Genome-wide CRISPR screen identifies regulators of MAPK and MTOR pathways mediating sorafenib resistance in acute myeloid leukemia

Haematologica. 2022 Jan 1;107(1):77-85. doi: 10.3324/haematol.2020.257964.

Abstract

Drug resistance impedes the long-term effect of targeted therapies in acute myeloid leukemia (AML), necessitating the identification of mechanisms underlying resistance. Approximately 25% of AML patients carry FLT3 mutations and develop post-treatment insensitivity to FLT3 inhibitors, including sorafenib. Using a genome-wide CRISPR screen, we identified LZTR1, NF1, TSC1 or TSC2, negative regulators of the MAPK and MTOR pathways, as mediators of sorafenib resistance. Analyses of ex vivo drug sensitivity assays in FLT3-ITD AML patient samples revealed lower expression of LZTR1, NF1, and TSC2 correlated with sorafenib sensitivity. Importantly, MAPK and/or MTOR complex1 (MTORC1) activity were upregulated in AML cells made resistant to several FLT3 inhibitors, including crenolanib, quizartinib, or sorafenib. These cells were sensitive to MEK inhibitors, and the combination of FLT3 and MEK inhibitors showed enhanced efficacy, suggesting its effectiveness in AML patients with FLT3 mutations and those with resistance to FLT3 inhibitors.

Publication types

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

MeSH terms

  • Antineoplastic Agents* / pharmacology
  • Antineoplastic Agents* / therapeutic use
  • Cell Line, Tumor
  • Clustered Regularly Interspaced Short Palindromic Repeats
  • Humans
  • Leukemia, Myeloid, Acute* / drug therapy
  • Leukemia, Myeloid, Acute* / genetics
  • MAP Kinase Signaling System
  • Mutation
  • Niacinamide / pharmacology
  • Niacinamide / therapeutic use
  • Phenylurea Compounds / pharmacology
  • Phenylurea Compounds / therapeutic use
  • Protein Kinase Inhibitors / pharmacology
  • Protein Kinase Inhibitors / therapeutic use
  • Sorafenib* / pharmacology
  • TOR Serine-Threonine Kinases / genetics
  • Transcription Factors
  • fms-Like Tyrosine Kinase 3 / genetics

Substances

  • Antineoplastic Agents
  • LZTR1 protein, human
  • Phenylurea Compounds
  • Protein Kinase Inhibitors
  • Transcription Factors
  • Niacinamide
  • Sorafenib
  • MTOR protein, human
  • fms-Like Tyrosine Kinase 3
  • TOR Serine-Threonine Kinases