Drug intelligence / Profile preview

venetoclax + cytarabine + metformin

Development stage
Unknown
Lead developer
Hospital Municipal São José
Modality
Small Molecules
Administration
Oral, Subcutaneous
01

Overview

VenCM is an experimental combination therapy comprising venetoclax, cytarabine, and metformin, currently under investigation for the treatment of relapsed-refractory and induction-ineligible acute myeloid leukemia (AML). This regimen, sponsored by Hospital Municipal São José, explores the repositioning of metformin—a biguanide typically used for type 2 diabetes—as an antineoplastic enhancer. Metformin inhibits complex I of the mitochondrial electron transport chain, which reduces oxidative phosphorylation (OXPHOS). This metabolic shift is hypothesized to synergize with venetoclax, a BCL-2 inhibitor, by overcoming mitochondrial-dependent resistance mechanisms in leukemia cells. Cytarabine is included as a standard cytotoxic antimetabolite that interferes with DNA synthesis, providing a multi-targeted approach to treating aggressive AML.

Other names
VenCM regimen
02

Targets

DNAND1 (Mitochondrial electron transport chain complex I)

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