Canada’s regenerative medicine sector poised to soar with better funding and collaboration: report

Canadian expertise and achievements in regenerative medicine (RM) could be at a turning point if strategic steps are taken to increase stable funding and achieve greater coordination among the many federal and provincial players. Those are the key observations of a report issued by the Council of Canadian Academies (CCA), which was based on a two-day workshop held last October to provide policy makers with pointers for growing the sector and realizing greater economic and health benefits.

The workshop and report —Building on Canada’s Strengths in Regenerative Medicine — were commissioned by Innovation, Science and Economic Development (ISED) and Health Canada to better understand the current state of RM in Canada. They found that Canadian-based RM researchers are highly collaborative global leaders, but they require “stable strategic investment in researchers, collaborative networks, and infrastructure” to “accelerate the translation of stem cell research discoveries to bedside and industry”.

Optimism in the sector was further fueled with the US $225-million investment in Toronto-based start-up BlueRock Therapeutics. – announced in December as the report was being written. The investment by Bayer AG and Versant Ventures represents the second largest series ‘A’ financing of a biotech company in history and a signal that Canada punches far above its weight in the RM field.

“BlueRock is an unprecedented level of investment for this field in Canada. It’s an important signal to the sector that the rate of discovery and invention is as high in Canada as it is anywhere,” says Dr Michael Rudnicki, a participant in the RM workshop and director of the RM program at the Univ of Ottawa’s Sprott Centre for Stem Cell Research. “In the past, a weak ecosystem and low levels of venture capital often meant that discovery left the country. But we’re maturing and this could begin to snowball and go to the next level.”

Dr Janet Rossant, chair of the CCA steering committee and workshop, agrees the scale of the BlueRock investment constitutes “a tipping point” that demonstrates confidence in Canadian RM capabilities and may encourage other investors, though she notes that the field is still in the early stages.

“Canada is moving into the clinical space better than others but we lack stable and strategic funding at all stages including discovery,” says Rossant, a senior scientist in the Developmental & Stem Cell Biology program at Toronto’s Hospital for Sick Children. “The US and UK have invested in large pipeline efforts but in Canada we do this in bits and pieces.”

A recent KPMG study found that RM in Canada comprises more than 400 stem cell scientists who work on a range of conditions at 68 centres across the country. This includes the Univ of Toronto’s Medicine By Design, an ambitious RM initiative that received $114 million from Canada First Research Excellence Fund.

Rudkicki says the federal government is taking a serious interest in RM, citing last year’s federal Budget which provided an additional $12 million over two years to the sunsetting Stem Cell Network (SCN), making possible a launch a new program in support of early-stage trials.

The same Budget also provided the pioneering Centre for Drug Research and Development (CDRD — a key participant in the RM workshop — with $32 million over two years to scale up its drug development and accelerator activities to achieve critical mass, while strengthening its extensive international and industry linkages.

“SCN functions as the glue that holds all the pieces together … We created this ecosystem with multiple organizations working in a coordinated fashion and forged a national community,” says Rudnicki. “We also have public-private organizations like CDRD and CCRM (Centre for Commercialization of Regenerative Medicine). The report didn’t address the funding issue directly but what’s implicit is that different organizations — federal and provincial — have an extremely important role to take research to fruition, including commercialization.”

The strengths in RM cited in the report include:

• A collaborative and multidisciplinary culture;
• Recognized scientific leaders and track record;
• Canada’s publicly funded provincial healthcare systems;
• Openness in the regulatory environment;
• Highly qualified personnel (HQP) and the university system;
• International leadership in governance and policy as well as the development of alternative reimbursement models; and
• Demonstrated corporate success (STEMCELL Technologies Inc, BlueRock Therapeutics, Zymeworks, Tissue Regeneration, ExCellThera, RepliCel ).

The CCA report also found several weaknesses it says need to be addressed:

• Lack of stable and strategic funding;
• Thin pharmaceutical and biotechnology ecosystem;
• Misaligned regulatory and reimbursement frameworks;
• Limited cross-training of HQP (i.e. entrepreneurship, product development, large-scale manufacturing and clinical trial implementation);
• Lack of operational funds for cell manufacturing;
• Engagement of philanthropic and patient communities;
• Constraining and inefficient clinical trial and ethics environment; and
• Unrealistic expectations of short- and medium-terms pay-offs.

“Canada has an opportunity to leverage its strength in regenerative medicine to yield further benefits for patients, healthcare systems, and the economy as a whole,’ concludes the report. “Developing and manufacturing novel regenerative medicine therapies would build an industry that has the potential to reduce treatment costs for some diseases, create new jobs for highly qualified personnel, and bring money into the economy.”

New coordinating body

An immediate impact of the RM workshop was the decision to create a new coordinating body —the Regenerative Medicine Alliance of Canada (RMAC) — which will initially be housed at the Stem Cell Network in Ottawa with Rudnicki acting as interim chair. The voluntary organization is comprised of national, provincial and regional organizations, all of which have mandates relevant to RM and/or stem cell research.

In addition to serving as a mechanism to support strategic activities across the RM sector, RMAC members will also work collaboratively to share information and identify strategies that will help to grow the sector, estimated at US $49 billion by 2021. Alliance members have already met once and a second meeting is planned. Rudnicki says the chair and secretariat will be rotated on a regular basis.

“RMAC will help to identify gaps and synergies, speaking with a single voice for the public, funders and patient groups. Its role is to identify strengths and weaknesses,” says Rossant. “An ongoing challenge is the potential disconnect between the research, regulatory and investments systems — federal and provincial. It’s a big challenge. We have an opportunity to be world leaders in the way we treat diseases but we must work with the healthcare system and at reasonable cost.”

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