Marrow leptin-LEPR signaling rewires mitochondrial oxidative metabolism to confer chemoresistance in acute myeloid leukemia

Cell Death Dis. 2026 Feb 23;17(1):249. doi: 10.1038/s41419-026-08528-0.

Abstract

Leptin is abundant within marrow adipose tissue, yet its impact on acute myeloid leukemia (AML) therapy response is undefined. Here, we report that elevated bone-marrow leptin and blast-cell leptin-receptor (LEPR) levels strongly associate with poor cytarabine (Ara-C) clearance and reduced survival in newly diagnosed AML patients. Mechanistic and functional validation in human AML lines, primary blasts, and two syngeneic mouse models (MLL-AF9, AML1-ETO9a) shows that exogenous leptin markedly blunts Ara-C cytotoxicity, whereas the high-affinity LEPR antagonist Allo-aca restores chemosensitivity without altering baseline leukemia growth. Leptin up-regulates LEPR and triggers JAK2/STAT3 signaling that boosts mitochondrial complex Ⅰ activity, oxidative phosphorylation, and mitochondrial reactive oxygen species (mtROS); the resulting mtROS surge activates a compensatory antioxidant program that shields blasts from drug-induced oxidative damage. These data identify an adipokine-driven metabolic circuit governing AML chemoresistance and reveal LEPR blockade as a tractable strategy to improve outcomes, underscoring adipose-tumor crosstalk as a general therapeutic vulnerability.

MeSH terms

  • Animals
  • Bone Marrow* / metabolism
  • Bone Marrow* / pathology
  • Cell Line, Tumor
  • Cytarabine / pharmacology
  • Cytarabine / therapeutic use
  • Drug Resistance, Neoplasm* / drug effects
  • Female
  • Humans
  • Janus Kinase 2 / metabolism
  • Leptin* / metabolism
  • Leptin* / pharmacology
  • Leukemia, Myeloid, Acute* / drug therapy
  • Leukemia, Myeloid, Acute* / genetics
  • Leukemia, Myeloid, Acute* / metabolism
  • Leukemia, Myeloid, Acute* / pathology
  • Male
  • Mice
  • Mitochondria* / drug effects
  • Mitochondria* / metabolism
  • Oxidative Phosphorylation / drug effects
  • Reactive Oxygen Species / metabolism
  • Receptors, Leptin* / genetics
  • Receptors, Leptin* / metabolism
  • STAT3 Transcription Factor / metabolism
  • Signal Transduction / drug effects

Substances

  • Receptors, Leptin
  • Leptin
  • Cytarabine
  • Reactive Oxygen Species
  • STAT3 Transcription Factor
  • Janus Kinase 2
  • LEPR protein, human