Novel stem cell therapy shows promise for Type 1 diabetes patients

VC-02 [Courtesy of ViaCyte]

An experimental stem cell therapy for Type 1 diabetes proved its efficacy in an initial clinical trial, according to the latest study.

Type 1 diabetes occurs when the immune system, the body’s system for fighting infection, attacks the insulin-producing beta cells in the pancreas. Patients with Type 1 diabetes must inject themselves with insulin at least once a day throughout their lifetime.

The stem cell treatment, known as VC-02, involves a small medical implant containing millions of lab-grown pancreatic islet cells, including beta cells, derived from pluripotent stem cells. The implant in the size of a medical patch is designed to produce insulin and help Type-1 diabetes patients regulate their blood glucose levels.

The VC-02 treatment has many advantages over the conventional implant approach involving a healthy donor.

The treatment was developed by ViaCyte, Inc., a U.S. pharmaceutical firm acquired by Vertex Pharmaceuticals, and global phase 1 and 2 trials for the treatment have already been completed.

One of the trials was a study recently conducted by a research team led by David Thompson, a professor of endocrinology at UBC and director of VCH’s Vancouver General Hospital Diabetes Center, which confirmed the efficacy of the beta-cells implanted into patients who lack insulin.

The clinical trial involved ten participants with no detectable insulin production at the study’s outset, with each patient getting up to ten implants of beta cells injected in their body. The researchers measured the patients’ blood C-peptide concentration after a meal, the marker of insulin production, every three months to assess the impacts of the implants on blood sugar and insulin levels.

Six months later, three participants exhibited significant improvements in insulin production, and maintained these levels throughout the year-long study.

One of the three showed remarkable improvement, with an increase in time spent in the target blood glucose range to 85 percent from 55 percent, and a 44 percent reduction in daily insulin administration.

The research team is now exploring a version of the implant containing cells genetically engineered to evade the immune system using CRISPR gene-editing technology in an ongoing trial.

The researchers hope that the treatment can bring hope for a future free of insulin injections for patients.