Azacitidine and the beginnings of therapeutic epigenetic modulation.
BACKGROUND
Although originally developed as a cytarabine analog more than 40 years ago, azacitidine has been the subject of renewed interest in the era of cancer epigenetics.


OBJECTIVE
What is the history of the clinical development of azacitidine and how has it been applied successfully to the treatment of myelodysplastic syndromes (MDS)?


METHODS
We review the evolution of the use of azacitidine for the therapy of human disease and review the major studies that have laid the groundwork for its current clinical indication.


CONCLUSIONS
The use of azacitidine has changed the approach to the treatment of MDS and has resulted in improved outcomes for patients.
O'Dwyer
Kristen
K
Memorial Sloan-Kettering Cancer Center, Leukemia Service, Department of Medicine, New York, 10021, USA.
Maslak
Peter
P
D000964:Antimetabolites, Antineoplastic / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D001374:Azacitidine / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D044127:Epigenesis, Genetic; D006801:Humans; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D016896:Treatment Outcome
D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine