ascorbic acid + azacitidine

ascorbic acid + azacitidine is an investigational combination therapy being evaluated in the AIMS trial (NCT05743036) for the treatment of myelodysplastic syndrome (MDS). The regimen involves high-dose intravenous ascorbic acid (Vitamin C) administered in conjunction with the hypomethylating agent azacitidine. Ascorbic acid acts as a necessary cofactor for Ten-Eleven Translocation (TET) enzymes, particularly TET2, which are frequently mutated or functionally deficient in myeloid malignancies. By enhancing TET2 activity, ascorbic acid promotes the conversion of 5-methylcytosine to 5-hydroxymethylcytosine, facilitating DNA demethylation. Azacitidine, a pyrimidine nucleoside analog, complements this by inhibiting DNA methyltransferase (DNMT) enzymes. This dual approach aims to synergistically reverse global DNA hypermethylation and restore normal hematopoietic cell differentiation, potentially improving clinical outcomes in patients with higher-risk MDS.

Other names

High-dose ascorbate and azacitidineIV HDA and azacitidineVitamin C and azacitidine