ViaCyte, Inc., a privately held regenerative medicine company developing a stem cell-derived islet replacement therapy for the treatment of diabetes, called VC-01, has been selected to receive a $16.6 million Accelerated Development Pathway Award from the California Institute for Regenerative Medicine (CIRM).

According to CIRM, the Accelerated Development Awards were created to “provide selected applicants who are making rapid progress with additional resources and financial support to accelerate their stem cell-based therapy towards demonstrating evidence of an acceptable safety profile and clinical proof of concept.”

ViaCyte’s innovative stem cell-based therapy, which is initially targeting type 1 diabetes, has been supported by several prior rounds of funding from CIRM.  The financial and technical support from CIRM has played a crucial role in the development of VC-01, which was recently cleared by the US Food and Drug Administration (US FDA) to begin evaluation in human clinical trials.

ViaCyte’s VC-01 combination product candidate consists of pancreatic progenitor cells, called PEC-01 cells, derived from a proprietary human stem cell line, encapsulated in ViaCyte’s proprietary Encaptra device.  When implanted under the skin, the PEC-01 cells are designed to mature and further differentiate into insulin-producing beta and other endocrine cells that regulate blood glucose in a manner similar or identical to the islets that normally comprise the endocrine pancreas.

“Once again we are expressing our gratitude to CIRM and the citizens of California for supporting the work we are doing to develop a new approach for the treatment of type 1 diabetes and concurrently demonstrate the potential of stem cell-derived therapy,” stated Dr. Paul Laikind, president and chief executive officer, of ViaCyte.  “Today’s grant allows us to continue to accelerate our efforts as we move into the critically important stage of evaluating VC-01 in human clinical trials.”

ViaCyte recently announced the acceptance of its Investigational New Drug (IND) Application by the FDA, which allows clinical testing of the VC-01 product candidate to commence.  CIRM funding will help support the recently initiated phase 1/2 clinical trial in patients with type 1 diabetes, as well as additional clinical testing in the future.  JDRF, the leading global organisation focused on type 1 diabetes research, is also providing support for the VC-01 development programme.

The phase 1/2 clinical study will evaluate the VC-01 product candidate directly in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function.  In addition to evaluating the safety of the product candidate in these patients, the study is designed to demonstrate the effectiveness of the VC-01 product candidate in replacing the lost endocrine function that is central to the disease.  In an open-label, dose-escalating format, ViaCyte expects to enroll approximately 40 patients in the study at multiple clinical sites.

Type 1 diabetes mellitus (previously called juvenile diabetes) is a life-threatening chronic condition in which the pancreas produces little or no insulin, a hormone needed to allow glucose to enter cells to produce energy.  It is typically diagnosed during childhood or adolescence, though it can also arise in adults.  Though less common than type 2 diabetes, which occurs when the body becomes resistant to insulin, type 1 diabetes affects several million Americans, according to JDRF.  Currently, there is no cure for type 1 diabetes and the risk of long-term complications is high even with diligent treatment.  Standard treatment involves multiple daily injections of insulin and rigorous management of diet and lifestyle.