Effect of helical conformation and side chain structure on γ-secretase inhibition by β-peptide foldamers: insight into substrate recognition

J Med Chem. 2013 Feb 28;56(4):1443-54. doi: 10.1021/jm301306c. Epub 2013 Feb 6.

Abstract

Substrate-selective inhibition or modulation of the activity of γ-secretase, which is responsible for the generation of amyloid-β peptides, might be an effective strategy for prevention and treatment of Alzheimer's disease. We have shown that helical β-peptide foldamers are potent and specific inhibitors of γ-secretase. Here we report identification of target site of the foldamers by using a photoaffinity probe. The photoprobe directly and specifically labeled the N-terminal fragment of presenilin 1, in which the initial substrate docking site is predicted to be located. We also optimized the foldamer structure by preparing a variety of derivatives and obtained two highly potent foldamers by incorporation of a hydrophilic and neutral functional group into the parent structure. The class of side chain functional group and the position of incorporation were both important for γ-secretase-inhibitory activity. The substrate selectivity of the inhibitory activity was also quite sensitive to the class of side chain group incorporated.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amyloid Precursor Protein Secretases / antagonists & inhibitors*
  • Amyloid Precursor Protein Secretases / chemistry*
  • Cyclopentanes / chemical synthesis
  • Cyclopentanes / chemistry
  • Cyclopentanes / pharmacology
  • HEK293 Cells
  • Humans
  • Hydrophobic and Hydrophilic Interactions
  • Molecular Mimicry
  • Oligopeptides / chemical synthesis
  • Oligopeptides / chemistry*
  • Oligopeptides / pharmacology
  • Proline / analogs & derivatives
  • Proline / chemical synthesis
  • Proline / chemistry
  • Proline / pharmacology
  • Protein Structure, Secondary
  • Stereoisomerism
  • Substrate Specificity

Substances

  • (1S,2S)-2-aminocyclopentane-1-carboxylic acid
  • Cyclopentanes
  • Oligopeptides
  • Proline
  • Amyloid Precursor Protein Secretases