the last two years.
Melton joins host Robin Young to talk about his Vertex trial. Young is also joined by Amanda Smith, a Canadian patient who is now insulin-independent after participating in the trial.
This article was originally published on WBUR.org.
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