Bringing medical discoveries to clinical trials
The greatest engines for medical discovery in human history are U.S. academic medical centers, funded by the National Institutes of Health. These centers’ faculty have been awarded the majority of Nobel Prizes for Medicine and have discovered most of the pathways targeted by current drugs. However, this engine for discovery is running at half speed because many high-impact discoveries never make it to clinical trials. This is because of the infamous “valley of death” — the gap between the discovery of an important target and bringing an intervention for that target to clinical trials.
It is possible that there are innumerable cures for complex diseases sitting in freezers in academic medical centers across the nation that will never see the light of day. There are two crucial reasons for this. First, academic medical centers excel at target identification but not at synthesis of interventions. Second, once they identify a compound that can interact with the target, they cannot de-risk that compound sufficiently for biotechnology investors to support its development. And there is little funding to overcome these two barriers. Only a tiny fraction of NIH funds goes toward medicinal chemistry, and these funds are only available if there are already strong candidates available. Even fewer federal dollars are available for synthesizing an optimized lead compound in pure form under good manufacturing practices.
It is possible that there are innumerable cures for complex diseases sitting in freezers in academic medical centers across the nation that will never see the light of day.
Investing in a medicinal chemistry group is a relatively inexpensive proposition for an academic medical center, and just one licensing agreement for a given compound can result in an immeasurably greater return on that investment. Even so, few centers have taken this step, seeing it as the purview of pharmaceutical firms with far greater resources. Yet such an investment could exponentially multiply research in an academic medical center, like infliximab did for New York University or pregabalin for Northwestern University.
Many academic medical centers have often blamed the NIH or biotechnology firms for the valley of death because of their lack of investment. At UT Health San Antonio, we have found relatively inexpensive ways that centers like ours can attract investment, improve the chance that our discoveries will make it to clinical trials and enhance the prospects for Food and Drug Administration approval. In the pages that follow, we provide evidence of how UT Health San Antonio and its investigators — in collaboration with a strong research-focused regional economy — are successfully bringing a range of drug, device and diagnostic discoveries through clinical trials that directly benefit patient care both here in South Texas and far beyond.
Robert Hromas, MD, FACP
Dean, Joe R. and Teresa Lozano Long School of Medicine
