Fragment-based approaches to the discovery of kinase inhibitors

Methods Enzymol. 2014:548:69-92. doi: 10.1016/B978-0-12-397918-6.00003-3.

Abstract

Protein kinases are one of the most important families of drug targets, and aberrant kinase activity has been linked to a large number of disease areas. Although eminently targetable using small molecules, kinases present a number of challenges as drug targets, not least obtaining selectivity across such a large and relatively closely related target family. Fragment-based drug discovery involves screening simple, low-molecular weight compounds to generate initial hits against a target. These hits are then optimized to more potent compounds via medicinal chemistry, usually facilitated by structural biology. Here, we will present a number of recent examples of fragment-based approaches to the discovery of kinase inhibitors, detailing the construction of fragment-screening libraries, the identification and validation of fragment hits, and their optimization into potent and selective lead compounds. The advantages of fragment-based methodologies will be discussed, along with some of the challenges associated with using this route. Finally, we will present a number of key lessons derived both from our own experience running fragment screens against kinases and from a large number of published studies.

Keywords: Fragment libraries; Fragment screening; Fragment-based drug design; Fragments; Kinase inhibitors; Structure-based drug design.

Publication types

  • Review

MeSH terms

  • Biocatalysis / drug effects
  • Catalytic Domain
  • Databases, Protein
  • Drug Design
  • Drug Discovery / methods*
  • High-Throughput Screening Assays
  • Humans
  • Models, Chemical*
  • Peptide Fragments / antagonists & inhibitors*
  • Peptide Fragments / chemistry
  • Peptide Fragments / metabolism
  • Peptide Library
  • Protein Conformation
  • Protein Kinase Inhibitors / chemistry
  • Protein Kinase Inhibitors / metabolism
  • Protein Kinase Inhibitors / pharmacology*
  • Protein Kinase Inhibitors / therapeutic use
  • Protein Kinases / chemistry*
  • Protein Kinases / metabolism
  • Small Molecule Libraries

Substances

  • Peptide Fragments
  • Peptide Library
  • Protein Kinase Inhibitors
  • Small Molecule Libraries
  • Protein Kinases