Combined treatment with CBP and BET inhibitors reverses inadvertent activation of detrimental super enhancer programs in DIPG cells

Cell Death Dis. 2020 Aug 21;11(8):673. doi: 10.1038/s41419-020-02800-7.

Abstract

Diffuse intrinsic pontine gliomas (DIPG) are the most aggressive brain tumors in children with 5-year survival rates of only 2%. About 85% of all DIPG are characterized by a lysine-to-methionine substitution in histone 3, which leads to global H3K27 hypomethylation accompanied by H3K27 hyperacetylation. Hyperacetylation in DIPG favors the action of the Bromodomain and Extra-Terminal (BET) protein BRD4, and leads to the reprogramming of the enhancer landscape contributing to the activation of DIPG super enhancer-driven oncogenes. The activity of the acetyltransferase CREB-binding protein (CBP) is enhanced by BRD4 and associated with acetylation of nucleosomes at super enhancers (SE). In addition, CBP contributes to transcriptional activation through its function as a scaffold and protein bridge. Monotherapy with either a CBP (ICG-001) or BET inhibitor (JQ1) led to the reduction of tumor-related characteristics. Interestingly, combined treatment induced strong cytotoxic effects in H3.3K27M-mutated DIPG cell lines. RNA sequencing and chromatin immunoprecipitation revealed that these effects were caused by the inactivation of DIPG SE-controlled tumor-related genes. However, single treatment with ICG-001 or JQ1, respectively, led to activation of a subgroup of detrimental super enhancers. Combinatorial treatment reversed the inadvertent activation of these super enhancers and rescued the effect of ICG-001 and JQ1 single treatment on enhancer-driven oncogenes in H3K27M-mutated DIPG, but not in H3 wild-type pedHGG cells. In conclusion, combinatorial treatment with CBP and BET inhibitors is highly efficient in H3K27M-mutant DIPG due to reversal of inadvertent activation of detrimental SE programs in comparison with monotherapy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetylation
  • Astrocytoma / genetics
  • Azepines / pharmacology*
  • Brain Stem Neoplasms / genetics
  • Bridged Bicyclo Compounds, Heterocyclic / pharmacology*
  • CREB-Binding Protein / antagonists & inhibitors
  • CREB-Binding Protein / metabolism
  • Cell Cycle Proteins / antagonists & inhibitors
  • Cell Cycle Proteins / metabolism
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Diffuse Intrinsic Pontine Glioma / drug therapy*
  • Diffuse Intrinsic Pontine Glioma / metabolism
  • Gene Expression Regulation, Neoplastic / drug effects
  • Glioma / genetics
  • Histones / genetics
  • Histones / metabolism
  • Humans
  • Mutation / genetics
  • Nuclear Proteins / metabolism
  • Nucleosomes / metabolism
  • Proteins / antagonists & inhibitors
  • Proteins / metabolism
  • Pyrimidinones / pharmacology*
  • Transcription Factors / antagonists & inhibitors
  • Transcription Factors / metabolism
  • Transcriptional Activation / drug effects
  • Triazoles / pharmacology*

Substances

  • (+)-JQ1 compound
  • Azepines
  • BRD4 protein, human
  • Bridged Bicyclo Compounds, Heterocyclic
  • Cell Cycle Proteins
  • Histones
  • ICG 001
  • Nuclear Proteins
  • Nucleosomes
  • Proteins
  • Pyrimidinones
  • Transcription Factors
  • Triazoles
  • bromodomain and extra-terminal domain protein, human
  • CREB-Binding Protein
  • CREBBP protein, human