Enhanced activation of STAT pathways and overexpression of survivin confer resistance to FLT3 inhibitors and could be therapeutic targets in AML

Blood. 2009 Apr 23;113(17):4052-62. doi: 10.1182/blood-2008-05-156422. Epub 2009 Jan 14.

Abstract

To further investigate potential mechanisms of resistance to FLT3 inhibitors, we developed a resistant cell line by long-term culture of MV4-11 cells with ABT-869, designated as MV4-11-R. Gene profiling reveals up-regulation of FLT3LG (FLT3 ligand) and BIRC5 (survivin), but down-regulation of SOCS1, SOCS2, and SOCS3 in MV4-11-R cells. Hypermethylation of these SOCS genes leads to their transcriptional silencing. Survivin is directly regulated by STAT3. Stimulation of the parental MV4-11 cells with FLT3 ligand increases the expression of survivin and phosphorylated protein STAT1, STAT3, STAT5. Targeting survivin by short-hairpin RNA (shRNA) in MV4-11-R cells induces apoptosis and augments ABT-869-mediated cytotoxicity. Overexpression of survivin protects MV4-11 from apoptosis. Subtoxic dose of indirubin derivative (IDR) E804 resensitizes MV4-11-R to ABT-869 treatment by inhibiting STAT signaling activity and abolishing survivin expression. Combining IDR E804 with ABT-869 shows potent in vivo efficacy in the MV4-11-R xenograft model. Taken together, these results demonstrate that enhanced activation of STAT pathways and overexpression of survivin are important mechanisms of resistance to ABT-869, suggesting that the STAT pathways and survivin could be potential targets for reducing resistance developed in patients receiving FLT3 inhibitors.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis / drug effects
  • Cell Line, Tumor
  • Drug Resistance, Neoplasm / drug effects
  • Epoxy Compounds / pharmacology
  • Female
  • Gene Expression Regulation, Neoplastic / genetics*
  • Humans
  • Indazoles / pharmacology
  • Inhibitor of Apoptosis Proteins
  • Leukemia, Myeloid, Acute / genetics
  • Leukemia, Myeloid, Acute / metabolism*
  • Leukemia, Myeloid, Acute / pathology*
  • Ligands
  • Mice
  • Mice, Inbred BALB C
  • Mice, Nude
  • Microtubule-Associated Proteins / genetics
  • Microtubule-Associated Proteins / metabolism*
  • Phenylurea Compounds / pharmacology
  • STAT3 Transcription Factor / genetics
  • STAT3 Transcription Factor / metabolism*
  • Sesquiterpenes / pharmacology
  • Signal Transduction / drug effects*
  • Substrate Specificity
  • Survivin
  • Up-Regulation / genetics
  • Xenograft Model Antitumor Assays
  • fms-Like Tyrosine Kinase 3 / antagonists & inhibitors*
  • fms-Like Tyrosine Kinase 3 / metabolism

Substances

  • BIRC5 protein, human
  • Epoxy Compounds
  • IDR-804
  • Indazoles
  • Inhibitor of Apoptosis Proteins
  • Ligands
  • Microtubule-Associated Proteins
  • Phenylurea Compounds
  • STAT3 Transcription Factor
  • STAT3 protein, human
  • Sesquiterpenes
  • Survivin
  • linifanib
  • FLT3 protein, human
  • fms-Like Tyrosine Kinase 3