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azacitidine + venetoclax + all-trans retinoic acid
- Development stage
- Unknown
- Modality
- Small Molecules
- Administration
- Oral, Subcutaneous, Intravenous
01
Overview
Azacitidine + venetoclax + all-trans retinoic acid is an investigational combination therapy for acute myeloid leukemia (AML), particularly in newly diagnosed patients who are ineligible for intensive chemotherapy. Azacitidine is a hypomethylating agent that inhibits DNA methyltransferase, leading to DNA hypomethylation and reactivation of tumor suppressor genes. Venetoclax is a selective BCL-2 inhibitor that promotes apoptosis in AML cells by antagonizing the anti-apoptotic protein BCL-2. All-trans retinoic acid (ATRA, also known as tretinoin) acts as a differentiation agent by binding to the retinoic acid receptor alpha (RARα), promoting maturation of leukemic blasts. The triplet regimen aims to combine these mechanisms for synergistic antileukemic effects and improved remission rates compared to standard regimens[1][4][5]. This combination has been studied in phase II clinical trials with induction, consolidation, and maintenance phases specifically designed for AML treatment.
- Other names
- azacitidine + venetoclax + ATRAazacitidine + venetoclax + tretinoin
02
Targets
BCL-2 (BCL-2 family)RARA (Retinoic acid receptor alpha)DNMT (DNA methyltransferase)
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