Chemical Proteomics Identifies Druggable Vulnerabilities in a Genetically Defined Cancer

Cell. 2017 Oct 19;171(3):696-709.e23. doi: 10.1016/j.cell.2017.08.051. Epub 2017 Sep 28.

Abstract

The transcription factor NRF2 is a master regulator of the cellular antioxidant response, and it is often genetically activated in non-small-cell lung cancers (NSCLCs) by, for instance, mutations in the negative regulator KEAP1. While direct pharmacological inhibition of NRF2 has proven challenging, its aberrant activation rewires biochemical networks in cancer cells that may create special vulnerabilities. Here, we use chemical proteomics to map druggable proteins that are selectively expressed in KEAP1-mutant NSCLC cells. Principal among these is NR0B1, an atypical orphan nuclear receptor that we show engages in a multimeric protein complex to regulate the transcriptional output of KEAP1-mutant NSCLC cells. We further identify small molecules that covalently target a conserved cysteine within the NR0B1 protein interaction domain, and we demonstrate that these compounds disrupt NR0B1 complexes and impair the anchorage-independent growth of KEAP1-mutant cancer cells. Our findings designate NR0B1 as a druggable transcriptional regulator that supports NRF2-dependent lung cancers.

Keywords: KEAP1; NR0B1; NRF2; activity-based profiling; chemical proteomics; covalent; fragment; lung cancer; mass spectrometry.

MeSH terms

  • Carcinoma, Non-Small-Cell Lung / chemistry*
  • Carcinoma, Non-Small-Cell Lung / genetics*
  • Carcinoma, Non-Small-Cell Lung / metabolism
  • Cell Line, Tumor
  • Cysteine / metabolism
  • DAX-1 Orphan Nuclear Receptor / metabolism
  • Gene Regulatory Networks
  • Humans
  • Kelch-Like ECH-Associated Protein 1 / genetics
  • Kelch-Like ECH-Associated Protein 1 / metabolism
  • Ligands
  • Lung Neoplasms / chemistry*
  • Lung Neoplasms / genetics*
  • Lung Neoplasms / metabolism
  • Proteome / analysis*
  • Transcriptome*

Substances

  • DAX-1 Orphan Nuclear Receptor
  • KEAP1 protein, human
  • Kelch-Like ECH-Associated Protein 1
  • Ligands
  • NR0B1 protein, human
  • Proteome
  • Cysteine