Vertex Pauses Diabetes Trial Following Two Patient Deaths
Vertex Pharmaceuticals put recruiting for its clinical trial investigating a stem cell therapy for type 1 diabetes on hold after two participants died. Vertex has said the deaths are unrelated to the trial treatment.
Following the deaths, Vertex Pharmaceuticals paused further recruiting of the ongoing trial of VX-880, an investigational stem cell therapy used to restore insulin production in people with type 1 diabetes.
Brian Shelton, 66, one of the patients who died, was said to be the first person “cured” of type 1 diabetes with VX-880. Shelton successfully started producing his own insulin in 2021, six months after being treated with VX-880. Vertex has not identified or released any information about the other participant who died.
Global regulators and an independent monitoring committee are reviewing study data and are expected to meet later this month. The committee will announce a decision on whether the deaths were related to the investigational treatment and if the trial should resume recruiting new participants.
Based on the latest data analysis, all 14 current participants dosed with VX-880 produced insulin. Though still in its early phases, study data supported VX-880 as a promising and potentially revolutionary treatment for type 1 diabetes.
What does the trial pause mean for study participants?
While the study is on hold, no new patients can start VX-880 therapy. However, the trial is continuing for existing participants who will carry on treatment with VX-880.
Vertex has reported VX-880 as being well-tolerated with no serious side effects. So far, the safety profile of VX-880 is similar to what would be expected given the use of immunosuppressants, the surgical procedure to implant the cells, and the participants’ medical history.
This is the second time the VX-880 trial has been paused. In May 2022, the FDA put a clinical hold on the VX-880 study due to concerns around dose escalation, though the hold was lifted a few months later.
It’s unclear how the clinical trial pause may affect Vertex’s timeline for completing the study and submitting VX-880 to regulators.
What does the latest data about VX-880 show?
Last year, Vertex shared promising updates from the trial, finding that all 14 participants who received VX-880 therapy were able to naturally produce insulin. This is up from the three participants with insulin independence announced in October 2023.
Before VX-880 treatment, study participants had a history of severe, recurring episodes of hypoglycemia and were unable to produce insulin. In addition to no longer requiring insulin injections, participants also achieved an A1C lower than 7% – the goal recommended by the American Diabetes Association – and improved time in range.
The data has shown promise for VX-264, another Vertex stem cell therapy that does not require immunosuppressants currently being tested in clinical trials.
Learn more about stem-cell therapies for type 1 diabetes: