July 8, 2022
Vertex Pharmaceuticals announced on Tuesday that the FDA will allow the company to continue its human trial of VX-880, the novel line of insulin-producing cells derived from stem cells. The trial has received considerable media attention since a New York Times story at the end of 2021 profiled a 65-year-old Ohio man whose need to take external insulin was eliminated after he received a half-dose of VX-880 alongside full-body immunosuppressive drugs.
In May, the FDA delivered a setback to Vertex when it halted the progress of the trial at a critical juncture. At that time, the agency determined there was "insufficient information to support dose escalation" from a half-dose, given with an abundance of caution to ensure the safety of the trial participants, to a full dose of VX-880. This week's announcement will allow Vertex to continue into the next stage of its trial, in which the company will recruit five people to receive full doses of VX-880, measuring the drug's ability to replace the need to take external insulin.
At this stage, VX-880 is limited by the need for full-body immunosuppression; it will take considerable innovation and testing to deliver a solution that can be available to everyone with T1D. We are excited that FDA has lifted the hold on this trial as research like this needs to progress quickly if there is any chance of that it will deliver a Practical Cure for T1D in the next 15 years.