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This combination therapy consists of the BCL-2 inhibitor venetoclax, a hypomethylating agent (either azacitidine or decitabine), and low-dose cytarabine. It is being evaluated by The First Affiliated Hospital of Soochow University for the treatment of newly diagnosed, unfit patients with CEBPA-mutated acute myeloid leukemia (AML). Venetoclax works by binding to and inhibiting the anti-apoptotic protein BCL-2, thereby restoring the process of apoptosis in cancer cells. Hypomethylating agents (HMAs) like azacitidine and decitabine function as epigenetic modulators by inhibiting DNA methyltransferase, which leads to the reactivation of tumor suppressor genes. Cytarabine is a pyrimidine analog that interferes with DNA synthesis by inhibiting DNA polymerase. The combination of these agents is intended to provide a synergistic antineoplastic effect in molecularly defined subsets of AML.
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