Decitabine is a cytosine nucleoside analog that acts as a potent inhibitor of DNA methylation. Decitabine can be incorporated into DNA and inhibits further DNA methylation and induce apoptosis and senescence in leukemic cells. [1]

In a panel of primary (acute myeloid leukemia) AML cultures, Decitabine significantly inhibited proliferation and reduced cell viability with IC50 10-60 nM. [2]

Decitabine shows activity in a broad range of hematologic disorders. Currently, Decitabine is being studied in a Phase III clinical trials in patients with myelodysplastic syndrome (MDS) [3] and in a Phase II clinical trials in patients with AML [4].