Targeting transcription factors in cancer - from undruggable to reality

Nat Rev Cancer. 2019 Nov;19(11):611-624. doi: 10.1038/s41568-019-0196-7. Epub 2019 Sep 11.

Abstract

Mutated or dysregulated transcription factors represent a unique class of drug targets that mediate aberrant gene expression, including blockade of differentiation and cell death gene expression programmes, hallmark properties of cancers. Transcription factor activity is altered in numerous cancer types via various direct mechanisms including chromosomal translocations, gene amplification or deletion, point mutations and alteration of expression, as well as indirectly through non-coding DNA mutations that affect transcription factor binding. Multiple approaches to target transcription factor activity have been demonstrated, preclinically and, in some cases, clinically, including inhibition of transcription factor-cofactor protein-protein interactions, inhibition of transcription factor-DNA binding and modulation of levels of transcription factor activity by altering levels of ubiquitylation and subsequent proteasome degradation or by inhibition of regulators of transcription factor expression. In addition, several new approaches to targeting transcription factors have recently emerged including modulation of auto-inhibition, proteolysis targeting chimaeras (PROTACs), use of cysteine reactive inhibitors, targeting intrinsically disordered regions of transcription factors and combinations of transcription factor inhibitors with kinase inhibitors to block the development of resistance. These innovations in drug development hold great promise to yield agents with unique properties that are likely to impact future cancer treatment.

Publication types

  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / therapeutic use
  • Catalytic Domain
  • Cell Line, Tumor
  • Cell Proliferation
  • DNA / genetics
  • Drug Design
  • Gene Expression Regulation, Neoplastic*
  • Humans
  • Ligands
  • Mutation
  • Neoplasms / genetics*
  • Neoplasms / metabolism*
  • Phosphorylation
  • Proteasome Endopeptidase Complex
  • Protein Binding
  • Protein Interaction Mapping
  • Protein Processing, Post-Translational
  • Proteolysis
  • Transcription Factors / metabolism*
  • Ubiquitination

Substances

  • Antineoplastic Agents
  • Ligands
  • Transcription Factors
  • DNA
  • Proteasome Endopeptidase Complex