Investigational Stem Cell-Derived Islet Cell Therapy Shows Promise in Treatment of Type 1 Diabetes

An investigational stem cell-derived islet cell therapy, VX-880, demonstrated as a potential future treatment for individuals with type 1 diabetes (T1D), according to results from a small, ongoing phase 1/2, multicenter, single-arm, open-label clinical trial (NCT04786262).

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“These new findings demonstrate the potential of stem cell–derived islets as a future treatment for patients with [T1D], signaling a new era that could potentially remove the need for exogenously administered insulin to achieve glycemic control,” Trevor Reichman, MD, PhD, surgical director of Pancreas and Islet Cell Transplantation at the Ajmera Transplant Center at the University of Toronto, said in a statement. “We are hopeful that this first-of-its-kind research could be a gamechanger for the treatment of [T1D].”

Investigators found that all individuals had restored insulin secretion, improved glycemic control, improved time-in-range reduction, or elimination of exogenous insulin usage. Additionally, investigators found that there was also a complete absence of recurrent severe hypoglycemic events (SHEs) in the post–day 90 evaluation period.

All individuals who were treated with the investigational therapy had undetectable insulin secretion and a history of SHEs in the prior year before treatment. There was a focus on adults who had T1D with impaired hypoglycemia awareness and severe hypoglycemia. This can affect diet, sleep, stress, and activity levels.

According to the statement from the American Diabetes Association (ADA), if T1D is left untreated, it can lead to severe hypoglycemic events, causing loss of consciousness, coma, seizures, or serious injury. The current standards of care do not address the underlying causes and there are limited treatment options for the management of T1D.

Individuals in part A of the study received half of the target dose while those in part B received the full target dose. The 2 individuals treated with VX-880 in part A were treated for at least 12 months of follow-up. Investigators evaluated the individuals’ responses between day 90 and month 12 for the primary efficacy end point of elimination of SHEs with hemoglobin A1c (HbA1c) of less than 7%.

Both individuals were treated for more than 12 months and were insulin independent, with one individual having HbA1c of 5.3% at month 12 compared with 8.6% at baseline while the other had HbA1c of 6% at month 12 compared with 7.6% at baseline. Investigators noted that both individuals were below the diagnostic threshold for diabetes.

Additionally, both individuals also showed over 95% time-in-range, which is above the ADA recommended target of 70%.

The 3 patients included in part B were given the full target dose as a single infusion, had up to 90 days of follow-up, and showed insulin production, reduction in HbA1c, improvements in time-in-range, and reductions in daily insulin usage.

Investigators reported that the trajectory is consistent with the individuals in part A with more than 1 year of follow-up at equivalent periods after VX-880 infusion.

Vx-880 was generally well tolerated and safe for all individuals who were administered the drug to date. The safety profile is consistent with immunosuppressant therapies and cadaveric islet cell transplantation, with the majority of adverse events being mild or moderate in severity.

Because of the positive findings, an independent data review committee has recommended moving forward with part C of the trial, which will include the concurrent dosing of individuals at the full target dose. The trial will also expand to additional sites including Norway, Switzerland, and the Netherlands.

The results of the study were presented at the 83rd Scientific Sessions of the ADA, which was held in San Diego, California.

Reference

Novel stem cell-derived islet cell therapy continues to show promise for achieving insulin independence for individuals with type 1 diabetes. News release. American Diabetes Association. June 23, 2023. Accessed June 27, 2023. Email.