Non-redox-active lipoate derivates disrupt cancer cell mitochondrial metabolism and are potent anticancer agents in vivo

J Mol Med (Berl). 2011 Nov;89(11):1137-48. doi: 10.1007/s00109-011-0785-8. Epub 2011 Jul 19.

Abstract

We report the analysis of CPI-613, the first member of a large set of analogs of lipoic acid (lipoate) we have investigated as potential anticancer agents. CPI-613 strongly disrupts mitochondrial metabolism, with selectivity for tumor cells in culture. This mitochondrial disruption includes activation of the well-characterized, lipoate-responsive regulatory phosphorylation of the E1α pyruvate dehydrogenase (PDH) subunit. This phosphorylation inactivates flux of glycolysis-derived carbon through this enzyme complex and implicates the PDH regulatory kinases (PDKs) as a possible drug target. Supporting this hypothesis, RNAi knockdown of the PDK protein levels substantially attenuates CPI-613 cancer cell killing. In both cell culture and in vivo tumor environments, the observed strong mitochondrial metabolic disruption is expected to significantly compromise cell survival. Consistent with this prediction, CPI-613 disruption of tumor mitochondrial metabolism is followed by efficient commitment to cell death by multiple, apparently redundant pathways, including apoptosis, in all tested cancer cell lines. Further, CPI-613 shows strong antitumor activity in vivo against human non-small cell lung and pancreatic cancers in xenograft models with low side-effect toxicity.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology*
  • Antioxidants / pharmacology
  • Caprylates / chemistry
  • Caprylates / pharmacology*
  • Cell Line, Tumor
  • Drug Screening Assays, Antitumor
  • Gene Knockdown Techniques
  • Humans
  • Mice
  • Mitochondria / enzymology*
  • Neoplasm Transplantation
  • Neoplasms / drug therapy*
  • Neoplasms / enzymology
  • Neoplasms / genetics
  • Oxidation-Reduction / drug effects
  • Oxidative Phosphorylation / drug effects*
  • Pyruvate Dehydrogenase Complex / genetics
  • Pyruvate Dehydrogenase Complex / metabolism*
  • Sulfides / chemistry
  • Sulfides / pharmacology*
  • Thioctic Acid / chemistry
  • Thioctic Acid / pharmacology*
  • Transplantation, Heterologous

Substances

  • Antineoplastic Agents
  • Antioxidants
  • Caprylates
  • Pyruvate Dehydrogenase Complex
  • Sulfides
  • Thioctic Acid
  • devimistat