BETHESDA, MD (March 1, 2014) — Well, there you go again. Getting your hopes up for another possible ” cure” for Type 1 Diabetes. That’s what I told myself as I listened to Dr. Eugene Brandon, Ph.D. — an obviously intelligent scientist who was also able to speak in layman’s language — present his case at the JDRF Research Summit hosted by the Greater Chesapeake & Potomac Chapter of JDRF last Saturday.
As I listened and tried to understand the science behind his talk, it was difficult to stay realistic. If they can implant regenerative cells under the skin and these cells will function like healthy pancreatic cells, then this crazy idea just might work for some children or adults living with T1Ds. So my next question was: When is the first human clinical trial and how do I sign up? Damn the risks; my time is running out. That’s what went through my mind and probably a few others listening to him speak.
Before I booked my flight to San Diego, I settled down and realized one simple fact: I’m attending a diabetes research summit where “hope” is always the key word. Dr. Brandon, Director of Strategic Relations & Project Management at San-Diego based ViaCyte, spoke to a full ballroom of T1Ds and their families at the Bethesda North Marriott during the 4th annual JDRF Research Summit. Dr. Brandon talked about his company’s VC-01™ combination product. It is a stem cell-derived, cell therapy product that the company believes could transform the way patients with Type 1 diabetes manage their disease.
The product is comprised of pancreatic progenitor cells contained in a proprietary device that is designed to be inserted under the skin. Upon implant, the product is expected to vascularize as the cells further differentiate to islet-like structures that generate insulin and other expected regulatory factors in response to blood glucose levels, essentially providing patients with a replacement for the cells lost as a result of the disease. The company has reviewed the VC-01™ combination product development plans with regulatory authorities at the US Food and Drug Agency and Health Canada.
By acting essentially as a replacement endocrine pancreas, the source of insulin and other regulatory hormones produced in our bodies, VC-01 combination product has the potential to be a virtual cure for type 1 diabetes. The VC-01 therapy is the combination of:
Pending regulatory authority review of its planned application, ViaCyte is planning to initiate clinical development in patients with Type 1 diabetes this year! As he finished his presentation, Dr. Brandon answered a few of the lingering questions from the optimistic but realistic Type 1 Summit attendees.
The testing has worked in mice. But is it safe for humans?
“By all accounts, it is a stable product. Our testing shows it stays stable for the life of the animal,” Brandon said. “If we can get this biological process to work, we think we can replace the damaged islets.” The company also has developed a process for inserting and removing the implanted device quickly. “Something like this has never been tested in humans before.”
When will human clinical trials begin?
“We think we’ll be in pretty good shape to get this ready and approved by FDA for a first human trial planned in 2014,” Brandon said, adding that his company has already held discussions with the FDA on their proposed timeline. “You don’t want to spring something like this on the FDA.”
What is the lifecycle of the implanted device?
“That is the million dollar question to be determined in the clinical trial,” Brandon said. “How long will this last if it works?” Because the cells are contained in a ” tea bag” type of container, it is anticipated that the body’s immune system will not strike or reject the foreign object. “Theoretically, these implants could last for many years. That is the purpose of the clinical trials. We will start learning things that can only be discovered in a human clinical trial.”
At the end of the summit, I left with renewed hope for a cure and the sober realization that this chase for a miracle is nothing new to persons living with all types of deadly diseases. In fact, the JDRF was formed 40+ years ago by parents of children with Type 1 diabetes who were committed to pushing faster to fund a cure. In seeking a cure, we all jump into the fountain of scientific hope. Compared to the quality of life for diabetics before the discovery of insulin in 1921, Type 1 diabetics are living in a golden age of scientific and technology success. Things are improving at a rapid pace. So we keep chasing the illusion and hope to cheat our destiny for one more hour, for one more day, for one more year. Until there’s a cure … we march on.