The design and synthesis of human branched-chain amino acid aminotransferase inhibitors for treatment of neurodegenerative diseases

Bioorg Med Chem Lett. 2006 May 1;16(9):2337-40. doi: 10.1016/j.bmcl.2005.07.058. Epub 2005 Sep 6.

Abstract

The inhibition of the cytosolic isoenzyme BCAT that is expressed specifically in neuronal tissue is likely to be useful for the treatment of neurodegenerative and other neurological disorders where glutamatergic mechanisms are implicated. Compound 2 exhibited an IC50 of 0.8 microM in the hBCATc assays; it is an active and selective inhibitor. Inhibitor 2 also blocked calcium influx into neuronal cells following inhibition of glutamate uptake, and demonstrated neuroprotective efficacy in vivo. SAR, pharmacology, and the crystal structure of hBCATc with inhibitor 2 are described.

MeSH terms

  • Animals
  • Benzofurans / chemical synthesis*
  • Benzofurans / chemistry
  • Benzofurans / therapeutic use*
  • Calcium / antagonists & inhibitors
  • Calcium / metabolism
  • Cells, Cultured
  • Crystallography, X-Ray
  • Drug Design
  • Drug Evaluation, Preclinical
  • Enzyme Inhibitors / chemical synthesis*
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / therapeutic use*
  • Glutamic Acid / drug effects
  • Glutamic Acid / metabolism
  • Humans
  • In Vitro Techniques
  • Models, Molecular
  • Molecular Structure
  • Neurodegenerative Diseases / drug therapy*
  • Neurons / cytology
  • Neurons / drug effects
  • Rats
  • Rats, Inbred Lew
  • Stereoisomerism
  • Structure-Activity Relationship
  • Sulfonamides / chemical synthesis*
  • Sulfonamides / chemistry
  • Sulfonamides / therapeutic use*
  • Transaminases / antagonists & inhibitors*

Substances

  • Benzofurans
  • Enzyme Inhibitors
  • Sulfonamides
  • Glutamic Acid
  • Transaminases
  • branched-chain-amino-acid transaminase
  • Calcium