Stem Cell-Derived Beta Cells Show Promise for Blood Sugar Control in Type 1 Diabetes Patients, New Study Reveals

"An innovative stem cell-based therapy for Type 1 diabetes has shown the ability to effectively regulate blood glucose levels and decrease reliance on daily insulin injections," the researchers wrote.

The findings stem from a multicenter clinical trial investigating an experimental cell therapy developed by the U.S. biotechnology company ViaCyte, which has since been acquired by Vertex Pharmaceuticals and is currently being tested clinically in Canada.

Type 1 diabetes is characterized by the autoimmune destruction of insulin-producing β cells in the pancreas, leading to a dependence on insulin injections for blood glucose control. The new therapy is designed to replace the insulin-producing beta cells that individuals with T1D are missing. Known as VC-02, this small medical implant houses millions of lab-grown pancreatic islet cells, including beta cells, derived from a line of pluripotent stem cells.

The devices, roughly the size of a Band-Aid and no thicker than a credit card, are implanted just beneath the patient's skin. It is hoped that they will deliver a consistent, long-term supply of self-sustaining insulin.

The clinical trial took place at Vancouver General Hospital, along with additional sites in Belgium and the U.S. It involved ten participants, all of whom had no detectable insulin production at the start of the study. Each participant underwent surgery to receive up to ten device implants.

After six months, three participants exhibited significant markers of insulin production, maintaining these levels for the year-long study. These individuals experienced an increased duration within the optimal blood glucose range and a reduction in their reliance on externally administered insulin.

Notably, one participant demonstrated exceptional progress, with the time spent in the target blood glucose range rising from 55% to 85%, accompanied by a 44% decrease in daily insulin usage.

These results are the latest findings from a series of clinical trials funded by Canada's Stem Cell Network and carried out by the UBC-VCH team. The trials are designed to expedite the development and clinical evaluation of innovative stem cell-based therapies for the 300,000 Canadians affected by Type 1 diabetes. This condition is estimated to impose an annual cost of $29 billion on the Canadian healthcare system.

In a separate ongoing trial, the UBC-VCH team is exploring whether a version of the device that includes cells genetically engineered to evade the immune system, utilizing CRISPR gene-editing technology, could eliminate the need for participants to take immunosuppressant drugs in conjunction with the treatment.

Reference:

Keymeulen, B., De Groot, K., Thompson, D. M., Bellin, M. D., Kroon, E. J., Daniels, M., Wang, R., Jaiman, M., Kieffer, T. J., Foyt, H. L., & Pipeleers, D. (2023). Encapsulated stem cell–derived β cells exert glucose control in patients with type 1 diabetes. Nature Biotechnology, 1-8. https://doi.org/10.1038/s41587-023-02055-5