Epigenome-based personalized medicine in human cancer

Epigenomics. 2016 Jan;8(1):119-33. doi: 10.2217/epi.15.84. Epub 2015 Sep 7.


Cancer genome sequencing has created an opportunity for precision medicine. Thus far, genetic alterations can only be used to guide treatment for small subsets of certain cancer types with these key alterations. Similar to mutations, epigenetic events are equally suitable for personalized medicine. DNA methylation alterations have been used to identify tumor-specific drug responsive markers. Methylation of MGMT sensitizes gliomas to alkylating agents is an example of epigenetic personalized medicine. Recent studies have revealed that 5-azacytidine and decitabine show activity in myelodysplasia, lung and other cancers. There are currently at least 20 kinds of histone deacetylase inhibitors in clinical testing. Inhibitors targeting other epigenetic regulators are being clinically tested, such as EZH2 inhibitor EPZ-6438.

Keywords: DNA methylation; epigenome; histone modification; personalized medicine; targeted therapy.

Publication types

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

MeSH terms

  • Antineoplastic Agents / therapeutic use
  • Clinical Trials as Topic
  • Drug Synergism
  • Epigenesis, Genetic / drug effects*
  • Genome, Human
  • Histone Deacetylase Inhibitors / pharmacology*
  • Histone Deacetylase Inhibitors / therapeutic use
  • Humans
  • Neoplasms / drug therapy*
  • Neoplasms / genetics*
  • Precision Medicine
  • Sequence Analysis, DNA


  • Antineoplastic Agents
  • Histone Deacetylase Inhibitors