Cell therapy restores islet cell function

Friday 3 December 2021

United States

A stem cell-derived therapy has restored the function of pancreatic islet cells in a patient with Type 1 diabetes in what is being described as the first demonstration of a potentially curative treatment for the disease. The therapy is being developed by Vertex Pharmaceuticals Inc on the basis of research conducted by Douglas Melton, a professor at Harvard University and co-director of the Harvard Stem Cell Institute in Cambridge, US.

Vertex reported data from the first administration of the treatment on 18 October. On 29 November the international edition of The New York Times published an interview with the patient who no longer requires injections of insulin to control sugar in his bloodstream. Type 1 diabetes is a chronic condition in which islet cells of the pancreas produce little or no insulin, a hormone which is necessary for glucose control. The patient, 64-year-old Brian Shelton, received the treatment in late June as part of a Phase 1/2 trial of VX-880, a pancreatic islet cell replacement therapy.

Mr Shelton was given half the target dose in conjunction with immunosuppressive therapy. According to Vertex, he achieved a successful engraftment and immediately showed an improvement in glycaemic control. At Day 90, he had achieved a 91% decrease in his daily insulin requirement.

In a statement, James Markmann, head of transplant surgery at Massachusetts General Hospital, US said: “As a surgeon who has worked in the field if islet cell transplantation for decades this [cell therapy] approach, which obviates the need for an organ donor, could be a game changer.” Other healthcare experts expressed astonishment at the results but were also cautious, pending trial data from other patients, according to The New York Times.

Mr Shelton was diagnosed with Type 1 diabetes when he was in his twenties and since then, has been dependent on regular insulin injections. In the year prior to treatment with the new therapy, he experienced five severe, potentially life-threatening hypoglycaemic episodes.

Altogether, Vertex plans to treat about 17 patients in the trial. At the same time, it is progressing preclinical studies of the cell therapy encapsulated in a device. This is to protect it from the immune system. If successful, this would eliminate the need for immunosuppressive drugs.

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