Activating JAK-mutations confer resistance to FLT3 kinase inhibitors in FLT3-ITD positive AML in vitro and in vivo

Leukemia. 2021 Jul;35(7):2017-2029. doi: 10.1038/s41375-020-01077-1. Epub 2020 Nov 4.

Abstract

An important limitation of FLT3 tyrosine kinase inhibitors (TKIs) in FLT3-ITD positive AML is the development of resistance. To better understand resistance to FLT3 inhibition, we examined FLT3-ITD positive cell lines which had acquired resistance to midostaurin or sorafenib. In 6 out of 23 TKI resistant cell lines we were able to detect a JAK1 V658F mutation, a mutation that led to reactivation of the CSF2RB-STAT5 pathway. Knockdown of JAK1, or treatment with a JAK inhibitor, resensitized cells to FLT3 inhibition. Out of 136 patients with FLT3-ITD mutated AML and exposed to FLT3 inhibitor, we found seven different JAK family mutations in six of the cases (4.4%), including five bona fide, activating mutations. Except for one patient, the JAK mutations occurred de novo (n = 4) or displayed increasing variant allele frequency after exposure to FLT3 TKI (n = 1). In vitro each of the five activating variants were found to induce resistance to FLT3-ITD inhibition, which was then overcome by dual FLT3/JAK inhibition. In conclusion, our data characterize a novel mechanism of resistance to FLT3-ITD inhibition and may offer a potential therapy, using dual JAK and FLT3 inhibition.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Line, Tumor
  • Drug Resistance, Neoplasm / genetics*
  • Humans
  • Janus Kinases / genetics*
  • Leukemia, Myeloid, Acute / drug therapy
  • Leukemia, Myeloid, Acute / genetics*
  • Mutation / genetics*
  • Protein Kinase Inhibitors / pharmacology
  • Sorafenib / pharmacology
  • Staurosporine / analogs & derivatives
  • Staurosporine / pharmacology
  • Tandem Repeat Sequences / drug effects
  • Tandem Repeat Sequences / genetics*
  • fms-Like Tyrosine Kinase 3 / antagonists & inhibitors*

Substances

  • Protein Kinase Inhibitors
  • Sorafenib
  • FLT3 protein, human
  • fms-Like Tyrosine Kinase 3
  • Janus Kinases
  • Staurosporine
  • midostaurin