Researchers from the UBC Faculty of Medicine and Vancouver Coastal Health (VCH) have received $1 million from Canada’s Stem Cell Network to conduct research and a clinical trial for one of the world’s first genetically engineered cell replacement therapies for type 1 diabetes.
The researchers say the study is an important step toward developing a potential functional cure for type 1 diabetes.
The novel 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 that contains millions of pancreatic cells derived from CRISPR gene-edited stem cells.
“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 therapies,” says Dr. David Thompson, a clinical assistant professor of endocrinology at UBC and director of VCH’s Vancouver General Hospital Diabetes Centre who is leading the study. “I am very grateful to the Stem Cell Network for supporting this important work.”
Aiming to free patients from daily insulin injections
More than 300,000 Canadians live with type 1 diabetes and are required to inject daily infusions of insulin in order to live. In a major breakthrough last year, Dr. Thompson and his team published a first-of-its-kind study showing that a stem cell-based treatment can produce insulin in the human body.
However, such cell replacement therapies have traditionally faced a major hurdle — once implanted within the body, 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,” says Dr. Thompson.
The new investigative treatment will use cells that have been genetically modified using CRISPR/Cas9 gene-editing technology — a Nobel Prize-winning technology that can be harnessed to modify, delete or correct precise regions of DNA. 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, which would allow people to live their lives free from daily insulin injections and free from immune suppressing drugs,” says Dr. Thompson.
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.
“This is another great example of how UBC and VCH researchers are working with private sector partners to accelerate the translation of research into potentially life-changing treatments,” says Dr. Rob McMaster, vice dean, research, at the UBC faculty of medicine. “It’s these kinds of collaborations that are strengthening B.C.’s bio-innovation ecosystem and helping to bring new treatments to patients sooner.”
“Research is an integral building block of our provincial and national diabetes framework,” says Dr. David Granville, executive director, Vancouver Coastal Health Research Institute. “We are incredibly grateful to Dr. Thompson for his achievement and commitment to transforming the lives of people with type 1 diabetes. This funding support offers a cause for optimism towards reducing the impact of diabetes on individuals, communities and the health care system.”
The limitless potential of stem cell therapies
Dr. Thompson is hopeful that the 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,” he says.
Another 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.
“This is the next step toward a potential functional cure for type 1 diabetes, which would allow people to live their lives free from daily insulin injections and free from immune suppressing drugs.”
Dr. David Thompson
“We have the potential to produce a virtually unlimited supply of gene-edited stem cells,” says Dr. Thompson. “We could theoretically make these treatments available to people all across Canada and around the world.”
While it has been 100 years since Canadian scientists discovered insulin as a treatment for type 1 diabetes, Dr. Thompson says local scientists are once again on the forefront of the next big breakthrough.
“Much of this work has been built off of the pancreatic cell transplant program that we started at UBC and VCH back in 2003,” adds Dr. Thompson. “We have built tremendous expertise here in Vancouver for everything from basic science through to clinical translation. B.C. is leading the way and we just keep getting stronger and stronger.”