Funding agencies shift focus to "practical" research to bring genomics to the bedside

By Debbie Lawes

Canada's ability to capitalize on genomics research received a major boost this week with a nation-wide competition that will see $135 million invested to move personalized medicine from the lab to the clinic. Champions of personalized medicine are heralding the news, saying funding for large-scale projects is what's needed to translate Canada's international leadership in identifying disease-related genes and biomarkers into a more evidence-based approach to health that provides better and more cost-effective care.

Countries around the world are grappling with the same translational challenges. Canada's announcement on Tuesday — one of the single largest investments ever made in personalized medicine — will help to translate the more than $1 billion spent here on genomics research into new diagnostic tests for preventing, screening, monitoring and treating disease, says Dr Clarissa Desjardins, CEO of Cepmed, the Centre for Personalized Medicine at the Montreal Heart Institute.

"This competition is focusing on the practical implementation of personalized medicine and that is exactly the right area to be funding," she says. "I'm not saying we shouldn't fund fundamental research; it's just that we have a lot of biomarker discoveries on the shelf now. We need the evidence to show to policy makers and others whether a particular test saves money or not, and how it will work in the actual system."

Genome Canada received $65 million in Budget 2011 — $40 million of which will go towards the $67.5-million Large-Scale Applied Research Project Competition in Genomics and Personalized Health. Its partner in the competition — the Canadian Institutes of Health Research — is contributing $22.5 million with an additional $5 million coming from the Cancer Stem Cell Consortium, which is jointly funded by Genome Canada and CIHR. The other $67.5 million will come from matching contributions from the private sector, academia, the provinces or other Canadian and international partners.

Genome Canada expects to award $10 million over four years to about 13 multidisciplinary teams of 20-30 researchers studying the biomedical, clinical, population health, economic, legal, ethical and policy implications of genomics. Preference will be given to mature projects where there is a strong likelihood that results can be integrated into healthcare systems quickly. That means working with known biomarkers or molecules that have gone through at least an initial validation process.

"We're talking about large scale projects and teams that are ready to roll," says CIHR president Dr Alain Beaudet. "For instance, if they want to use human samples, these are teams that already have samples in the freezer... We want research that can bring results to the bedside rapidly."

Dr Tom Hudson, president and scientific director of the Ontario Institute for Cancer Research, says Canada has invested heavily in the discovery side of medical genomics "but we haven't invested in parallel in the clinical research to validate them so that Health Canada and the FDA (US Food and Drug Administration) will approve them and the payers will think they are useful. This competition is a real opportunity to do definitive studies to show clinical utility and to move personalized medicine from the theoretical to the practical."

Biomarkers are a major focus of personalized medicine. Using a simple diagnostic test, these biological footprints can reveal disease progression and how genetic differences in individuals affect the way people respond to drugs. But of the thousands of known biomarkers (and counting), only a handful have been validated and are used in the clinic.

"That validation means being able to look at large patient populations and see that the mutation is there, show how the information could predict outcome, like a response to a therapy or a good or bad prognosis. That's what's clinically useful and that is what's been lacking," says Hudson, who has helped develop a framework for putting personalized medicine into practice for cancer care (see sidebar). OICR has several personalized medicine programs in the pipeline and Hudson says they plan to apply for funding under the new funding competition.

Genome Canada president and CEO Dr Pierre Meulien says it isn't science that has failed to address this translation gap. Rather, it's been a question of how granting agencies direct their funding.

"It's up to the funding bodies to design programs that deliver what you want to be delivered," he says. In this competition, applicants will be required, for example, to provide an economic rationale for how a particular diagnostic test or approach will improve the economic sustainability of Canada's healthcare system.

"Bringing technology into healthcare is always a challenge because the healthcare providers — the payers — often see technology as an add-on cost," adds Meulien. "If we don't provide the evidence that that's not the case, then why should they listen to us."

Some of the new research will address what Meulien refers to as the "no brainers of personalized medicine": using genetic tests to reduce adverse drug reactions, which cost the Canadian healthcare system $14 billion annually, and provide targeted cancer treatments.

Genome Canada's stronger emphasis on translational research and industry partnerships is central to a new strategic plan it hopes will convince the federal government to provide stable, multi-year funding between 2012 and 2017 (R$, Jan. 20/12).

Results-based research is also a priority in CIHR's strategic plan. Over the past two years, it has focused more of its resources on priority areas — including personalized medicine and community-based care — that can translate discoveries into better health products, health services and health policies.

"Personalized medicine is a priority for us and for Genome Canada," says Beaudet. "We realized we could massively increase the impact, particularly the impact on care and the impact on the uptake of the results of the research by decision makers."

Full applications are due August 20 with winners to be announced in December.

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