Neuronal differentiation and cell-cycle programs mediate response to BET-bromodomain inhibition in MYC-driven medulloblastoma

Nat Commun. 2019 Jun 3;10(1):2400. doi: 10.1038/s41467-019-10307-9.

Abstract

BET-bromodomain inhibition (BETi) has shown pre-clinical promise for MYC-amplified medulloblastoma. However, the mechanisms for its action, and ultimately for resistance, have not been fully defined. Here, using a combination of expression profiling, genome-scale CRISPR/Cas9-mediated loss of function and ORF/cDNA driven rescue screens, and cell-based models of spontaneous resistance, we identify bHLH/homeobox transcription factors and cell-cycle regulators as key genes mediating BETi's response and resistance. Cells that acquire drug tolerance exhibit a more neuronally differentiated cell-state and expression of lineage-specific bHLH/homeobox transcription factors. However, they do not terminally differentiate, maintain expression of CCND2, and continue to cycle through S-phase. Moreover, CDK4/CDK6 inhibition delays acquisition of resistance. Therefore, our data provide insights about the mechanisms underlying BETi effects and the appearance of resistance and support the therapeutic use of combined cell-cycle inhibitors with BETi in MYC-amplified medulloblastoma.

Publication types

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

MeSH terms

  • Animals
  • Azepines / pharmacology*
  • Basic Helix-Loop-Helix Transcription Factors / drug effects
  • Basic Helix-Loop-Helix Transcription Factors / metabolism
  • CRISPR-Cas Systems
  • Cell Cycle / drug effects*
  • Cell Cycle Proteins / drug effects
  • Cell Cycle Proteins / metabolism
  • Cell Line, Tumor
  • Cell Lineage
  • Cerebellar Neoplasms / drug therapy*
  • Cerebellar Neoplasms / genetics
  • Cyclin D2 / drug effects
  • Cyclin D2 / metabolism
  • Cyclin-Dependent Kinase 4 / antagonists & inhibitors
  • Cyclin-Dependent Kinase 6 / antagonists & inhibitors
  • Drug Resistance, Neoplasm
  • Gene Expression Profiling
  • Humans
  • Medulloblastoma / drug therapy*
  • Medulloblastoma / genetics
  • Mice
  • Neural Stem Cells / drug effects
  • Neural Stem Cells / metabolism
  • Neurogenesis / drug effects*
  • Proteins / antagonists & inhibitors*
  • Proto-Oncogene Proteins c-myc / genetics
  • S Phase / drug effects
  • Triazoles / pharmacology*

Substances

  • (+)-JQ1 compound
  • Azepines
  • Basic Helix-Loop-Helix Transcription Factors
  • Cell Cycle Proteins
  • Cyclin D2
  • MYC protein, human
  • Proteins
  • Proto-Oncogene Proteins c-myc
  • Triazoles
  • bromodomain and extra-terminal domain protein, human
  • Cyclin-Dependent Kinase 4
  • Cyclin-Dependent Kinase 6