Stem Cell & Regenerative Medicine Research: A Canadian Science & An Innovation Winner

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1 Stem Cell & Regenerative Medicine Research: A Canadian Science & An Innovation Winner Author: Dr. Michael Rudnicki, OC, FRSC Scientific Director & CEO, Stem Cell Network Director, Sprott Centre for Stem Cell Research, Ottawa Hospital Research Institute Important advances are being made every day in Canada by innovators who work in fields such as Information and Communication Technology (ICT), manufacturing, engineering, agriculture and the health sciences. Innovation, while not always linear, often starts with science and technology, where Canada has clearly defined areas of strategic strength. As we consider what an national innovation agenda should look like, I hope that we will not forget that at the core we must continue to nurture our science culture while providing a stable and supportive environment for research and discovery. More than 50 years ago, Canadian researchers Drs. James Till and Ernest McCulloch first proved the existence of stem cells; their seminal studies launched the international field of stem cell research. Stem cells have the unique ability to develop into any cell and repair damaged and diseased tissues or organs. They hold great promise for the development of new therapies and treatments for diseases such as heart disease, diabetes and multiple sclerosis diseases that take Canadians out of the workforce and out of active economic participation. Canada continues to lead in stem cell research and the time is now ripe for strategic support and investment. Moving Forward: The Health & Economic Imperative Today, innovative countries such as Japan, the U.K. and the United States are all vigorously pursuing stem cell research and development in the hopes of leading the field globally. Like Canada, they recognize the power stem cells hold for improving health and lowering costs associated with the treatment and care of chronic diseases including economic loss from workplace under-participation. In Canada, chronic diseases absorb 67 percent of all direct healthcare costs and their burden on the health-care system continues to outpace economic growth. Treating chronic disease costs a staggering $68 billion a year, 3.6% of GDP. However, this is a story that will change, as stem cell based treatments and therapeutics begin to enter the market place and are more commonly available to clinicians and patients. Estimates suggest today s total global stem cell and regenerative medicine market, even in its infancy, is approximately $3.8 billion. With an annual growth rate of 21%, it is projected to surpass $20 billion by Canada is well positioned to excel and compete in this market in the years ahead.

2 Stem Cell Research & Global Science Excellence In March 2016 the federal government announced its intention to provide $800 million for innovative networks and clusters, starting in In stem cell and regenerative medicine research, Canada already has a strong, emerging and innovative national network to build on, with the combined expertise of the Stem Cell Network (SCN) and partner organizations such as the Centre for the Commercialization of Regenerative Medicine (CCRM), the Centre for Drug Research and Development (CDRD), the Ontario Institute for Regenerative Medicine (OIRM), CellCAN, Thé Cell, and Medicine by Design. SCN and its partners have developed an integrated, end-to-end national cluster that has the potential to compel economic growth over the next decade and make a real difference in the lives and health of Canadians. In 2001, SCN supported 50 research groups across Canada. That number increased over time, and today stands at almost 150 research groups from coast to coast. In-depth studies of Canada s S&T strength show that Canada s stem cell and regenerative medicine sector is highly ranked for international collaboration and impact of scientific output (CCA, State of S&T, 2012) Consequently, Canada is well positioned to capitalize on the value of our global leadership in stem cell and regenerative medicine. Now is the time to build on the outstanding scientific strength that exists across Canada. Today, Canadian investigators are conducting world leading studies that have high potential to significantly impact patient care in the near future. For example: Dr. Tim Kieffer and his team at the University of British Columbia have developed a protocol that can turn stem cells into insulin-producing cells in a matter of weeks. As a result, we are one step closer to having an unlimited supply of insulin-producing cells to treat patients with Type 1 diabetes. Dr. Kieffer s research has been supported by SCN throughout the years; he is one of the many health research superstars that Canada can claim as its own. 2

3 Dr. Freda Miller from the University of Toronto, also supported by SCN, is best known for proving skin can be a viable source of stem cells. This ground-breaking discovery opened the door to learning how to treat injured nervous systems, more specifically spinal cord injuries. Today, Dr. Miller and her team are conducting a promising clinical trial using the diabetes drug metformin to stimulate brain regeneration in children following treatment for brain tumors. The results are very promising and offer hope for timely clinical application. Drs. Harry Atkins and Mark Freedman from the Ottawa Hospital Research Institute had their major breakthrough for treating early, aggressive forms of multiple sclerosis published in The Lancet this June. The procedure uses stem cells and chemotherapy to eradicate multiple sclerosis (MS) in a small subset of patients. This paradigm shifting approach is anticipated to become the new standard of care internationally, and with more research could benefit a larger population of MS patients. Canadian stem cell researchers are not only making important discoveries, they are also moving their findings into products and services to meet global demand and need. For example, Montreal and Toronto-based ExCellThera, started by two SCN investigators, is growing blood stem cells for therapeutic use, ensuring that greater numbers of cancer patients have access to blood stem cell transplantations. Vancouver-based STEMCELL Technologies employs more than 800 highly skilled workers (the majority based in Vancouver) and offer more than 2,000 cell biology research tools. Aspect Biosystems, also located in B.C., is an award winning biotech company specializing in 3D bioprinting and tissue engineering. Aspect s highly skilled team is currently partnering with SCN investigators to determine a suitable and stable bioink formulation for printing specialized stem cells (hipsc-derived neural progenitors). Once developed, the bioink could be used for printing physiologically relevant neural tissues. These three companies are just the tip of the iceberg; there is significant opportunity for further stem cell-based treatments, products and technologies to come to market. An emerging area of demand is for facilities that focus on cell-therapy manufacturing. Canada has seven large cell therapy manufacturing centres, each led by an SCN researcher two in Montreal and one in each of Quebec City, Toronto, Ottawa, Edmonton and Vancouver. These facilities are advancing the translation of stem cell research into therapies and treatments. Canada is also well positioned for drug discovery with multiple high-throughput screening facilities, some with medicinal medicine capabilities, across Canada including Montreal, Toronto, Hamilton, and Vancouver; several are also led by SCN researchers. While other countries, such as the U.S., are vying to become the leading global providers of cell and drug therapies, Canada 3

