An inhibitor of DNA methyltransferases that reduces hypermethylation associated with certain diseases, including myelodysplastic syndromes (IC50s = 2.4 and 2.6 μM for in vitro anti-myeloma activity) and cancer (IC50s ~ 0.4 μM for inhibiting proliferation of various cancer cell lines).

Azacytidine is a cytidine analog that is incorporated into DNA, inhibiting DNA methyltransferase (DNMT) activity and preventing DNA repair. This demethylating agent is clinically used to treat myelodysplastic syndromes and potentiates the effects of other chemotherapeutics, such as cisplatin.…