Finding New Tricks For Old Drugs: An Efficient Route For Public-Sector Drug Discovery

Abstract

A case-study approach is used to show that screening approved, and frequently off-patent, drugs efficiently yields candidate medications suitable for clinical trials. The first two cases highlight cell-based screens that identified medications potentially useful for amyotrophic lateral sclerosis and progressive multifocal leukoencephalopathy, and clinical trials are being planned to test both types of medications. Screening off-patent and off-market medications also led to the discovery of the molecular target responsible for the serious side-effects of the 'Fen/Phen' appetite suppressant combination and to a rational approach to anorectic drug discovery and development. Using a similar approach, candidate medications have also been identified for multiple myeloma (fluphenazine), Alzheimer's disease (non-steroidal anti-inflammatory drugs and glitazones) and various psychiatric disorders Screening all clinically approved medications against both the 'druggable genome' and an array of cell-based readouts is predicted to yield a large number of new therapeutics.