“We are really just at the beginning of the revolution. We know over the next decade cell and gene therapies are going to fundamentally change how we treat disease.” — David Llewellyn, Chief Strategy Officer, Business Operations, STEMCELL Technologies
One highlight of last month’s Access to Innovation event, hosted by Life Sciences BC, was a panel on the promise of cell and gene therapies. Here are our notes.
CAR-T
CAR-T has shown success in clinical trials in leukemia and lymphoma (with remission response rates as high as 90% for certain types of leukemia). However, commercial products approved by the FDA and Health Canada remain enormously expensive, with an average price tag of several hundred thousand dollars per patient.
In 2016, a group of Canadian researchers—including Dr. Brad Nelson, a world-renowned immunotherapy researcher with the BC Cancer Agency in Victoria—came together to see if they could create a made-in-Canada version of CAR-T. The group launched a clinical trial in 2019 targeting the CD19 protein for patients with specific kinds of leukemia and lymphoma. Latest results show 43% of participants have achieved complete remission. Now the team is entering into a second clinical trial targeting the CD22 protein and is also developing a therapy for solid tumours, like ovarian and pancreatic cancers.
iPSCs
Another area where B.C. researchers are making a significant impact is induced pluripotent stem cells (iPSCs).
This new avenue of stem cell research—where cells are sourced from an adult patient rather than embryos—has opened significant doors in regenerative medicine. Dr. Peter Zandstra, founding director of UBC’s School of Biomedical Engineering, is at the forefront of this research. He also helped found ExCellThera, a world leader in blood cell expansion, and Notch Therapeutics, which is building best-in-class T cell therapies from iPSCs.
Zandstra said the field is going in two different directions. The first is making a therapy like a pill, which requires a renewable source of cells that can be engineered and differentiated into specific products. The second is bringing the manufacturing closer and closer to the patient, until eventually the manufacturing is done within the patient.
“I think we are going to see big changes and advances in the field in the next five years that will really give us the opportunity to think of this as another pillar of medicine,” said Zandstra.
Bioprinting
Meanwhile, Vancouver-based preclinical startup Aspect Biosystems is leading the charge on bioprinted tissue.
Sam Wadsworth, Chief Scientific Officer, discussed the company’s unique bioprinting technology that combines biomaterials, therapeutic cells and implantable therapeutic tissues. The results can be applied to a variety of different diseases caused by the loss of a specific cell type or function.
An example of the power of this approach can be seen with Type I diabetes, an autoimmune disease that leads to the loss of pancreatic islets. Patients lose the ability to respond to blood glucose changes and release insulin. Aspect is working to print implantable tissue with allogeneic pancreatic islets, allowing cells to respond appropriately to glucose.
Armed with promising preclinical data, one of the largest partnerships in B.C. history, and one of the largest financings, Aspect is hoping to be able to treat its first patients in a few years.
Regulatory environment
As more B.C. companies come forward in the cell and gene therapy space, regulation needs to catch up with the science.
Jennifer Solomon, Senior Director, Clinical Programs, at STEMCELL Technologies is a key opinion leader in the regulatory environment. Her leadership includes several white papers for the FDA, Health Canada and other bodies to understand how to regulate the space and ensure quality and safety for patients.
One of the biggest areas she is focused on is guidance and standards around the use of raw materials in the manufacturing of cell and gene therapies, which will continue to be a large focus as these therapies move forward.
Looking forward
What does the future hold? As Zandstra suggested, perhaps the current shift in the scientific sector offers an opportunity for further Canadian leadership in this field.