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New Vertex Type 1 Stem Cell Therapy Gets FDA Nod to Enter Clinical Trials

Published: 3/9/23 2:24 pm
By Andrew Briskin

The FDA announced that a new type 1 diabetes stem cell therapy from Vertex has been cleared to enter clinical trials in the United States. The therapy, which hopes to avoid the need for immunosuppressive drugs when replacing damaged insulin-producing cells in someone with type 1 diabetes, is scheduled to enter clinical trials in the first half of 2023.

Although there are several challenges that remain towards finding a safe and effective cure for type 1 diabetes, developments in stem cell therapy over the past year have offered much promise.

Biopharmaceutical company Vertex has been investigating its stem cell therapy, VX-880, as a way to replace the damaged insulin-producing beta cells in someone with type 1 diabetes with healthy beta cells created from stem cells. 

Stem cells are cells that have not yet matured into one of the many different types of cells in the body. Grown in a lab and given the right “biological instructions,” Vertex has developed a way to produce a virtually unlimited supply of insulin-producing beta cells for people with type 1 diabetes. 

Data released in June 2022 from the first person to receive VX-880, revealed that the participant increased his time in range to more than 99%, with glucose levels that nearly mirrored a person without diabetes. A major challenge, however, is that the participant required long-term immunosuppressive medications to stop his own immune system from destroying the new beta cells.

To address this issue, Vertex is testing another new therapy, VX-264, that keeps the beta cells in an enclosed device, physically protecting them from the body's immune cells while still allowing nutrients to reach the beta cells. This is a strategy known as encapsulation. 

The FDA clearance on March 9 allows Vertex to begin clinical trials on encapsulated stem cell therapy (VX-264). Vertex has announced that it plans to begin clinical trials for encapsulated insulin-producing stem cells in the first half of 2023, The therapy had also previously been cleared for clinical trials in Canada, with an early clinical trial already ongoing.

VX-264 uses the same stem cell-derived beta cells that were used in VX-880. Additionally, Vertex’s channel array device has been in development for many years and was acquired through its acquisition of Semma Therapeutics in September 2019.

Because Vertex has already been able to show that the cells, when protected, can restore insulin production in someone with T1D, there is hope that, when encapsulated in a specially-designed device, the therapy will be safer and more convenient for people with type 1 while eliminating the need for long-term immunosuppressive medication.

“Research advances only have the potential to make an impact if they reach clinical trials and people with T1D have an opportunity to participate in those trials," said Felicia Pagliuca, Disease Area Executive, Type 1 Diabetes, at Vertex.  This trial will be the first clinical study of device-encapsulated stem cell derived islets. We are committed to continuing to advance the development of potential new therapies for people living with T1D.”

For more about stem cell therapies and type 1 diabetes, read:

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About the authors

Drew Briskin joined the diaTribe Foundation in 2021 after graduating from the University of Pennsylvania where he majored in Health and Societies with a minor in Chemistry. As an undergraduate,... Read the full bio »