Grant Helps ViaCyte Pursue T1D Cure
ViaCyte has long been at the forefront of stem cell research and regenerative medicine. And its aim has always been taking down type 1 diabetes (T1D).
In August the San Diego-based company began testing its PEC-Direct product candidate in clinical trials.
Two months later, the California Institute for Regenerative Medicine (CIRM) awarded ViaCyte a $20 million dollar grant to continue its cutting-edge research towards finding a functional cure for T1D.
“This has been our focus for a long time,” said ViaCyte president and CEO Paul Laikind, Ph.D. “We’ve been around over a decade and in that time we’ve been entirely focused on developing a cure for type 1 diabetes. We are grateful to CIRM and the citizens of California for their continued support of our potentially transformative stem cell-derived cell replacement therapies for diabetes.”
The funding will be used to offset costs already incurred by the development of PEC-Direct and to continue funding medical trials.
PEC-Direct presents a fascinating prospect to those with T1D: insulin independence and a functional cure. Each implant is about half the size of a credit card and carries healthy, insulin-producing cells built from harvested stem cells. Each device also has open ports. The device is implanted just below a patient’s skin. The cells mature inside the device over three to four months, and while the cells remain in the device even after implantation, the ports allow direct vascularization with a patient’s blood stream. When your body needs insulin, the cells release it. Furthermore, the product candidate is designed to not need supplementing: the plan is to provide enough cells to create just enough insulin to control glucose like a normal pancreas.
PEC-Direct is designed to treat only high-risk patients. In the first cohort of the clinical trial safety is being established.
“We’re initially dosing at a low dose to prove safety and evaluate the engraftment of the cells into patient’s tissue,” Laikind said. “We put in a smaller version of the product, which we call sentinels. We can pull those out periodically to evaluate how the cells are doing. When we move to cohort two we’ll put in a full dose of cells that are expected to be fully effective.”
Three to six patients are being enrolled in cohort one, while 36 to 40 patients will be enrolled in cohort two. Laikind said the company hopes to finish the preliminary efficacy studies by the middle of next year and demonstrate full product efficacy in early 2019.
While PEC-Direct is focused on just high-risk patients, many of who are facing immediate and potentially fatal complications and are candidates to receive a transplant with immunosuppression, Laikind is adamant that the company’s goal remains treating everyone with T1D.
The $20 million CIRM grant is the latest in a line of advocacy funding for ViaCyte and the largest of the company’s awards.
The company is relatively unique in that, while it has been funded by venture capitalists, a significant portion of its research and development funding has come from CIRM, non-profit JDRF and other non-profit grantors (including Beyond Type 1). With the latest CIRM grant, ViaCyte has raised about $90 million in project funding from investors and $85 million from JDRF and CIRM.
“In terms of focus on a single program or project I believe this is the largest funding CIRM has done,” said Laikind
CIRM was created in 2004 when Californians voted to pass Proposition 71: the California Stem Cell Research and Cures Initiative. When a CIRM funding application is made it goes before a panel of 15 grant reviewers. Each reviewer rates the proposal on a scale of one to three. One is a vote for full approval. Two is a rejection praised for significant merit and welcomed for resubmittal later. Three is a straight rejection with no option to resubmit. ViaCyte’s application, Laikind said, was unanimously approved, receiving 15 number-one ratings.
With the grant approval, ViaCyte is negotiating with CIRM to put together a budget and a payment schedule. While some of the payment will come upfront and be applied already incurred to the PEC-Direct costs, the remainder of the funding will be delivered on a performance basis if and when pre-set progress milestones are met on the project.
“Many people don’t appreciate how much of an impact type 1 has on patients,” Laikind said. “So many patients with T1D diabetes, especially many young people I meet, are overachievers. They do fantastic things. I think part of it is that T1D forces responsibility on patients over time. At the same time, some people look at it and think, ‘Oh they have type 1 diabetes but look how well they’re doing.’ When you look behind that screen you see a very, very difficult and very negatively impactful disease. We believe we are on a path to doing work that has the potential to change people’s lives.”
ViaCyte is a grantee of Beyond Type 1. Learn more about Beyond Type 1 grants.