Potent γ-secretase inhibitors/modulators interact with amyloid-β fibrils but do not inhibit fibrillation: a high-resolution NMR study

Biochem Biophys Res Commun. 2014 May 16;447(4):590-5. doi: 10.1016/j.bbrc.2014.04.041. Epub 2014 Apr 18.

Abstract

Recently, γ-secretase modulators (GSM) have been shown to interact directly with the amyloid precursor protein (APP) and simultaneously inhibit the activity of the Presenilin domain of γ-secretase. A clear understanding of the molecular recognition pathways by which GSM can target both γ-secretase and Aβ precursor protein can lead to the development of more effective inhibitors. To examine whether this direct interaction with APP affects the downstream Aβ fibril formation, we chose to investigate three different molecules in this study: Sulindac sulfide, Semagacestat and E2012 from the class of generation I GSMs, γ-secretase inhibitors (GSI), and generation II GSM molecules, respectively. Firstly, through NMR based ligand titration, we identified that Sulindac sulfide and Semagacestat interact strongly with Aβ40 monomers, whereas E2012 does not. Secondly, using saturation transfer difference (STD) NMR experiments, we found that all three molecules bind equally well with Aβ40 fibrils. To determine if these interactions with the monomer/fibril lead to a viable inhibition of the fibrillation process, we designed an NMR based time-dependent assay and accurately distinguished the inhibitors from the non-inhibitors within a short period of 12h. Based on this pre-seeded fibril assay, we conclude that none of these molecules inhibit the ongoing fibrillation, rather ligands such as Semagacestat and E2012 accelerated the rate of aggregation.

Keywords: Abeta40; GSM; STD NMR; Sulindac sulfide NMR fibril assay.

MeSH terms

  • Alanine / analogs & derivatives
  • Alanine / pharmacology
  • Amyloid / chemistry
  • Amyloid / metabolism
  • Amyloid Precursor Protein Secretases / antagonists & inhibitors*
  • Amyloid Precursor Protein Secretases / metabolism*
  • Amyloid beta-Peptides / chemistry*
  • Amyloid beta-Peptides / metabolism*
  • Amyloid beta-Protein Precursor / chemistry
  • Amyloid beta-Protein Precursor / metabolism
  • Azepines / pharmacology
  • Binding Sites
  • Humans
  • Imidazoles / pharmacology
  • Kinetics
  • Models, Molecular
  • Nuclear Magnetic Resonance, Biomolecular
  • Peptide Fragments / chemistry
  • Peptide Fragments / metabolism
  • Piperidines / pharmacology
  • Protease Inhibitors / pharmacology*
  • Protein Conformation
  • Protein Multimerization / drug effects
  • Sulindac / analogs & derivatives
  • Sulindac / pharmacology

Substances

  • 1-(1-(4-fluorophenyl)ethyl)-3-((3-methoxy-4-(4-methyl-1H-imidazol-1-yl)phenyl)methylene)-2-piperidinone
  • Amyloid
  • Amyloid beta-Peptides
  • Amyloid beta-Protein Precursor
  • Azepines
  • Imidazoles
  • N2-((2S)-2-(3,5-difluorophenyl)-2-hydroxyethanoyl)-N1-((7S)-5-methyl-6-oxo-6,7-dihydro-5H-dibenzo(b,d)azepin-7-yl)-L-alaninamide
  • Peptide Fragments
  • Piperidines
  • Protease Inhibitors
  • amyloid beta-protein (1-40)
  • Sulindac
  • sulindac sulfide
  • Amyloid Precursor Protein Secretases
  • Alanine