Identification of a low-molecular weight TrkB antagonist with anxiolytic and antidepressant activity in mice

J Clin Invest. 2011 May;121(5):1846-57. doi: 10.1172/JCI43992. Epub 2011 Apr 18.

Abstract

The neurotrophin brain-derived neurotrophic factor (BDNF) and its receptor tropomyosin-related kinase B (TrkB) have emerged as key mediators in the pathophysiology of several mood disorders, including anxiety and depression. However, therapeutic compounds that interact with TrkB receptors have been difficult to develop. Using a combination of structure-based in silico screening and high-capacity functional assays in recombinant and neuronal cells, we identified a low-molecular weight TrkB ligand (ANA-12) that prevented activation of the receptor by BDNF with a high potency. ANA-12 showed direct and selective binding to TrkB and inhibited processes downstream of TrkB without altering TrkA and TrkC functions. KIRA-ELISA analysis demonstrated that systemic administration of ANA-12 to adult mice decreased TrkB activity in the brain without affecting neuronal survival. Mice administered ANA-12 demonstrated reduced anxiety- and depression-related behaviors on a variety of tests predictive of anxiolytic and antidepressant properties in humans. This study demonstrates that structure-based virtual screening strategy can be an efficient method for discovering potent TrkB-selective ligands that are active in vivo. We further propose that ANA-12 may be a valuable tool for studying BDNF/TrkB signaling and may constitute a lead compound for developing the next generation of therapeutic agents for the treatment of mood disorders.

MeSH terms

  • Animals
  • Anti-Anxiety Agents / pharmacology*
  • Antidepressive Agents / pharmacology*
  • Anxiety / metabolism
  • Azepines / chemistry
  • Azepines / pharmacology*
  • Behavior, Animal
  • Benzamides / chemistry
  • Benzamides / pharmacology*
  • Brain / metabolism
  • Brain-Derived Neurotrophic Factor / metabolism
  • Chemistry, Pharmaceutical / methods
  • Depression / metabolism
  • Drug Design
  • Enzyme-Linked Immunosorbent Assay / methods
  • Humans
  • Ligands
  • Mice
  • Molecular Weight
  • Mood Disorders / drug therapy
  • Neurons / metabolism
  • Receptor, trkA / metabolism
  • Receptor, trkB / metabolism*
  • Signal Transduction

Substances

  • ANA 12 compound
  • Anti-Anxiety Agents
  • Antidepressive Agents
  • Azepines
  • Benzamides
  • Brain-Derived Neurotrophic Factor
  • Ligands
  • Receptor, trkA
  • Receptor, trkB