Stem cell-derived islet cell therapy may be future treatment option for patients with T1D

American Diabetic Associations in its 83rd Scientific session report's findings from the ongoing clinical trial of VX-880, a phase 1/2, multi-center, single-arm, open-label study in patients with type 1 diabetes (T1D) demonstrating the potential of stem cell-derived islet cell therapy as a future treatment option for patients with T1D.

Type 1 diabetes is characterized as the body’s abnormal immune system response, attacks and destroys the cells in the pancreas that make insulin.T1D patients usually presents with impaired hypoglycemic awareness and severe hypoglycemia, that often occurs because of imbalances between insulin administered relative to an individual’s requirements at a particular time that can be affected by a multitude of factors.

The researchers presented the study results were, all patients treated with VX-880 had undetectable insulin secretion and a history of recurrent severe hypoglycemic events (SHE) in the year prior to treatment; following treatment, all six patients demonstrated restored insulin secretion, improved glycemic control, improved time-in-range, reduction or elimination of exogenous insulin usage and complete absence of SHEs in the post-Day 90 evaluation period.

The study treated two patients with VX-880 (one in Part A of the study who, per protocol, received half the target dose, and one in Part B who received the full target dose) were treated for at least 12 months of follow-up and were evaluable for the study’s primary efficacy endpoint of elimination of SHEs between Day 90 and Month 12 with HbA1c of <7%.

The key findings of the trial are

• Both patients treated for more than 12 months are insulin independent; Patient A1 had HbA1c of 5.3% at Month 21 Patient B1 had HbA1c had HbA1c of 6.0% at Month 12 (compared to 7.6% at baseline).

• This level of glucose control is highly unusual in T1D patients treated with exogenous insulin, with recent data indicating that only approximately 25% of people with T1D meet the recommended HbA1c target of 7.0%.

• In fact, both patients displayed HbA1c levels that are below the diagnostic threshold for diabetes (6.5%).

• Both patients also showed over 95% time-in-range, well above the ADA recommended target of 70%, and substantial improvement over baseline.

• The three additional patients in Part B, each administered the full target dose of VX-880 given as a single infusion, have had up to 90 days of follow-up and also have shown insulin production, reduction in HbA1c, improvements in time-in-range and reductions in daily insulin usage.

• Their trajectory is consistent with that observed in the two patients with more than one year of follow up at equivalent periods of follow-up after VX-880 infusion.

The researchers concluded “These new findings demonstrate the potential of stem cell-derived islets as a future treatment for patients with type 1 diabetes, signaling a new era that could potentially remove the need for exogenously administered insulin to achieve glycemic control”.

Reference: Glucose-Dependent Insulin Production and Insulin-Independence in Type 1 Diabetes from Stem Cell–Derived, Fully Differentiated Islet Cells—Updated Data from the VX-880 Clinical Trial, Presented on Friday, June 23, 2023 in San Diego, California at 3:50pm PT as an oral presentation.