JDRF, the leading research and advocacy organization funding type 1 diabetes (T1D) research announced today ViaCyte, Inc., a leading regenerative medicine company, has filed an Investigational New Drug application (IND) with the U.S. Food and Drug Administration (FDA) seeking to initiate a Phase 1/2 clinical trial in patients with type 1 diabetes. The trial would evaluate the safety and efficacy of the VC-01 product candidate, a stem cell-derived, encapsulated cell replacement therapy. In a related development, ViaCyte submitted a Medical Device Master File (called MAF) to the FDA in support of the Encaptra drug delivery system, the device component of the VC-01 product candidate.
"[This] IND announcement is a significant milestone and provides momentum on the path to conducting the first clinical study of this potentially transformative therapy for people with type 1 diabetes (T1D),” said Jeffrey Brewer, JDRF president and CEO. “We are excited to continue our collaboration with ViaCyte and believe beta cell encapsulation therapy may one day virtually eliminate the daily management burden for those living with T1D. The ability to encapsulate and thereby protect implanted insulin-producing cells has been a focus for JDRF and a product candidate like VC-01 could potentially play a key role in helping us achieve our vision of creating a world without type 1 diabetes.”
Beta cell encapsulation therapy is a minor procedure that involves putting cells with the capacity to ultimately secrete insulin in a glucose-responsive manner in a protective barrier and implanting them in the body. These encapsulated beta cells will sense a person’s glucose levels and produce insulin as needed, while the barrier shields them from the body’s autoimmune attack that initially triggered the onset of T1D. Encapsulation keeps the newly implanted cells alive by hiding them from the immune system, providing a safe environment where they can function normally. The cells can constantly assess the amount of glucose in the blood and release exactly the correct amount of insulin. Importantly, encapsulation therapy overcomes one of the major obstacles that has slowed progress in the islet transplant field, which is the need for lifetime administration of powerful and toxic immunosuppressive drugs to prevent the immune system from destroying the newly introduced islets.
ViaCyte’s VC-01 product candidate, a cell replacement therapy could potentially transform the way individuals with T1D manage their disease by supplying an alternative source of insulin-producing cells with the potential to free individuals from a dependence on external insulin use. If the product candidate performs as intended, it has the potential to provide individuals who have T1D with a replacement for the beta cells lost or impaired as a result of their disease.
Beta cell encapsulation research is a high priority research area for JDRF because of its potential benefits for individuals with T1D. JDRF has provided substantial funding for multiple scientific research programs to advance discovery and development research in this area.
Date: July 17, 2014