Mechanism of action of compound-13: an α1-selective small molecule activator of AMPK

Chem Biol. 2014 Jul 17;21(7):866-79. doi: 10.1016/j.chembiol.2014.05.014.

Abstract

AMPK is a sensor of cellular energy status and a promising target for drugs aimed at metabolic disorders. We have studied the selectivity and mechanism of a recently described activator, C2, and its cell-permeable prodrug, C13. C2 was a potent allosteric activator of α1-complexes that, like AMP, also protected against Thr172 dephosphorylation. Compared with AMP, C2 caused only partial allosteric activation of α2-complexes and failed to protect them against dephosphorylation. We show that both effects could be fully restored by exchanging part of the linker between the autoinhibitory and C-terminal domains in α2, containing the equivalent region from α1 thought to interact with AMP bound in site 3 of the γ subunit. Consistent with our results in cell-free assays, C13 potently inhibited lipid synthesis in hepatocytes from wild-type and was largely ineffective in AMPK-knockout hepatocytes; its effects were more severely affected by knockout of α1 than of α2, β1, or β2.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • AMP-Activated Protein Kinases / chemistry
  • AMP-Activated Protein Kinases / metabolism*
  • Adenosine Monophosphate / pharmacology
  • Amino Acid Sequence
  • Animals
  • Enzyme Activation / drug effects
  • Enzyme Activators / metabolism
  • Enzyme Activators / pharmacology*
  • Esterification / drug effects
  • Fatty Acids / metabolism
  • Hepatocytes / cytology
  • Hepatocytes / drug effects
  • Lipogenesis / drug effects
  • Mice
  • Molecular Sequence Data
  • Prodrugs / metabolism
  • Prodrugs / pharmacology
  • Protein Subunits / agonists
  • Protein Subunits / chemistry
  • Protein Subunits / metabolism
  • Recombinant Proteins / chemistry
  • Recombinant Proteins / metabolism
  • Signal Transduction / drug effects
  • Small Molecule Libraries / metabolism
  • Small Molecule Libraries / pharmacology*
  • Substrate Specificity

Substances

  • Enzyme Activators
  • Fatty Acids
  • Prodrugs
  • Protein Subunits
  • Recombinant Proteins
  • Small Molecule Libraries
  • Adenosine Monophosphate
  • AMP-Activated Protein Kinases