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This combination therapy consists of the hypomethylating agent azacitidine, the BCL-2 inhibitor venetoclax, and the corticosteroid dexamethasone. It is primarily being investigated for the treatment of acute lymphoblastic leukemia (ALL), particularly in relapsed or refractory settings. Azacitidine works by inhibiting DNA methyltransferase, leading to DNA hypomethylation and apoptosis of hematopoietic cells. Venetoclax selectively inhibits B-cell lymphoma 2 (BCL-2), an anti-apoptotic protein, thereby restoring the apoptotic process in cancer cells. Dexamethasone provides additional lymphocytotoxic effects and helps manage inflammation. The combination is being studied by institutions like The First Affiliated Hospital of Nanjing Medical University to improve outcomes in patients with difficult-to-treat ALL, including T-cell ALL and mixed-phenotype acute leukemia (MPAL).
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