The Vanderbilt Center for Neuroscience Drug Discovery

The Vanderbilt Center for Neuroscience Drug Discovery (VCNDD) extends traditional academic pursuits in basic science to take the most exciting advances in our understanding of human disease and drug targets to a point where these breakthroughs can directly impact patient care.

The most innovative and potentially high impact new approaches for treatment of human disease that arise from academic basic science efforts are often untested and pose unacceptable risks for investment of the tremendous capital required to discover and develop a new drug in an industry setting. This is especially true in discovery of new treatment strategies for the most serious of brain disorders, such as Alzheimer’s disease, Parkinson’s disease, schizophrenia, autism, and others. By incorporating the highest level of drug discovery into academic research, we are able to advance the most exciting scientific breakthroughs beyond the lab and toward the development of patentable and marketable drugs suited for clinical studies.

This new model for academic drug discovery pioneered by the VCNDD is crucial to the drug discovery process because it “de risks” new treatment strategies with the potential to transform the standard of patient care. By investing in the early stages of research that pharmaceutical companies are reluctant to undertake, we can provide strong validation of innovative approaches to treatment of severe brain disorders. Of the myriad compounds discovered in these early stages, only a few translate into marketable medicine; thus, a company mainly concerned with profits is often unable to engage in this research. Scientists in the Vanderbilt Center for Neuroscience Drug Discovery, unhindered by constraints that are unavoidable in an industry setting, are free to conduct early-stage work that serves as the basis of major therapeutic breakthroughs. By learning the defining mechanisms behind a disease, research scientists can tailor therapeutics to fit an individual’s biology, thereby significantly raising the chances of efficacy for a new drug in clinical trials. We take a “bench to bedside” approach in all of our endeavors and maintain a clear focus on the ultimate goal in patient care.

We also focus tremendous effort on taking observations from clinical studies back to the laboratory, taking a “bedside to bench” approach to identify new treatment strategies that increases confidence that clinical efficacy can be achieved. The Vanderbilt Center for Neuroscience Drug Discovery combines the expertise of four groups of specialists: Medicinal Chemistry, In Vivo Pharmacology, Molecular Pharmacology, and Drug Metabolism and Pharmacokinetics (DMPK), to identify compounds with a specific target, enhance the compound’s selectivity and efficacy, and ensure chemical and behavioral safety, in vivo. These groups work in close collaboration with the tremendous research infrastructure of other institutes and departments at Vanderbilt to advance new treatment strategies for testing in a clinical setting.

The Vanderbilt Center for Neuroscience Drug Discovery is staffed by dozens of scientists, most of which bring industry experience to this collaborative, academic setting. Since 2007, VCNDD has made significant progress in finding treatments and possible cures for neurological disorders such as Fragile X syndrome, schizophrenia, dystonia, Parkinson’s disease, Alzheimer’s disease, and post-traumatic stress disorder. VCNDD research has been funded publicly by the NIMH, NIDA, and NINDS, as well as privately by the Michael J Fox Foundation, Seaside Therapeutics, Johnson & Johnson, Astra Zeneca, and other foundation and corporate partners.