Bioactive venetoclax-lipid nanomedicine enhances therapeutic efficacy in acute myeloid leukemia.

Acute myeloid leukemia (AML) remains a highly aggressive hematological malignancy with frequent relapse and limited therapeutic durability. Although venetoclax has provided an important therapeutic option for AML, suboptimal response and treatment resistance continue to restrict its clinical benefit. Here, we developed a venetoclax-loaded lipid nanomedicine, Vene-lipo, to enhance anti-leukemic efficacy. Vene-lipo displayed uniform nanoscale morphology, favorable colloidal stability, and good preliminary biosafety. Compared with free venetoclax, Vene-lipo more effectively suppressed AML cell growth and promoted ROS-associated DNA damage and apoptosis. In vivo, Vene-lipo reduced leukemic burden, alleviated leukemia-associated organ involvement, improved hematological abnormalities, and prolonged survival. These findings demonstrate that lipid nanomedicine-based delivery can potentiate the therapeutic activity of venetoclax and provide a promising strategy for AML treatment.