Earlier this month, San Diego-based ViaCyte announced that it was conducting human tests using an islet-cell replacement candidate product for patients with type 1 diabetes. The product, PEC-Direct, produces pancreatic progenitor cells that can replace damaged or missing pancreatic cells. Pancreatic progenitor cells derive from stem cells and develop into islet cells, which produce insulin.
University of Albert and UC San Diego School of Medicine research teams are conducting the clinical trials. ViaCyte expects to expand the initial testing at other medical centers in the US and Canada in the coming months.
The first trial focuses on subjects with type 1 diabetes who are at serious risk of developing life-threatening hypoglycemia. Type 1 diabetes results when the body’s immune system incorrectly attacks and destroys the body’s own insulin-producing islet cells. Only about 10% of diabetic patients have type 1 diabetes.
ViaCyte will evaluate the first PEC-Direct devices, which are implanted in the patient’s forearm, for safety and conceptual verification. Following device implantation, researchers expect the progenitor cells to mature into human islet tissue that will produce insulin. Patients with type 1 diabetes have virtually no ability to produce insulin on their own. Researchers will monitor the test subjects to see if the implanted cells begin producing insulin.
Replacement therapies using stem cells have been tried before
Researchers have used replacement therapy to treat type 1 diabetes for about 20 years. In that time, cadaver pancreas transplants have shown promise in managing unstable type 1 diabetes. Currently, about 42 million people suffer from type 1 diabetes. That means transplantation is not a large-scale treatment option. The use of progenitor cells as an on-demand islet cell replacement therapy could provide a theoretically unlimited source of the desired tissue.
As part of the evaluation, the researchers will remove the first implants to examine the maturity of the progenitor cells. They will also look for evidence of direct vascularization of the cells in the device. A second group of as many as 40 test subjects will receive the device to evaluate both its safety and long-term function.
If the PEC-Direct device works as expected, it could represent a functional cure for type 1 diabetes. Since the device does not treat the underlying immune system malfunction, patients would still have type 1 diabetes. By manufacturing new islet cells, however, the device could provide adequate glycemic control in these patients.
In addition to looking for evidence of insulin production, researchers will also evaluate subjects’ use of injectable insulin. The researchers expect to gain insight into how well the device works by mid-2018. To prevent rejection or other immune system complications, test subjects will need to use immunosuppression therapy with the PEC-Direct device.
The PEC-Direct device underscores the importance of stem cells in both disease research and the development of new therapies for life-threatening conditions. If you would like more information about stem cells and the work of the National Dental Pulp Laboratory, please visit our website at http://ndpl.net or call us at (774) 843-2984.
Photo Credit: Community Eye Health, via Flickr