ONTARIO—Following a year of promising milestones, the team leading the clinical trial of Sernova’s Cell Pouch System to potentially modulate diabetes is reporting positive first-round results. The current protocol involved implanting a pouch subcutaneously in a patient until it is naturally absorbed into the surrounding tissues and vascularizes—meaning blood vessels flow through the pouch as regular tissue—when islet-rich cells are introduced. The preliminary data from the first patient to receive islet cells transplanted into the Cell Pouch was presented at the 17th World Congress of the International Pancreas and Islet Transplantation Association (IPITA). Their highlights included:
- No incidences of adverse events determined to be related to the Cell Pouch implant;
- The Cell Pouch was well incorporated with vascularized tissue, which enabled successful transplant of the purified islets;
- A weight reduction of 6.35kg (12 percent total body weight);
- Stabilizing improvements in all glycemic control parameters as indicated by Continuous Glucose Monitoring (CGM);
- 87.5-percent reduction in hypoglycemic events from baseline collected over a two-week monitoring period; and
- Presence of stimulated blood levels of C-peptide and insulin at the observed 90-day post-transplant point as indicated in a mixed meal tolerance test.
“We are very encouraged by glucose stimulated blood levels of C-peptide and insulin in the blood of [the first] subject and improvement in a number of important blood sugar control parameters,” states Dr. Philip Toleikis, president and CEO at Sernova. “This shows that the Cell Pouch is the right technology for housing and demonstrating function of the therapeutic cells within the Cell Pouch.”
Sernova describes the pouch as a potential “game-changer in diabetes management,” because it will theoretically provide “a natural environment in the body for the housing and long-term survival and function of therapeutic cells.” These cells could then replace key functions that might be missing in a person with a chronic disease, minimizing the impact while potentially reducing the need for ongoing pharmaceutical intervention.
“Over 10 years ago, we began on our quest for a better treatment for diabetes,” says Toleikis. “We knew that replacement of the cells that have been lost in the pancreas in people with diabetes would be the ultimate cure. We knew that these cells would need to be housed within a medical device placed under the skin that created a highly vascularized environment. That stimulated our biologists, device engineers, surgeons and immunologists to work together to develop the Cell Pouch. After much research identifying the parameters of the device that would be biocompatible and would encourage development of an organ-like environment, we developed our technologies and began testing to optimize the device design. After many proof-of-concept studies validating the technology, we began [these] human clinical studies.”
The Cell Pouch is manufactured from medical-grade materials, previously approved by the FDA for permanent use in the body in other products. Once the pouches are implanted and vascularized, following immunosuppressant treatment, the protocol involves implanting purified islets directly into the pouch. The transplanted islets naturally produce insulin regulated by biocommunication between insulin and glucose, in the hopes of eliminating the need for insulin injections.
“I am quite pleased with these interim results in the first implanted patient,” noted Dr. Piotr Witkowski, director of Pancreatic and Islet Transplant Program at the University of Chicago and the study’s principal investigator. “The first of two doses of islets transplanted into the Cell Pouch is showing safety and early indicators of efficacy. Importantly, demonstration of glucose-stimulated C-peptide and insulin present in the bloodstream is definitive proof of islet survival and function in the Cell Pouch. These results are an important first step towards a paradigm shift in the treatment of this debilitating disease.”
According to Toleikis, the Cell Pouch shows promise for both type 1 and type 2 diabetes, as well as potential efficacy in other clinical indications such as chronic pancreatitis, hemophilia, thyroid disease and possibly modified for treatment of rare diseases where a protein or hormone is required to treat the disease.
“Regarding outcomes,” Toleikis comments, “improving the quality of life for people with hypoglycemia unawareness through significant reduction of hypoglycemic unawareness events would be important; however, additional measures of efficacy in terms of glucose control would add to the benefit of this technology ... We see a potential approval of this product within three to four years, pending our discussions with regulatory authorities and ongoing positive results from advancing clinical evaluation. Sernova is thrilled to see the benefits of the many years of work with positive results in treating this disease in humans in a way that could significantly improve the quality of life for these people, something that families with diabetic children have been requesting for over 100 years.”