The BCL-2 selective inhibitor ABT-199 sensitizes soft tissue sarcomas to proteasome inhibition by a concerted mechanism requiring BAX and NOXA

Cell Death Dis. 2020 Aug 24;11(8):701. doi: 10.1038/s41419-020-02910-2.

Abstract

Soft tissue sarcomas (STS) are a heterogeneous group of malignancies predominantly affecting children and young adults. Despite improvements in multimodal therapies, 5-year survival rates are only 50% and new treatment options in STS are urgently needed. To develop a rational combination therapy for the treatment of STS we focused on ABT-199 (Venetoclax), a BCL-2 specific BH3-mimetic, in combination with the proteasome inhibitor bortezomib (BZB). Simultaneous inhibition of BCL-2 and the proteasome resulted in strongly synergistic apoptosis induction. Mechanistically, ABT-199 mainly affected the multidomain effector BAX by liberating it from BCL-2 inhibition. The combination with BZB additionally resulted in the accumulation of BOK, a BAX/BAK homologue, and of the BH3-only protein NOXA, which inhibits the anti-apoptotic protein MCL-1. Thus, the combination of ABT-199 and BZB sensitizes STS cells to apoptosis by simultaneously releasing several defined apoptotic restraints. This synergistic mechanism of action was verified by CRISPR/Cas9 knock-out, showing that both BAX and NOXA are crucial for ABT-199/BZB-induced apoptosis. Noteworthy, efficient induction of apoptosis by ABT-199/BZB was not affected by the p53 status and invariably detected in cell lines and patient-derived tumor cells of several sarcoma types, including rhabdomyo-, leiomyo-, lipo-, chondro-, osteo-, or synovial sarcomas. Hence, we propose the combination of ABT-199 and BZB as a promising strategy for the treatment of STS, which should warrant further clinical investigation.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents / pharmacology
  • Apoptosis / drug effects
  • Apoptosis Regulatory Proteins
  • Biphenyl Compounds / pharmacology
  • Bortezomib / pharmacology*
  • Bridged Bicyclo Compounds, Heterocyclic / metabolism
  • Bridged Bicyclo Compounds, Heterocyclic / pharmacology*
  • Cell Line, Tumor
  • Drug Synergism
  • Female
  • Humans
  • Male
  • Middle Aged
  • Myeloid Cell Leukemia Sequence 1 Protein / metabolism
  • Nitrophenols / pharmacology
  • Proteasome Endopeptidase Complex / drug effects
  • Proteasome Inhibitors / metabolism
  • Proto-Oncogene Proteins c-bcl-2 / metabolism
  • Sarcoma / drug therapy*
  • Sarcoma / metabolism
  • Sulfonamides / metabolism
  • Sulfonamides / pharmacology*
  • bcl-2-Associated X Protein / metabolism
  • bcl-X Protein / metabolism

Substances

  • Antineoplastic Agents
  • Apoptosis Regulatory Proteins
  • BCL2 protein, human
  • Biphenyl Compounds
  • Bridged Bicyclo Compounds, Heterocyclic
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Nitrophenols
  • PMAIP1 protein, human
  • Proteasome Inhibitors
  • Proto-Oncogene Proteins c-bcl-2
  • Sulfonamides
  • bcl-2-Associated X Protein
  • bcl-X Protein
  • Bortezomib
  • Proteasome Endopeptidase Complex
  • venetoclax