Drug intelligence / Profile preview

azacitidine + valproic acid

Development stage: Unknown
Lead developer: University of Texas MD Anderson Cancer Center
Modality: Small Molecules
Administration: Subcutaneous, Oral, Intravenous

Overview

Azacitidine + valproic acid is an investigational combination therapy consisting of the DNA methyltransferase inhibitor azacitidine and the histone deacetylase (HDAC) inhibitor valproic acid. This combination targets epigenetic mechanisms by inducing DNA demethylation and histone acetylation, aiming to synergistically reactivate silenced tumor suppressor genes. It has been extensively studied in Phase I and II clinical trials for the treatment of high-risk myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), particularly in elderly patients, those with poor-risk cytogenetics, and as a maintenance strategy following allogeneic stem cell transplantation.

Other names: 5-azacytidine + valproic acidAZA + VPA5-AZA + VPAazacitidine + sodium valproate

Targets

DNMT1 (DNA (cytosine-5)-methyltransferase 1)HDAC1 (Histone Deacetylase 1)

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