Skip to main content

Drug Repurposing

Using Human Genetic Information to Find Effective and Affordable Therapies

Drug Paradigm Many people suffer from rare and/or complex diseases with no known treatment options.  Despite an understanding of the molecular basis of almost six thousand diseases, we only have about 500 approved therapies.  Our hope is to drive a shift in the drug development paradigm in order to efficiently identify drug repurposing candidates that are safe, affordable, and most importantly effective, to help improve the lives of these patients.

The goal of the Accelerating Drug Discovery and Repurposing Incubator (ADDRI) is to discover new uses for FDA-approved drugs based on an understanding of diseases driven by human genetics.  The genetic data we use is from BioVU, Vanderbilt’s deidentified human DNA biobank. 

Identify Discover Validate

Capitalizing on our extensive knowledge of chemistry, biochemistry,  and protein structure, the Center for Knowledge Management (CKM) collaborates with the Drug Repurposing team as they develop new project concepts.  CKM is primarily involved in the three early phases of analysis depicted to the right.

CKM contributions include:

Elements of a Repurposing Package

  • Identifying variant and wild type alleles for specific mutations
  • Ensuring the frequency of variant alleles is consistent with reported allele frequencies in our population(s) of study
  • Reviewing PheWAS results in the context of how each genetic mutation may affect the expression, structure, function, and/or pathway interactions of our protein drug target of interest
  • Analyzing all current evidence and prediction algorithm output to support the biological plausibility of hypotheses and new drug indications
  • Collaborating to find already approved drugs that target our protein of interest with the desired pharmacological effect and safety profile

Subsequent to validation, the repurposing team aims to develop a commercially viable package for licensing to support larger scale registration trials and development activities.

Publications/Presentations

  • Naylor HM, Shirey-Rice JK, Zaleski NM, Jerome RN, Lavieri RR, Prusijssers AJ, Chettiar SN, Pulley JM, Giuse NB. Vanderbilt University Medical Center’s drug repurposing program: using human genetic information to find effective and affordable therapies. American Medical Informatics Association (AMIA) Informatics Summit, Washington, DC, March 2018.