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Profile of azacitidine

Francsco D\'Alo, Maria Teresa Voso and Giuseppe Leone

Abnormal DNA methylation patterns have been described in different tumor types, including myelodysplastic syndromes and acute myeloid leukemia. DNA hypermethylation in CpG-rich promoters leads to transcriptional repression of several genes, contributing to cancer development. Demethylating agents are able to revert epigenetic silencing induced by hypermethylation showing anticancer affects both in vitro and in vivo. Azacitidine, a DNA-methyltransferase inhibitor approved by the US Food and Drug Administration for the treatment of myelodysplastic syndrome, resulted in reduced transfusion dependence and risk of leukemic transformation and improved quality of life and survival when compared with best supportive care in patients with myelodysplastic syndromes. Future development will include combination therapy with histone deacetylase inhibitors, such as valproic acid, and the development of new oral demethylating agents.