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5-azacytidine + valproic acid + retinoic acid
- Development stage
- Unknown
- Lead developer
- University of Texas MD Anderson Cancer Center
- Modality
- Small Molecules
- Administration
- Oral, Subcutaneous
01
Overview
This is a combination therapy consisting of three agents: - **5-azacytidine (azacitidine)**, a DNA hypomethylating agent that inhibits DNA methyltransferase and leads to reactivation of silenced genes. - **Valproic acid**, a histone deacetylase (HDAC) inhibitor that induces chromatin remodeling and promotes gene expression changes. - **All-trans retinoic acid (ATRA; tretinoin)**, which acts as a differentiating agent by binding to the retinoic acid receptor and promoting differentiation in myeloid cells. The rationale for this combination is based on synergistic epigenetic modulation. 5-azacytidine demethylates DNA, while valproic acid increases histone acetylation; together they can reactivate tumor suppressor genes. ATRA may further enhance differentiation of malignant cells. This regimen has been studied primarily in high-risk acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), especially in elderly or poor-prognosis patients[2][4][6][7]. Clinical trials have shown the combination is feasible with manageable toxicity and some clinical activity.
- Other names
- 5-aza + VPA + ATRAazacitidine + valproic acid + all-trans retinoic acid5-AZA/VPA/ATRA
02
Targets
RARA (Retinoic acid receptor alpha)DNMT (DNA methyltransferase)HDAC (HDAC family)
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