Reduced NF1 expression confers resistance to EGFR inhibition in lung cancer

Cancer Discov. 2014 May;4(5):606-19. doi: 10.1158/2159-8290.CD-13-0741. Epub 2014 Feb 17.

Abstract

Activating mutations in the EGF receptor (EGFR) are associated with clinical responsiveness to EGFR tyrosine kinase inhibitors (TKI), such as erlotinib and gefitinib. However, resistance eventually arises, often due to a second EGFR mutation, most commonly T790M. Through a genome-wide siRNA screen in a human lung cancer cell line and analyses of murine mutant EGFR-driven lung adenocarcinomas, we found that erlotinib resistance was associated with reduced expression of neurofibromin, the RAS GTPase-activating protein encoded by the NF1 gene. Erlotinib failed to fully inhibit RAS-ERK signaling when neurofibromin levels were reduced. Treatment of neurofibromin-deficient lung cancers with a MAP-ERK kinase (MEK) inhibitor restored sensitivity to erlotinib. Low levels of NF1 expression were associated with primary and acquired resistance of lung adenocarcinomas to EGFR TKIs in patients. These findings identify a subgroup of patients with EGFR-mutant lung adenocarcinoma who might benefit from combination therapy with EGFR and MEK inhibitors.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / administration & dosage*
  • Antineoplastic Agents / therapeutic use
  • Carcinoma, Non-Small-Cell Lung / drug therapy*
  • Carcinoma, Non-Small-Cell Lung / genetics
  • Cell Line, Tumor
  • Drug Resistance, Neoplasm*
  • Erlotinib Hydrochloride / administration & dosage*
  • Erlotinib Hydrochloride / therapeutic use
  • Humans
  • Lung Neoplasms / drug therapy*
  • Lung Neoplasms / genetics
  • MAP Kinase Signaling System
  • Mice
  • Neoplasms, Experimental
  • Neurofibromin 1 / genetics*
  • Neurofibromin 1 / metabolism
  • Pyridones / administration & dosage*
  • Pyridones / therapeutic use
  • Pyrimidinones / administration & dosage*
  • Pyrimidinones / therapeutic use

Substances

  • Antineoplastic Agents
  • Neurofibromin 1
  • Pyridones
  • Pyrimidinones
  • trametinib
  • Erlotinib Hydrochloride