Writing on his popular blog, stem cell researcher Paul Knoepfler, Ph.D., associate professor at UC Davis School of Medicine, shares his excitement that ViaCyte’s investigational new drug (IND) application was accepted by the FDA in August 2014. The acceptance paved the way for a Phase 1/2 clinical trial to test the safety and efficacy of our VC-01™ treatment for type 1 diabetes in humans. The VC-01 product uses the implantable Encaptra® pouch that holds ViaCyte’s PEC-01™ cells, which are derived from stem cells and are expected to further differentiate and mature into the full complement of islet cells, including beta cells, upon implant. The beta cells are able to release insulin into the blood stream, but the pouch is designed to protect them from attack by the immune system.
Knoepfler notes that the initial trials would enroll 40 patients with type 1 diabetes who have little or no insulin-producing beta cell function. Based on the positive outcomes testing the VC-01 product candidate in animal models, Knoepfler says “I really think there’s some hope here for human patients down the road with this and other similar kinds of approaches.”