Vertex Resumes Trial After Pause Due to Two Patient Deaths
By Anna BrooksApril Hopcroft
An independent monitoring committee determined the deaths were unrelated to the study drug, a stem cell therapy for type 1 diabetes.
Following a three month pause in early 2024, Vertex resumed the trial of VX-880, an investigational stem cell therapy used to restore insulin production in people with type 1 diabetes.
Vertex paused the study in January 2024 after two participants died. During the pause, an independent data monitoring committee reviewed the data and determined that the deaths were not related to the trial treatment. The study has since resumed and Vertex is now recruiting new participants again.
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 was on hold, no new patients were able to start VX-880 therapy. During the pause, the existing participants continued their regularly scheduled 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 was 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 if the pause will affect Vertex’s timeline for completing the study, advancing into phase 3 clinical trials, and applying for FDA approval.
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.
We will continue to keep you informed about this exciting story – more information will be shared at the upcoming 84th American Diabetes Association Scientific Sessions conference.
Learn more about stem-cell therapies for type 1 diabetes:
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New Vertex Type 1 Stem Cell Therapy Gets FDA Nod to Enter Clinical Trials
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ViaCyte and CRISPR Introduce New Stem Cell Therapy for Type 1 Diabetes
