Chemical genomics identifies small-molecule MCL1 repressors and BCL-xL as a predictor of MCL1 dependency

Cancer Cell. 2012 Apr 17;21(4):547-62. doi: 10.1016/j.ccr.2012.02.028.

Abstract

MCL1, which encodes the antiapoptotic protein MCL1, is among the most frequently amplified genes in human cancer. A chemical genomic screen identified compounds, including anthracyclines, that decreased MCL1 expression. Genomic profiling indicated that these compounds were global transcriptional repressors that preferentially affect MCL1 due to its short mRNA half-life. Transcriptional repressors and MCL1 shRNAs induced apoptosis in the same cancer cell lines and could be rescued by physiological levels of ectopic MCL1 expression. Repression of MCL1 released the proapoptotic protein BAK from MCL1, and Bak deficiency conferred resistance to transcriptional repressors. A computational model, validated in vivo, indicated that high BCL-xL expression confers resistance to MCL1 repression, thereby identifying a patient-selection strategy for the clinical development of MCL1 inhibitors.

Publication types

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

MeSH terms

  • Animals
  • Apoptosis / genetics
  • Cell Line, Tumor
  • Gene Expression Regulation, Neoplastic
  • Gene Silencing
  • Genomics
  • Humans
  • Mice
  • Models, Genetic
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Proto-Oncogene Proteins c-bcl-2 / antagonists & inhibitors
  • Proto-Oncogene Proteins c-bcl-2 / genetics*
  • RNA, Small Interfering
  • Small Molecule Libraries
  • bcl-2 Homologous Antagonist-Killer Protein / genetics
  • bcl-2 Homologous Antagonist-Killer Protein / metabolism
  • bcl-2 Homologous Antagonist-Killer Protein / physiology
  • bcl-X Protein / physiology*

Substances

  • BAK1 protein, human
  • Mcl1 protein, mouse
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Proto-Oncogene Proteins c-bcl-2
  • RNA, Small Interfering
  • Small Molecule Libraries
  • bcl-2 Homologous Antagonist-Killer Protein
  • bcl-X Protein