UBC and Vancouver Coastal Health (VCH) have received $1 million to conduct research and a clinical trial aimed at developing a diabetic’s ability to produce their own insulin.
“This has the potential to transform the management of Type 1 diabetes, hopefully eliminating the need for daily insulin injections and overcoming the drawbacks of previous cell replacement therapies,” said study leader Dr. David Thompson, a clinical assistant professor of endocrinology at UBC and director of VCH’s Vancouver General Hospital Diabetes Centre.
“I’m hoping in 2023 somebody will be off insulin,” he said.
Canadians providing breakthroughs in diabetes treatment isn't new.
It was Canadian surgeon Dr. Frederick Banting and medical student Charles Best of the University of Toronto, who successfully isolated insulin for the first time in 1921.
On Jan. 11, 1922, 14-year-old Leonard Thompson (no relation) was the first person to take insulin for diabetes.
Now, some 300,000 Canadians live with Type 1 and must have daily insulin injections in order to live.
Researchers said the funds from Canada’s Stem Cell Network would be used for one of the world’s first genetically engineered cell replacement therapies for Type 1 diabetes.
In December, UBC and VCH researchers released a first-of-its-kind study demonstrating a treatment for Type 1 diabetes involving stem cells. They called it a major breakthrough.
Now, they said, the investigative treatment, developed by biotechnology companies CRISPR Therapeutics and ViaCyte, is designed to enable Type 1 diabetes patients to produce insulin through a small medical implant containing millions of pancreatic cells derived from CRISPR gene-edited stem cells.
The situation has not been without its hurdles. Once the cells are implanted, the immune system treats them as a foreign invader and begins to attack the implanted cells.
To overcome this, patients are prescribed long-term immunosuppressive drugs, which come with a host of potentially harmful side effects, such as increased risk of infection.
“It was great that we could reduce people’s dependence on insulin, but it was almost like trading one disease for another,” Thompson said.
The ability to do a transplant of such cells without an immuno-suppressants could be a revolution in transplants generally, he added.
By engineering the cells, the researchers hope the cells will be able to evade detection and destruction by the immune system.
“This is the next step toward a potential functional cure for Type 1 diabetes. I’m hoping within the next few years somebody will be using the device to live their life free from daily insulin injections and free from immune-suppressing drugs.”
The UBC and VCH team will begin research and a clinical trial for the new treatment later this year. They are one of multiple sites working with CRISPR Therapeutics and ViaCyte to study the safety of the investigative therapy.
Thompson said there will be a safety study, with 40 to 50 trial participants in Vancouver, Edmonton and Toronto.
Other stem cell uses
Thompson hopes genetically engineered stem cells will have implications beyond diabetes, helping address a range of conditions: from heart damage to liver disease.
“Theoretically, you could apply this same gene-editing technology to heart cells, brain cells or liver cells, opening up a range of possibilities for cell replacement therapies,” he said.
A further benefit is how accessible the treatment could be for a broad range of patients. Because the stem cells are grown in a lab, there are opportunities to quickly scale-up production.
“We have the potential to produce a virtually unlimited supply of gene-edited stem cells,” said Thompson. “We could theoretically make these treatments available to people all across Canada and around the world.”