THE BLOG

Medical Research: When Scientific 'Breakthroughs' Get Lost in Translation

Dec 12, 2010 | Updated May 25, 2011

Every day we see stories in the media about the latest medical "breakthroughs" that could lead to treatments or cures for dreaded diseases. We are overwhelmed with snippets about stem cells, genes linked to Alzheimer's disease, autism and diabetes. We hear that cancer drugs are being tailored to treat an individual tumor. And yet, many breakthroughs like these have not borne fruit for patients.

Whether it's because science is hard and unpredictable, or that resources are limited, or there is lack of prioritization -- too many great scientific ideas sit at the laboratory bench waiting for translation. But, there are successful models that have turned a basic discovery into an effective therapeutic option. These models can provide pathways to a healthier future.

We are at a critical inflection point in current discussions within the biomedical research establishment about what actions need to be taken to push the science toward cures where possible. We need to take advantage of this moment, and we need to bring patients, the public and policymakers into the conversation. Next week from December 13-15 FasterCures is convening in New York City all of the sectors involved in medical research to do just that. Partnering for Cures is a meeting like no other, a place to forge collaborations and participate in outcome-focused dialogue about the challenges facing medical research. We have always maintained that each of the sectors plays a vital role, whether it is government, industry, philanthropy, academia, finance or the non-profits. And the need for their ability to seamlessly pass the baton from one to the other has never been greater or the stakes higher.

Successful models have integrated all of these sectors. Everyone needs to be at this table. Few life-saving therapies have come to market without the resources of private industry. Increasingly, patients have become more sophisticated and disease groups are ever innovating with new models for collaboration with academia and industry. The U.S. government is recognizing that it can play a special and critical role in providing an environment where successful partnerships can grow and proliferate. Partnering for Cures provides an opportunity for all sectors to productively collide, creating an ultimate open source opportunity to shine a light on these models so others can learn and build on them.

One area of keen interest to us is identifying solutions and models to cross the so-called "Valley of Death" -- an ever-widening gap in funding and support for the kind of research that moves basic science down the path toward treatments.

In a new report released today by FasterCures, "Crossing Over the Valley of Death," we highlight the productivity gap that currently exists today, how research moves from molecule to marketplace, how we can traverse the Valley of Death and what all of the sectors are doing towards that end. Many players are marching into that valley, but we are far from reaching the other side.

The need to keep marching with the resources required to make the passage is recognized by advisors to U.S. National Institutes of Health (NIH) Director Dr. Francis Collins, who have recommended that a new translational medicine and therapeutics development center be created by the federal agency post-haste. This proposed center would bring together existing NIH activities in translational research and medicine and allow greater coordination and collaboration internally as well as externally, and ultimately, we hope, produce greater outcomes.

We need to support this recommendation, which if implemented would shine a light on the critically needed but under-resourced and under-appreciated area of translational research. In our jump to embrace this incredible opportunity, however, we need to ensure that Peter is not robbed to pay Paul. NIH's other strengths include supporting the nation's basic biomedical research enterprise, and that focus cannot be lost or diminished in our impatience with the pace of progress. Basic science is still as important as ever, but we also want and deserve concrete outcomes.

Dr. Robert Beall, President and CEO of the Cystic Fibrosis Foundation often talks about the Foundation's own model for de-risking research as providing "more shots on goal." Never has the need been greater to have all of the sectors implement that same approach, to take bold steps to move forward swiftly.

Our nation's wise and prolonged investment in basic science has produced discoveries that now need translation. Knowledge gained from basic discoveries allows us to take more strategic and informed shots on goal. This new and important focus on translation no doubt makes some in the research community uncomfortable, as they worry about focus, changing research priorities and competition over scarce dollars.

We recognize those concerns, but at the same time, the need for accountability and outcomes prevail. Patients need to know that we, the collective "we" in the medical research system, are doing everything we can to get new preventive, diagnostic and treatment options through the pipeline and into the clinic. The alternative to this change is the status quo -- 15 years for an intervention to go from bench to bedside. Clearly that isn't acceptable. Is it?

Join us at Partnering for Cures, December 13-15 in New York City. www.partneringforcures.org.