The regenerative medicine field, which includes cell and gene therapies (CGTs), is still abuzz with the fall approvals of three CGTs by the U.S. Food and Drug Administration: Novartis’ Kymriah, Gilead/Kite Pharma’s Yescarta and Spark Therapeutics Inc.’s Luxturna.
At the Cell and Gene Therapy World conference in Miami, Florida (January 22-25), many of the talks were either about the approved treatments or congratulating the industry on these significant milestones.
Reni Benjamin, of Raymond James Financial, reminded delegates that the pharmaceutical industry is also feeling confident about CGTs. Acquisitions in 2017 were worth billions: Gilead acquired Kite for $11.9 billion, Takeda bought Ariad for $5.2 billion and Roche acquired Ignyta for $1.7 billion. As the conference was just getting underway, the news was announced that Celgene is buying Juno Therapeutics for $9 billion.
Illustrating the future of the field are the more than 1,300 currently open clinical trials listing stem cells (from sources other than blood) as the primary therapeutic, the 1,000 clinical trials in gene therapy, and clinical trials involving chimeric antigen receptor (CAR) T cells (a type of immune system cell) accounted for around half of clinical trials in 20162.
As such, global investment in the CGT and regenerative medicine industry is booming. For example, public and private investment in immuno-oncology has grown to $1.5 billion2. When it comes to gene therapies, the forecast for the year 2025 ranges from $4.3 billion to $10 billion2 due to recent advances in the understanding of genetic disease, and innovation in genetic engineering tools. Altogether, it is estimated that the regenerative medicine industry will explode to a valuation of up to $20 billion by the year 2025.
Where does Canada sit in terms of being an innovator in these advanced therapeutic technologies?
Let’s start with the good news. Canada is a prominent force in this emerging global field. We have a strong backbone of Canada-based researchers who are recognized scientific leaders, and a robust system for the development of highly-qualified personnel through Canada’s universities. We have also benefited from strategic investments in research, collaborative networks and infrastructure, and are developing a deep understanding of how to translate these advanced therapies from the bench to the bedside.
One way to sustain Canada’s leadership position is to nurture the right skills and education within our borders. Encouraging STEM (science, technology, engineering and mathematics) education from a young age is a necessary first step. Extending STEM-based education with biomedical engineering programs at the university level is a good strategy for supporting the growth of Canada’s CGT and regenerative medicine industry. Biomedical engineering – where engineering design principles and mathematics are applied to medicine and biology, allowing students to make significant contributions to improving human health by finding new diagnostic or therapeutic solutions – is an area Canadian universities are increasingly focusing on.
An illustration of how biomedical engineers are already impacting the regenerative medicine field can be found at the University of Toronto’s Institute of Biomaterials and Biomedical Engineering (IBBME) and at Medicine by Design. The 55-year-old IBBME fosters a multidisciplinary research community where students and investigators in engineering, medicine and dentistry collaborate to develop innovative solutions that address global challenges in human health. Their impact can be seen in the development of breakthrough biomedical devices and new biomaterial products.
Funded in 2015 with a generous federal grant, Medicine by Design builds on IBBME’s successful multidisciplinary model to conceive, create and test strategies to address critical problems in regenerative medicine. By working across disciplines and generating and using emerging methods, like genome editing, computational modelling and synthetic biology, Medicine by Design is generating a deeper understanding of core biological concepts controlling stem cell fate, and devising new therapeutic approaches that will improve health outcomes.
This successful approach is now receiving a significant boost in Vancouver, where the University of British Columbia (UBC) has launched a new School of Biomedical Engineering as a partnership between the Faculty of Medicine and the Faculty of Applied Science.
“UBC’s School of Biomedical Engineering is cultivating and inspiring the future problem-solvers of health care,” says Dr. Dermot Kelleher, Dean of the Faculty of Medicine. “Biomedical engineers are helping to drive the industry forward and could hold the keys to commercializing new technologies and treatments. Creating this educational pipeline is critical as Canada continues to stake its claim as a leader in regenerative medicine, and as the full promise of this field comes to fruition.”
Moving from Education to industry
CCRM, a Toronto-based leader in developing and commercializing CGTs and regenerative medicine technologies, understands how the intersection of engineering and medicine, introduced by biomedical engineers, can help provide the tools that will advance the industry now and into the future.
“At CCRM, we recognize that highly-trained personnel, including biomedical engineers, are in high demand on a global scale as companies worldwide expand their regenerative medicine products,” says Michael May, PhD, president and CEO, CCRM. “CCRM aims to retain and attract the best and brightest who can create the technologies required to enable Canada to make a difference in solving the field’s commercialization challenges. In doing so, the Canadian regenerative medicine industry will be strengthened, creating opportunities for more research funding, a strong foundation for company creation, and stickiness for investors.”
One area where CCRM employs biomedical engineers is in its Centre for Advanced Therapeutic Cell Technologies (CATCT), a joint investment by GE Healthcare and the Government of Canada. Biomedical engineers work on process development strategies and solutions, and on projects involving reprogramming and engineering cells, immunotherapies and gene therapies. Operational for over a year, CATCT was created to accelerate the development and adoption of cell manufacturing technologies that improve patient access to novel regenerative medicine-based therapies. The team introduces new technologies to solve emerging technical challenges and closes gaps in current and future workflows.
Moving from industry to adoption
Our next challenge is to make certain that we have the people, technologies, processes and infrastructure to ensure Canadians have equitable access to these potentially game-changing therapies. Biomedical engineering programs are a start. Engineers are trained to look for efficiencies through cost reductions and improved technologies.
We need to build a Canadian innovation cluster that will attract talent and business expertise to capture the intellectual property developed in Canada and mobilize it for the benefit of Canadians.
We also need to work with government to position our health-care system as part of our competitive advantage. As Dr. May proposes, “If the provinces become early adopters of the medical products and therapies designed and tested in Canada, both patients and the economy would gain tremendously.” A big part of getting to this step in getting to this solution is starting to look at health economic models that integrate therapeutic costs and savings from development through to long term patent treatment costs.
Together, Canada’s companies, networks, researchers, start-ups and innovative centres are starting to deliver on the promise of regenerative medicine. With the technical know-how and a spirit of collaboration, biomedical engineers are a driving force in the country’s quest to lead the CGT and regenerative medicine industry into the future.
Professor Peter Zandstra is UBC’s Director of the School of Biomedical Engineering and Director of Michael Smith Laboratories. Zandstra’s research integrates engineering and biological approaches using computer modelling and strict control of the microenvironment (niche engineering) to develop a deeper understanding of the regulatory networks that determine stem cell fate.