Small-molecule Bax agonists for cancer therapy

Nat Commun. 2014 Sep 17:5:4935. doi: 10.1038/ncomms5935.

Abstract

Bax, a central death regulator, is required at the decisional stage of apoptosis. We recently identified serine 184 (S184) of Bax as a critical functional switch controlling its proapoptotic activity. Here we used the structural pocket around S184 as a docking site to screen the NCI library of small molecules using the UCSF-DOCK programme suite. Three compounds, small-molecule Bax agonists SMBA1, SMBA2 and SMBA3, induce conformational changes in Bax by blocking S184 phosphorylation, facilitating Bax insertion into mitochondrial membranes and forming Bax oligomers. The latter leads to cytochrome c release and apoptosis in human lung cancer cells, which occurs in a Bax- but not Bak-dependent fashion. SMBA1 potently suppresses lung tumour growth via apoptosis by selectively activating Bax in vivo without significant normal tissue toxicity. Development of Bax agonists as a new class of anticancer drugs offers a strategy for the treatment of lung cancer and other Bax-expressing malignancies.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents / chemistry
  • Apoptosis
  • Binding Sites
  • Cell Line
  • Cell Line, Tumor
  • Drug Design
  • Drug Screening Assays, Antitumor
  • Fibroblasts / metabolism
  • Humans
  • Lung Neoplasms / drug therapy
  • Male
  • Mice
  • Mice, Nude
  • Molecular Dynamics Simulation
  • Neoplasms / drug therapy*
  • Phosphorylation
  • Protein Multimerization
  • Serine / chemistry
  • bcl-2-Associated X Protein / agonists*
  • bcl-2-Associated X Protein / chemistry

Substances

  • Antineoplastic Agents
  • BAX protein, human
  • bcl-2-Associated X Protein
  • Serine