Small Molecules in Oncology

Volume 184 of the series Recent Results in Cancer Research pp 131-157



  • Michael DaskalakisAffiliated withDivision of Hematology and Oncology, Freiburg University Medical Center Email author 
  • , Nadja Blagitko-Dorfs
  • , Björn Hackanson

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The pyrimidine analogs, 5-azacytidine (azacitidine, Vidaza®) and its deoxy derivative, 5-aza-2’-deoxycytidine (decitabine, Dacogen®), are the most widely used inhibitors of DNA methylation which trigger demethylation leading to a consecutive reactivation of epigenetically silenced tumor suppressor genes in vitro and in vivo.

Although the antileukemic capacity of decitabine has been known for almost 40 years, its therapeutic potential in hematologic malignancies is still under intensive investigation. Multiple clinical trials have shown the promising activity of low-dose decitabine in AML, MDS, CML, and hemoglobinopathies, whereas its efficacy in solid tumors is rather limited.

Clinical responses appear to be induced by both epigenetic alterations and the induction of cell-cycle arrest and/or apoptosis. Recent clinical trials have been investigating new dosing schedules, routes of administration, and combination of decitabine with other agents, including histone deacetylase (HDAC) inhibitors.