In silico repositioning of approved drugs for rare and neglected diseases

Drug Discov Today. 2011 Apr;16(7-8):298-310. doi: 10.1016/j.drudis.2011.02.016. Epub 2011 Mar 1.


One approach to speed up drug discovery is to examine new uses for existing approved drugs, so-called 'drug repositioning' or 'drug repurposing', which has become increasingly popular in recent years. Analysis of the literature reveals many examples of US Food and Drug Administration-approved drugs that are active against multiple targets (also termed promiscuity) that can also be used to therapeutic advantage for repositioning for other neglected and rare diseases. Using proof-of-principle examples, we suggest here that with current in silico technologies and databases of the structures and biological activities of chemical compounds (drugs) and related data, as well as close integration with in vitro screening data, improved opportunities for drug repurposing will emerge for neglected or rare/orphan diseases.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Databases, Factual
  • Drug Design
  • Drug Discovery
  • Drug Industry
  • Drug Repositioning*
  • High-Throughput Screening Assays
  • Humans
  • Neglected Diseases / drug therapy*
  • Orphan Drug Production / methods*
  • Rare Diseases*