Decitabine, a new star in epigenetic therapy: the clinical application and biological mechanism in solid tumors

Cancer Lett. 2014 Nov 1;354(1):12-20. doi: 10.1016/j.canlet.2014.08.010. Epub 2014 Aug 14.


Epigenetic alterations are strongly associated with cancer development and drug resistance. The use of the DNA methylation inhibitor decitabine (Dacogen®) has been approved in the treatment of hematological malignancies, and its clinical effects on solid tumors have gained attention. Here, we present a review of the molecular regulation mechanisms, clinical experiences and biological evaluation for novel decitabine-based therapies in solid tumors. We also discuss the following questions: What is the best administration schedule of decitabine in solid tumors? Is there tumor type specificity for decitabine-based epigenetic therapy? What are the biological function and mechanism of decitabine in suppressing tumor development? Is there a correlation between DNA demethylation and clinical response? Importantly, low-dose decitabine and combined therapy show significant improvement in solid tumor treatment. However, the correlation studies are preliminary, and key biomarkers for prognosis need further investigation.

Keywords: Decitabine; Demethylating drug; Drug sensitivity; Tumor therapy.

Publication types

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

MeSH terms

  • Antimetabolites, Antineoplastic / administration & dosage
  • Antimetabolites, Antineoplastic / chemistry
  • Azacitidine / administration & dosage
  • Azacitidine / analogs & derivatives*
  • Azacitidine / chemistry
  • Biomarkers, Tumor
  • Clinical Trials as Topic
  • DNA Methylation / drug effects*
  • Decitabine
  • Epigenesis, Genetic*
  • Humans
  • Neoplasms / drug therapy*
  • Neoplasms / genetics*
  • Prognosis


  • Antimetabolites, Antineoplastic
  • Biomarkers, Tumor
  • Decitabine
  • Azacitidine