Source: Juvenile Diabetes Research Foundation
In February, Vertex announced they were beginning a clinical trial for VX-880, their stem cell-derived therapy for use in people with type 1 diabetes (T1D). Today, they announced that the first person to receive this therapy now needs 91% less insulin 90 days after receiving an infusion of these fully differentiated cells at just half the target dose.
Last month, Vertex announced promising data from its Phase I/II Clinical Trial, that the first person with T1D to receive a stem cell derived therapy, VX-880, required 91% less insulin 90 days after receiving an infusion of these fully differentiated islets at just half the target dose. This exciting news can be traced back to 2000, when JDRF gave Dr. Douglas Melton a grant to make insulin producing beta cells, which he did in 2014.
Curing T1D in the 1.6 million Americans living with it and millions more around the world requires a renewable source of beta cells that can be produced in laboratory—and they must work. Once placed into the body, they need to be up to the task of restoring insulin production in people and automatically regulating blood-glucose levels. Although Vertex only shared the data for one individual, their data shows that VX-880 checks this box.
The patient also saw a significant reduction in HbA1c, improving from 8.6% to 7.2% without severe hypoglycemic events. As a reminder, this therapy is only being tested in people with severe hypoglycemia. This lower HbA1c was achieved with a 91% daily reduction in insulin administration.
This clinical trial is in an extremely limited patient population—people with severe hypoglycemia. The cells in VX-880 do not have any sort of protection from the immune system, which is why immunosuppressive drugs are required. For this therapy to be applicable for the entire T1D population, the cells need to both work and they need to function without or with minimal immunosuppressive therapies.
That’s why it’s also exciting to hear that Vertex plans to file an Investigational New Drug application with the FDA in 2022 for their encapsulated islet cell program, which could eliminate the need for immunosuppressives.
In the meantime, Vertex will continue their clinical trial in T1D’s with severe hypoglycemia. They are currently enrolling in several sites in the United States.