Experiences in fragment-based drug discovery

Trends Pharmacol Sci. 2012 May;33(5):224-32. doi: 10.1016/j.tips.2012.02.006. Epub 2012 Mar 27.

Abstract

Fragment-based drug discovery (FBDD) has become established in both industry and academia as an alternative approach to high-throughput screening for the generation of chemical leads for drug targets. In FBDD, specialised detection methods are used to identify small chemical compounds (fragments) that bind to the drug target, and structural biology is usually employed to establish their binding mode and to facilitate their optimisation. In this article, we present three recent and successful case histories in FBDD. We then re-examine the key concepts and challenges of FBDD with particular emphasis on recent literature and our own experience from a substantial number of FBDD applications. Our opinion is that careful application of FBDD is living up to its promise of delivering high quality leads with good physical properties and that in future many drug molecules will be derived from fragment-based approaches.

MeSH terms

  • Amyloid Precursor Protein Secretases / chemistry
  • Animals
  • Aspartic Acid Endopeptidases / chemistry
  • Drug Discovery*
  • HSP90 Heat-Shock Proteins / chemistry
  • Humans
  • Pharmaceutical Preparations / chemistry*
  • Protein Conformation
  • Proto-Oncogene Proteins B-raf / chemistry

Substances

  • HSP90 Heat-Shock Proteins
  • Pharmaceutical Preparations
  • Proto-Oncogene Proteins B-raf
  • Amyloid Precursor Protein Secretases
  • Aspartic Acid Endopeptidases
  • BACE1 protein, human