In a development described as “unprecedented,” researchers recently presented the results of a trial in which a person with type 1 diabetes (T1D), after a single treatment, was able to entirely discontinue using insulin. The report was given at the annual meeting of the American Diabetes Association.
The subject was a 64-year-old man who had had type 1 diabetes, along with a host of complications, for 40 years. In the year previous to therapy, the subject had experience five severe episodes of low blood sugar (hypoglycemia). According to lead study author James F. Markmann, MD, of Massachusetts General Hospital in Boston, “His life was being destroyed by diabetes. He couldn’t work, he crashed his motorcycle from lows and really was tremendously appreciative that he could participate.”
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The patient was given a single half-target dose of a treatment known as VX-880, which was developed by Vertex Pharmaceuticals Incorporated, a biotech company based in Boston. VX-880 is what’s known as an “allogeneic” therapy, which means the procedure uses healthy stem cells from a donor to replace the patient’s own defective cells, in this case insulin-producing pancreatic islet cells. As Vertex explains it, “VX-880 has the potential to restore the body’s ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose-responsive insulin production.”
The VX-880 was infused into the patient’s hepatic portal vein — the vein that delivers blood to the liver from the intestines, spleen, gallbladder, and pancreas. As Dr. Markmann explained, “This is the first in-the-world administration of stem cell-derived islets in this manner where they’re infused into the liver.” As part of the procedure, the patient received immunosuppressive therapy to defend the islet cells from rejection.
Insulin independence achieved after administration of VX-880
Ninety days after the treatment, the patient’s HbA1c level (a measure of long-term glucose control) had dropped from 8.6% to 7.2%, his insulin usage had gone from 34 units per day to just 2.9, and his time spent in target range rose from just over 40% to 63.2%. By day 150, his HbA1c was 6.7% and his time spent in target range was 81.4%. After 270 days the subject had achieved complete insulin independence. His HbA1c was 5.2% and his time spent in target range was 99.9%.
The researchers also presented information on a second patient, who at the time of the American Diabetes Association meeting had been on the VX-880 therapy for 150 days. Over that period, the subject’s HbA1c had dropped from 7.5% to 7.1% Time spent in range rose from 35.9% to 51.9% and insulin usage decreased from 25.9 units per day to 18.2.
Like the first patient, the second required immunosuppression treatments, which have become standard procedure to prevent the body from rejecting transplanted cells. Dr. Markmann explained that not all patients are good candidates for immunosuppression, and therefore his team is concentrating on those for whom the therapy appears most promising. The ultimate goal, however, is to avoid the use immunosuppression as part of treatment. As Dr. Markmann put it, “If we had a way to transplant the cells without immunosuppression, then it could be really widely available. That’s one of the great opportunities hopefully in the future since these cells can be made in unlimited quantities.”
A third patient, the researchers reported, has just begun therapy. While the first two patients had received a half target dose, this one has been given a full target dose, and the researchers say that the next phase of the trial will be a “dose-escalation” study. “The study continues to enroll and dose patients,” the researchers reported, “These unprecedented results are the first evidence that stem cell-derived islets can restore insulin production and glucose control in T1D.”
Want to learn more about type 1 diabetes? Read “Type 1 Diabetes Questions and Answers,” “Six Type 1 Diabetes Symptoms You Need to Know” and see our type 1 diabetes videos.
