Barrie McKenna is a former Globe and Mail columnist and author of the Innovation Economy Council white paper Out of the Lab, Into the Patient: Canada’s Commercial Opportunity in Cell and Gene Therapy Manufacturing.
Massimiliano Paganelli, a pediatrician specializing in liver disease, watched for years in frustration as half the children with acute liver failure died waiting for transplants.
So, as head of the liver tissue engineering and cell therapy lab at Montreal’s Sainte-Justine children’s hospital, Mr. Paganelli in 2018 teamed up with his stem-cell biologist wife Claudia Raggi to develop a life-saving alternative. The result is Morphocell Technologies Inc., a Montreal-based startup company that aims to start clinical trials and get its cell therapy into patients within two years.
Morphocell is part of a new frontier in life sciences. Hundreds of promising cell and gene therapies are in various stage of development and commercialization in Canada and around the world targeting previously untreatable illnesses, including arthritis, diabetes, Alzheimer’s, numerous cancers and various rare conditions. And unlike many conventional drugs, these living therapies can be engineered to directly attack and reverse the course of disease, rather than to simply treat the symptoms.
A new Innovation Economy Council (IEC) report says Canada has the potential to create a multibillion-dollar industry built around cell and gene therapies. But to do that, the country will need to vastly expand its capacity to manufacture treatments, conduct clinical trials and train a large, skilled work force.
“This is the future of medicine,” says Michael May, president and chief executive of the Centre for Commercialization of Regenerative Medicine (CCRM) in Toronto. “If we want Canadians to have access to these life-saving medicines, then we need to be in the game.”
Thanks to pioneering Canadian stem-cell research in earlier decades, Canada is already a hotbed of discovery in the field of genomic medicine. Now, dozens of companies are transitioning from drug development to clinical trials and eventually full-scale manufacturing and commercial sales.
Cell and gene therapies are expected to be the backbone of global health care in the decades ahead. Regardless of what happens, Canadian taxpayers, insurers and patients will be paying for them. And as has previously been the case with vaccines, many of these treatments will be made abroad, depriving Canada of a vast economic opportunity. Canada can come out ahead by creating a major new industry that will generate broad economic gains, including creation of new startups, tens of thousands of high-paying jobs and lucrative exports, according to the IEC report.
CCRM, a public-private partnership, has already helped see and nurture nearly a dozen promising cell and gene therapy companies, including Morphocell. Combined, they employ 250 people and have attracted nearly $800-million in venture capital. The organization is now working to fill a much-needed industry gap by building the country’s first large-scale contract biomanufacturing plant for cell and gene therapies. Groundbreaking on the first phase of the 400,000-square-foot plant is expected this year at McMaster Innovation Park in Hamilton.
The plant will directly employ more than 1,000 people and it has the potential to be a powerful economic engine far beyond its own walls. An economic analysis commissioned by CCRM estimates the plant will create and preserve thousands of Canadian biotech jobs, lure foreign companies to Canada to develop, test and manufacture therapies, as well as become a magnet for investment. Projections show the project will generate $4-billion a year in GDP by 2032.
Getting there hinges on solving some significant manufacturing hurdles. Living therapies are inherently labour-intensive to make, and therefore costly. Producing cell and gene therapies requires sterile clean rooms, lab space, stringent quality control, expensive equipment and highly trained technicians. A gene therapy for treating a rare childhood neuromuscular disease – made by Swiss drug giant Novartis and approved for use in Canada – costs as much as $2.8-million a patient.
One way to overcome the cost and complexity is to refine the process of engineering and mass producing disease-killing cells from donors. For example, Vancouver-based Notch Therapeutics is refining the production of universal immune enhancing T-cells that combat various cancers.
“Once you set it up you should be able to have cells for everyone, at economies of scale,” explains Kamran Alam, Notch’s executive vice-president and chief financial officer. “In many ways, it’s democratizing cell therapy.”
It’s a given that Canadians will want access to the best life-saving therapies available anywhere in the world. But the best health care need not come at a prohibitive societal cost.
If we build a homegrown industry to mass produce and commercialize cell and gene therapies, Canadians can also enjoy the vast economic benefits that come from being in the game.
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