News that a California-based company has been cleared to conduct a Canadian clinical trial for a new device that would eliminate daily insulin injections for people with type 1 diabetes quietly slipped in under the radar of most mainstream media outlets early this year. The implications, however, are considerable. Despite their widespread use in blood-based cancer treatments, stem cells have long seemed to inhabit a Disneyesque tomorrow land in which amazing things will someday be routine. Finally, though, the field is beginning to fulfill its potential and -Canada can lead the way if we assemble a strong private/public funding program, take some of the twists and turns out of the regulatory pathway, invest in the right technologies and align our world’s best scientists with those who can bring their work to the market.
The stakes are high. If we get it right, we can reduce human suffering from chronic, debilitating diseases, ease the burden on an overstressed health care system that is currently costing more than $200 billion a year, boost our economy by creating thousands of new high-skill jobs and own an area of advanced technology that was born in Canada. If we don’t, other countries will.
The diabetes example is a good one. The biologic device was developed by Viacyte, which takes scientific advice from the University of Alberta’s James Shapiro, one of the codevelopers of the Edmonton Protocol for implanting pancreatic islets. Currently being tested on patients at the University of California San Diego Health System, it is about the same height and thickness but half the width of a credit card and stores stem-cell-derived pancreatic progenitor cells that, once implanted, secrete insulin. If the device works, it will be life-altering for Canada’s 3 million diabetics and could reconfigure how diabetes, which affects almost 350 million people worldwide, is treated. It’s no longer a question of whether we can use stem cells to replace insulin injections; it’s a case of when we start doing it. That will likely be before the decade ends.
At the Ottawa Hospital Research Institute, Duncan Stewart leads a two-year, 100-patient clinical trial to test the effectiveness of using genetically altered stem cells to reduce scarring from severe heart attacks. Most major medical centres already have the capacity to do this kind of cell manufacturing, so if the trial shows significant improvements over current care, the procedure could be easily adopted to treat some 23,000 Canadians who suffer extreme heart attacks every year. Also in Ottawa, Harry Atkins has had good success in treating patients with multiple sclerosis and other autoimmune-related diseases with a bone marrow stem cell transplant protocol he refined. Now that he has treated about 30 patients, the procedure is ready for uptake in other centres.
As a result of our stem cell research we are on the verge of producing therapies for diseases that have long baffled medical science.
Derek van der Kooy, who led the University of Toronto team that discovered retinal stem cells, has predicted that the eye will be the site of the first big stem cell therapy breakthrough because it is an easy target for injecting cells and observing how they perform. Indeed, early stage clinical trials using induced pluripotent stem cells (adult cells reprogrammed to their original stem cell state) are underway in Japan, which has invested more than $1 billion in this kind of cell-based therapy and streamlined its regulations to move things along more quickly. The Americans are all in. The Viacyte project blossomed from California’s $3-billion investment in stem cells over 10 years through the California Institute of Regenerative Medicine, whose mantra is ”œturning stem cells into cures.” Everywhere, the focus has moved well beyond pure, basic research. In the United Kingdom, which already has a Strategy for Regenerative Medicine, the House of Lords has recommended Britain ”œtake action now” by streamlining regulations, enhancing support for clinical trials and backing innovative funding models to prevent falling behind the United States and Japan.
Canada’s role in all of this is vital. We have enormous scientific credibility, given that Canadians virtually founded the field: James Till and Ernest McCulloch proved the existence of stem cells more than 50 years ago at the Princess Margaret Hospital and went on to train and inspire subsequent generations of scientists who have positioned Canada at the leading edge. People like Tak Mak, who revealed the secrets of the T-cell receptor and is working on a new class of ”œsharpshooter” cancer therapies that kill only diseased cells, and John Dick, whose discovery of cancer stem cells triggered a worldwide rethink of how to keep tumours from coming back, trace their scientific lineage directly to Till and McCulloch.
We are well organized on a number of fronts. Over its 14-year run as one of Industry Canada’s Networks of Centres of Excellence program, the Stem Cell Network succeeded in creating a multidisciplinary and cohesive research community out of Canada’s far-flung but top-tier scientific talent " a significant advantage in the global race for improved health and prosperity. Strategic investments in other organizations, such as the Centre for Commercialization of Regenerative Medicine and, more recently, the CellCAN Regenerative Medicine and Cell Therapy Network, have positioned Canada to lead in the next wave. Our infrastructure, largely because of bricks and mortar investments through the Canada Foundation for Innovation, is suitable for conducting the kind of large-scale clinical trials required. Entrepreneurs have also stepped up their game. STEMCELL Technologies of Vancouver started out supplying tissue culture media to the nearby Terry Fox Lab. It now generates $100 million in annual sales, employs 650 people and conducts business in 80 countries.
What was lacking, however, is a strategy to pull all these assets and actors together with a bold vision and align the private and public investments required to make it happen. Realizing this, the Canadian Stem Cell Foundation led a year-long national dialogue with experts from the scientific community, medical professions, health charities, industry and the philanthropic sector on how Canada can follow through on its stem cell legacy. The goal was not simply to find a way to keep Canada a player in the stem cell game, but also to position our country to produce new therapies and create a successful cell manufacturing and commercial sector. That coalition devised a private and public partnership for Canada to lead the way in delivering 5 to 10 new cell-based treatments to the clinic within 10 years.
Called the Canadian Stem Cell Strategy and Action Plan, it has been endorsed by a panel of international experts headed by Harvard University’s George Daley, director of the Stem Cell Transplantation Program at Boston Children’s Hospital, and backed up by an in-depth study conducted by KPMG analysts. The strategy calls for $1 billion in private support over the next 10 years, leveraged through a scaled average annual investment of $50 million by the Government of Canada. Private sector support is lining up behind the Strategy, with STEMCELL Technologies prepared to invest $350 million in stem cell R&D over the 10-year span. Several philanthropists, health charities and at least two provinces are readying major investments, if the federal government commits.
The benefits to be accrued in terms of health and economics are considerable if we get organized and align investment. Canada will become a destination for clinical trials to safely test new cell-based therapies under rigorous and robust regulations and controls " instead of seeing desperate Canadians travel abroad to receive experimental treatments in countries with lax regulatory regimes. Canadians could then be first in line to get access to new stem cell treatments as they are proven safe and effective. Because stem cell-based therapies are aimed at curing the condition and not merely managing its symptoms, the introduction of these treatments could go a long way to easing the strain on a health care system that simply can’t continue bending without breaking.
Then there are the exports of cell-based technologies and biologics and the thousands of jobs that developing them will create: the Centre for Regenerative Medicine estimates the strategy would generate in excess of 12,000 jobs via the growth of existing companies and the creation of new ones. While establishing a vibrant stem cell/regenerative medicine sector may never outmuscle auto manufacturing, it will help diversify our economy and reduce reliance on volatile, resource-based revenue generation.
National pride is on the line, too. Canadians proved the existence of stem cells and have done much of the heavy lifting to get the field to where it is today: on the verge of producing new therapies for diseases that have baffled medical science for centuries. Like the science itself, Canada is in position to do great things. All we have to do is get it right.
Alan Bernstein is president and CEO of the Canadian Institute for Advanced Research and chair of the Canadian Stem Cell Foundation’s Board of Directors. He was the founding president of the Canadian Institutes of Health Research. Allen Eaves is president and CEO of STEMCELL Technologies Inc. and founded the Terry Fox Laboratory for Hematology/Oncology Research at the BC Cancer Agency. James Price is president and CEO of the Canadian Stem Cell Foundation.