Promises and challenges of anticancer drugs that target the epigenome

Epigenomics. 2011 Oct;3(5):547-65. doi: 10.2217/epi.11.82.


The occurrence of epigenetic aberrations in cancer and their role in promoting tumorigenesis has led to the development of various small molecule inhibitors that target epigenetic enzymes. In preclinical settings, many epigenetic inhibitors demonstrate promising activity against a variety of both hematological and solid tumors. The therapeutic efficacy of those inhibitors that have entered the clinic however, is restricted predominantly to hematological malignancies. Here we outline the observed epigenetic aberrations in various types of cancer and the clinical responses to epigenetic drugs. We furthermore discuss strategies to improve the responsiveness of both hematological and solid malignancies to epigenetic drugs.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Antineoplastic Agents / pharmacology*
  • Chromatin / physiology*
  • DNA (Cytosine-5-)-Methyltransferases / antagonists & inhibitors
  • DNA (Cytosine-5-)-Methyltransferases / metabolism
  • DNA Methylation / physiology*
  • Epigenesis, Genetic / drug effects*
  • Epigenesis, Genetic / physiology
  • Histone Acetyltransferases / antagonists & inhibitors
  • Histone Acetyltransferases / metabolism
  • Histone Deacetylase Inhibitors / pharmacology
  • Histone Demethylases / metabolism
  • Histone Methyltransferases
  • Histone-Lysine N-Methyltransferase / metabolism
  • Histones / metabolism*
  • Humans
  • Models, Biological*
  • Neoplasms / drug therapy*


  • Antineoplastic Agents
  • Chromatin
  • Histone Deacetylase Inhibitors
  • Histones
  • Histone Demethylases
  • Histone Methyltransferases
  • DNA (Cytosine-5-)-Methyltransferases
  • Histone-Lysine N-Methyltransferase
  • Histone Acetyltransferases