Targeting ABCD1 inhibits peroxisomal fatty acid oxidation to selectively eliminate acute myeloid leukemia cells - PubMed
4 hours ago
- #acute myeloid leukemia
- #lipid metabolism
- #ABCD1 inhibitor
- Altered lipid metabolism supports the growth of acute myeloid leukemia (AML) cells.
- AML cells upregulate the peroxisomal very-long-chain fatty acid (VLCFA) transporter ABCD1 and increase peroxisomal fatty acid oxidation (pFAO).
- Genetic silencing or pharmacological inhibition of ABCD1 (using eicosenol) impairs pFAO, leading to VLCFA accumulation and selective AML cell death.
- Normal hematopoietic cells remain viable by upregulating glycolysis when ABCD1 is inhibited.
- In murine models, ABCD1 inhibition with eicosenol reduces leukemia burden and prolongs survival without toxicity.
- ABCD1 is identified as a novel anti-AML therapeutic target.