Selective killing of K-ras mutant cancer cells by small molecule inducers of oxidative stress

Proc Natl Acad Sci U S A. 2011 May 24;108(21):8773-8. doi: 10.1073/pnas.1105941108. Epub 2011 May 9.

Abstract

Activating K-RAS mutations are the most frequent oncogenic mutations in human cancer. Numerous downstream signaling pathways have been shown to be deregulated by oncogenic K-ras. However, to date there are still no effective targeted therapies for this genetically defined subset of patients. Here we report the results of a small molecule, synthetic lethal screen using mouse embryonic fibroblasts derived from a mouse model harboring a conditional oncogenic K-ras(G12D) allele. Among the >50,000 compounds screened, we identified a class of drugs with selective activity against oncogenic K-ras-expressing cells. The most potent member of this class, lanperisone, acts by inducing nonapoptotic cell death in a cell cycle- and translation-independent manner. The mechanism of cell killing involves the induction of reactive oxygen species that are inefficiently scavenged in K-ras mutant cells, leading to oxidative stress and cell death. In mice, treatment with lanperisone suppresses the growth of K-ras-driven tumors without overt toxicity. Our findings establish the specific antitumor activity of lanperisone and reveal oxidative stress pathways as potential targets in Ras-mediated malignancies.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology
  • Cell Death / drug effects
  • Cells, Cultured
  • Drug Evaluation, Preclinical
  • Fibroblasts / cytology
  • Mice
  • Neoplasms / drug therapy*
  • Neoplasms / metabolism
  • Neoplasms / pathology
  • Oxidative Stress / drug effects*
  • Reactive Oxygen Species / agonists
  • ras Proteins*

Substances

  • Antineoplastic Agents
  • Reactive Oxygen Species
  • ras Proteins