Epigenetic therapy for solid tumors: From bench science to clinical trials

Yen Yi Juo, Xue Jun Gong, Ankita Mishra, Xiao Cui, Stephen B. Baylin, Nilofer S. Azad, Nita Ahuja

Research output: Contribution to journalReview articlepeer-review


The cancer epigenome is characterized by global DNA methylation and chromatin changes, such as the hypermethylation of specific CpG island promoters. Epigenetic agents like DNA methyltransferase or histone deacetylase inhibitors induce phenotype changes by reactivation of epigenetically silenced tumor suppressor genes. Despite initial promise in hematologic malignancies, epigenetic agents have not shown significant efficacy as monotherapy against solid tumors. Recent trials showed that epigenetic agents exert favorable modifier effects when combined with chemotherapy, hormonal therapy, or other epigenetic agents. Due to the novel nature of their mechanism, it is important to reconsider the optimal patient selection, drug regimen, study design, and outcome measures when pursuing future trials in order to discover the full potential of this new therapeutic modality.

Original languageEnglish (US)
Pages (from-to)215-235
Number of pages21
Issue number2
StatePublished - Apr 1 2015


  • DNA methyltransferase inhibitor
  • cancer therapy
  • clinical trials
  • epigenetics
  • histone deacetylase inhibitor
  • solid tumor

ASJC Scopus subject areas

  • Genetics
  • Cancer Research


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