October 12, 2023

Last week, Vertex Pharmaceuticals shared continued positive results from its phase 1/2 human trial VX-880, presented at the annual meeting of the European Association for the Study of Diabetes that took place October 2-6.

The company announced that three trial participants are now fully insulin-independent after islet transplantation. Participants were dosed with VX-880 insulin-producing islets derived from stem cells.

VX-880 cells are being used as the basis for another phase 1/2 human trial underway called VX-264, classified as a potential Practical/Functional Cure. Although VX-880 itself is not a potential Practical Cure as it requires chronic immunosuppression to keep the cells safe from the autoimmune attack, this extended success is an undeniable mile marker on the road to a Practical Cure for type 1 diabetes.


Three Patients Insulin-Independent

Results shared by Vertex state that after a 90-day follow-up, all six patients in the VX-880 trial demonstrated improved glycemic control across the board, improved time-in-range, a decrease in HbA1c, and the reduction or complete elimination of external insulin use. Of these six patients, three have achieved insulin independence and report HbA1cs less than 7.0%. Before transplantation, all patients had long-term type 1 diabetes with no ability to produce their own insulin, experiencing dangerously low blood sugar levels.

The first patient achieved insulin independence 270 days after the initial procedure. The individual had long-term T1D for almost 42 years and required 34 units of insulin daily. Patient 2 achieved insulin independence 180 days after the treatment. This patient has been diagnosed with T1D for 19 years, requiring 45.1 units of insulin a day. After the cut-off date for evaluation, a third patient achieved insulin independence after 180 days, though the length of type 1 diagnosis has not yet been disclosed.


VX-264 Uses Same Cells in Potential Practical Cure

VX-264 is Vertex’s Practical Cure project that began in May of this year. The phase 1/2 trial utilizes VX-880 cells with an added encapsulation device to protect them from the autoimmune system’s attack. VX-264 is still in the early stages, but if successful, would negate the need for any immunosuppression. Thus, continued success of the VX-880 trial increases the success of VX-264, a promising contender in the race for a Practical Cure.

In addition, Vertex is collaborating with CRISPR Therapeutics to create immune-resistant islet cells and is partnering with Lonza Bioscience to manufacture these cells on a commercial scale.


Real Potential for a Practical Cure

Three patients achieving insulin independence for a sustained period of time is exciting and promising. It unequivocally moves us further in the direction of an eventual Practical Cure, but there is still a long way to go.

A much larger number of patients must be tested in clinical trials to prove Vertex’s cells are safe and effective. Then, the cells must be protected from the autoimmune response so that chronic immunosuppression is unnecessary. Cell protection currently being developed and tested by Vertex includes an encapsulation device and gene modification, but neither have posted results.

Links to Additional Information
Vertex Pharmaceuticals VX-880 Press Release: Click here.
VX-880 trial information: Click here.
VX-264 trial information: Click here.
JDCA report on Lonza stem cell facility: Click here.
JDCA list of current Practical Cure Projects (US): Click here.