Evaluation of difluoromethyl ketones as agonists of the γ-aminobutyric acid type B (GABAB) receptor

J Med Chem. 2013 Mar 28;56(6):2456-65. doi: 10.1021/jm301805e. Epub 2013 Mar 6.

Abstract

The design, synthesis, biological evaluation, and in vivo studies of difluoromethyl ketones as GABAB agonists that are not structurally analogous to known GABAB agonists, such as baclofen or 3-aminopropyl phosphinic acid, are presented. The difluoromethyl ketones were assembled in three synthetic steps using a trifluoroacetate-release aldol reaction. Following evaluation at clinically relevant GABA receptors, we have identified a difluoromethyl ketone that is a potent GABAB agonist, obtained its X-ray structure, and presented preliminary in vivo data in alcohol-preferring mice. The behavioral studies in mice demonstrated that this compound tended to reduce the acoustic startle response, which is consistent with an anxiolytic profile. Structure-activity investigations determined that replacing the fluorines of the difluoromethyl ketone with hydrogens resulted in an inactive analogue. Resolution of the individual enantiomers of the difluoromethyl ketone provided a compound with full biological activity at concentrations less than an order of magnitude greater than the pharmaceutical, baclofen.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Behavior, Animal / drug effects
  • Female
  • GABA-B Receptor Agonists / chemistry*
  • GABA-B Receptor Agonists / pharmacology*
  • Halogenation
  • Ketones / chemistry*
  • Ketones / pharmacology*
  • Male
  • Mice
  • Models, Molecular
  • Protein Conformation
  • Receptors, GABA-B / chemistry
  • Receptors, GABA-B / metabolism*

Substances

  • GABA-B Receptor Agonists
  • Ketones
  • Receptors, GABA-B