Vertex has presented new data on its clinical trial VX-880, hailed as a potentially revolutionary treatment for people with Type 1 diabetes.
VX-880 is a type of experimental stem cell therapy which aims to restore natural insulin production in people with Type 1 diabetes. It works by transplanting healthy insulin-producing cells into the body. If successful, the new cells would replace non-functioning ones and ultimately restore insulin production. Similar to people who receive organ transplants, those treated with VX-880 must take immunosuppressants to prevent the immune system from rejecting new beta cells. Because of the risk of infection associated with immunosuppressants, only those with severe hypoglycaemia unawareness have been eligible for the trial, as these have the most to gain from this type of therapy.
The results of the study so far have been promising. All 12 participants who received a full dose of VX-880 saw a reduction in A1C of less than 7% and no longer experienced severe hypoglycaemia. Eleven had either a reduced need for or complete elimination of exogenous insulin (injectable insulin not naturally produced by the body). In addition, all participants experienced significant improvements in A1C, time in range, as well as a reduced need for insulin injections.
Of the four participants who passed the one-year mark following treatment, all achieved insulin independence and maintained an A1C of less than 7%. This indicates that VX-880 could be a much-awaited functional “cure” for Type 1 diabetes. However, while the results so far are exciting, research is still in the early stages. The current trial is anticipated to wrap up in 2030, and additional studies with larger sample sizes would need to follow. After all of the studies are completed, Vertex would need to submit an application to the FDA before it can be approved for use.
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