Marked for death: targeting epigenetic changes in cancer

Nat Rev Drug Discov. 2017 Apr;16(4):241-263. doi: 10.1038/nrd.2016.256. Epub 2017 Mar 10.


In the past few years, it has become clear that mutations in epigenetic regulatory genes are common in human cancers. Therapeutic strategies are now being developed to target cancers with mutations in these genes using specific chemical inhibitors. In addition, a complementary approach based on the concept of synthetic lethality, which allows exploitation of loss-of-function mutations in cancers that are not targetable by conventional methods, has gained traction. Both of these approaches are now being tested in several clinical trials. In this Review, we present recent advances in epigenetic drug discovery and development, and suggest possible future avenues of investigation to drive progress in this area.

Publication types

  • Review

MeSH terms

  • Drug Discovery / methods
  • Drug Discovery / trends
  • Enzyme Inhibitors / therapeutic use
  • Epigenesis, Genetic / drug effects*
  • Epigenesis, Genetic / genetics*
  • Humans
  • Models, Genetic
  • Molecular Targeted Therapy / methods*
  • Mutation*
  • Neoplasms / drug therapy*
  • Neoplasms / genetics*


  • Enzyme Inhibitors