4 is well positioned to establish and maintain a strong global position and is already active in the field. Supporting cell-manufacturing and drug-screening facilities, innovative biotech companies and research establishments requires skilled workers of the highest caliber. SCN has played a critical role in training the next generation of multidisciplinary stem cell and regenerative medicine researchers. Trainees have been able to learn SCN s 2,500 trainees have the STEM skills that will be critical in the years to come as S&T advances are made, resulting in new labour markets & economic opportunities. about the latest scientific advances, how to identify research findings that have commercialization potential, and how to pursue clinical trials and ensure their goal directed work moves from bench to bedside. These are the individuals whose STEM skills will be critical in the years to come as scientific and technological advances are made, resulting in new labour markets and economic opportunities. If Canada is to maintain and/or build upon the global science excellence that exists across Canada then stable and sustained investments are required. Such investments will allow for: Leadership by Canadian scientists in ground-breaking research, including clinical trials; Development of novel IP, products & therapies, and the creation of innovative biotech companies; Training for young and early career investigators working to establish their research pursuits in academia or industry; and Attracting the best and brightest to Canada while retaining Canadian scientific talent. Research with Impact Approximately 100,000 Canadians suffer from septic shock every year. It accounts for 20 % of all ICU admissions and costs the health-care system roughly $4 billion each year. Dr. Duncan Stewart & Dr. Lauralyn McIntyre from the Ottawa Hospital Research Institute are leading the world s first clinical trial using mesenchymal stem cells to treat septic shock. The trial is highly promising and is already helping to save lives. The trial is based on research funded in part by SCN. The SCN supported research developed the therapy upon which the clinical trial is based, and supported pre-clinical testing for safety and effectiveness. SCN funding was also provided to optimize the manufacturing of clinical-grade stem cells for use in the clinical trial and for other illnesses where cell-based therapies have potential benefit. There are numerous sectors with the potential to be innovation winners for Canada. However, no other health science cuts across almost all disease areas like the stem cell research sector. This is a field that is seeing important health breakthroughs, generating novel IP, developing cuttingedge technologies and spurring company creation. SCN supported research has led to 4

5 approximately 570 patent applications, 93 issued patents, and more than 60 licenses granted, while SCN supported intellectual property has catalysed the growth or launch of approximately 11 start-up biotechnology companies. Previous investments in stem cell research, through the Stem Cell Network, have enabled SCN to facilitate the formation of multidisciplinary teams of investigators, who have established an internationally-recognized track record in bringing scientific discovery through translational development in a very short period of time. The economic and health benefits arising from advances in this emerging field are predicted to be enormous, especially for the treatment of diseases for which there is currently no effective therapy. CCRM has provided a separate submission to the consultation process on the innovation agenda, which addresses a national commercialization strategy for regenerative medicine. CCRM serves as important commercialization resource, particularly for stem cell researchers who are looking to move their research discoveries into the market place. Insights & Conclusion: Insights for the Innovation Agenda: Stem cell research and regenerative medicine are disruptive technologies that will transform the practice of medicine. Canada is a global leader in stem cell and regenerative medicine research. A national cluster of strength in regenerative medicine currently exists in Canada and will continue to grow over the coming decades, with more biotech companies starting up, and growing collaboration amongst academia and industry. The potential outcomes from stem cell and regenerative medicine research will benefit millions of people around the world who suffer from chronic diseases and debilitating illnesses. Sustained funding for stem cell research will catalyze significant long-term economic and health benefits for Canada. Stem cell science was pioneered in Canada and I am honoured to have led the Stem Cell Network, which has forged a national community over the past 15 years. The strength of the sector could not have been achieved without the strategic and financial support of the government. Today, stem cell research has reached the tipping point where important advances and breakthroughs will transform the practice of medicine. With strong and sustained support across the sector, Canada is poised to capitalize on previous investments and build upon a Canadian success story. Strategic investment through the innovation agenda will catalyze a 5

6 leading health innovation sector, and in-turn, allow the realization of real economic and health benefits for Canada. 6

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