Inhibition of the receptor tyrosine kinase Axl impedes activation of the FLT3 internal tandem duplication in human acute myeloid leukemia: implications for Axl as a potential therapeutic target

Blood. 2013 Mar 14;121(11):2064-73. doi: 10.1182/blood-2012-07-444018. Epub 2013 Jan 15.

Abstract

Approximately 20% to 25% of patients with acute myeloid leukemia (AML) have a constitutively activated FLT3-internal tandem duplication (FLT3-ITD), and these patients exhibit a poor prognosis. Here, we report that Axl, a receptor tyrosine kinase (RTK) overexpressed and constitutively active in human AML, targets the RTK FLT3 in FLT3-ITD(+) AML. Abrogation of Axl activation by soluble Axl chimeric protein (Axl-Fc) or small interfering RNA (siRNA) diminishes constitutive FLT3 phosphorylation in FLT3-ITD(+) AML. In addition, inhibition of Axl activation by Axl-Fc interferes with the physical interaction between Axl and FLT3. We found that Axl-Fc, a pharmacologic Axl inhibitor, or siRNA targeting Axl inhibits cell growth, induces cell-cycle arrest and apoptosis, and relieves a block in myeloid differentiation of FLT3-ITD(+) AML in vitro. Axl-Fc also suppresses the growth of human FLT3-ITD(+) AML in vivo. Collectively, our data suggest that Axl contributes to the pathogenesis of FLT3-ITD(+) AML through, at least in part, positive regulation of constitutive FLT3 activation. This also suggests that Axl should be pursued as a potential target for the treatment of FLT3-ITD(+) AML.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Anilides / pharmacology*
  • Anilides / therapeutic use
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use
  • Axl Receptor Tyrosine Kinase
  • Cell Transformation, Neoplastic / drug effects
  • Cell Transformation, Neoplastic / genetics
  • Cells, Cultured
  • Dose-Response Relationship, Drug
  • Drug Evaluation, Preclinical
  • Gene Duplication / drug effects*
  • Gene Expression Regulation, Enzymologic / drug effects
  • Gene Expression Regulation, Leukemic / drug effects
  • Humans
  • Leukemia, Myeloid, Acute / drug therapy
  • Leukemia, Myeloid, Acute / genetics*
  • Molecular Targeted Therapy
  • Protein Kinase Inhibitors / pharmacology*
  • Protein Kinase Inhibitors / therapeutic use
  • Proto-Oncogene Proteins / antagonists & inhibitors*
  • Quinolines / pharmacology*
  • Quinolines / therapeutic use
  • RNA, Small Interfering / pharmacology
  • Receptor Protein-Tyrosine Kinases / antagonists & inhibitors*
  • Tandem Repeat Sequences / drug effects
  • Tandem Repeat Sequences / genetics
  • fms-Like Tyrosine Kinase 3 / genetics*

Substances

  • Anilides
  • Antineoplastic Agents
  • GSK 1363089
  • Protein Kinase Inhibitors
  • Proto-Oncogene Proteins
  • Quinolines
  • RNA, Small Interfering
  • FLT3 protein, human
  • Receptor Protein-Tyrosine Kinases
  • fms-Like Tyrosine Kinase 3
  • Axl Receptor Tyrosine Kinase
  • AXL protein, human