Getting promising drug discoveries out of university research labs and into the drug pipeline is more difficult than it should be. by robert pozen
I have run large organizations but spent little time in medical labs. So when I write about translational medical research, I think about the famous announcement in the London Underground: "Mind the gap." Translational research is all about filling two organizational gaps. The first gap is between the scientists making discoveries in the lab and the clinicians seeking patient therapies. The second gap comes later-between finding attractive targets for drugs and diagnostics, for example, and getting the financing for clinical trials.
Researchers have estimated that only 14 percent of all scientific discoveries are ever translated into patient therapies, and even successful translations often take years to happen. This gap is a result of many factors. In some cases, there aren't enough resources to convert promising discoveries into therapies. In other cases, there is insufficient cooperation between scientists doing basic research and physicians working in clinics.
At the same time, the gap is widening between identifying a drug target and securing the funds for the extensive clinical trials needed to obtain approval from the US Food and Drug Administration. These trials have become increasingly expensive-up to $600 million for one drug approval. Because of this large expense and the high risk of failure, drug companies and venture capital firms have become reluctant to finance early-stage drug development.
Because of the urgency of delivering new drugs to combat debilitating diseases, institutions that engage in translational medical research must attempt to close both these gaps-by promoting collaboration between bench scientists and clinical doctors, and by developing drug targets to the point where they will be taken up for clinical trials by for-profit groups. Filling these two gaps is a core mission of the Harvard NeuroDiscovery Center, where I have served for more than five years on the advisory council.
Harvard Medical School established the center in 2001 to focus on neurodegenerative diseases, including Alzheimer's, ALS (also known as Lou Gehrig's disease), multiple sclerosis, and Parkinson's. In order to find therapies for these diseases, the center built a drug discovery lab to test relevant scientific discoveries to determine whether they could be translated into drug targets. Then, in theory, the most promising targets would be licensed to drug companies or venture capitalists to undertake further development and the extensive clinical trials required for drug approval.
The center's approach to closing this gap is clear and logical, but achieving success has not been easy. The center's experience illustrates important lessons about what it takes in practice to become successful at translational medical research.
closing the gap
Let's begin by considering the challenge of closing the gap between scientists and clinicians. At the start, the center informally solicited projects for the lab mainly from scientists at Harvard Universityrelated facilities. When the center received a multiyear grant from the US National Institutes of Health, the center was required to conduct a nationwide solicitation for projects, with only one winner per year allowed to come from a Harvard-affiliated institution. More recently, the selection process has been further constrained because some private funders have insisted that the center design projects to address specific diseases.
The lab began with the policy that every new project would require a post-doctoral fellow from the basic research side to work closely with the lab's staff, who were generally drawn from commercial industry. This seemed to be a sensible policy, because fellows were more readily available and less expensive than medical school professors. Yet the policy proved …