Part 2 “We now know we can actually do that [with stem cells], so we have to figure out how to scale that up,” said David Prowten, CEO of the Juvenile Diabetes Research Foundation Canada, which has funded Sernova’s research.
Because the immune system attacks foreign objects, Sernova and several other device developers have focused on building implantable organ-like mechanisms to host and protect the cells.
Sernova’s device, called the “Cell Pouch,” is a business-card-sized implant inserted in a person’s lower abdomen. The polymer device is porous, so tissues fuse in and around it, forming an organic chamber and on-ramps to the bloodstream large enough to let in enough blood to gauge the body’s glucose levels, but small enough to keep out immune cells. Once tissues have wrapped around and inside the pouch, donated islets are transplanted into the chamber.
The hope is that the pouch can function for years without replacement or replenishment, Dr. Toleikis said. Sernova is also developing other uses to counter hemophilia and thyroid loss.
Sernova has generated data since the early 2010s, showing the device is safe in pigs and humans. But last Friday, principal clinical investigator Dr. Piotr Witkowski presented a key sliver of information from a continuing study, sanctioned by the U.S. Food and Drug Administration, of seven people with a severe form of diabetes to an American Society of Transplant Surgeons symposium.
Dr. Witkowski reported that two patients who had islet transplants had shown “defined clinical benefit with a clinically meaningful reduction in daily insulin injections,” including an absence of serious hypoglycemic events. Blood levels indicated insulin from the cell pouch was getting into the blood. One patient was able to go without an insulin shot for nine months, although that person also gotanother islet transplant into the liver similar to what U of A researchers have done for years.
“If they can reproduce this in other patients, this is an incredible result,” said Paradigm Capital analyst Scott McAuley.
But the small number of patients, the need for multiple infusions and the use of immunosuppressants in the study mean the early results “are a positive step, but it’s not exactly at the edge of a major breakthrough,” said Dr. Breay Paty, a clinical associate professor at University of British Columbia who used to work with the U of A group. “They’re showing in principle the pouch works, the cell survives and that they produce insulin. That’s promising and positive, but I don’t see it as earth-shattering.”
But Dr. Toleikis is buoyed. If the study is successful, he expects the FDA to green-light another, expanded study. Sernova has raised $50-million to date and hopes to pursue a Nasdaq listing. Dr. Toleikis believes regulatory approval could come by 2025 “if everything goes well and moves smoothly,” he said.
Sernova is also developing coating technology that could further protect transplanted islets from immune cells.
Meanwhile, Dr. Toleikis is working to fill a gap in Sernova’s treatment by securing a steady, scaleable source of islets. He admits Sernova is behind in efforts to develop its own stem cells, so he’s pursuing partnerships with pharmaceutical companies to provide the material.
“You get a licensing deal like we’re working really hard to get and, all of a sudden, the stock will start taking off again,” he said.