BMS-708,163 targets presenilin and lacks notch-sparing activity

Biochemistry. 2012 Sep 18;51(37):7209-11. doi: 10.1021/bi301137h. Epub 2012 Sep 4.

Abstract

The "Notch-sparing" γ-secretase inhibitor (GSI) BMS-708,163 (Avagacestat) is currently in phase II clinical trials for Alzheimer's disease. Unlike previously failed GSIs, BMS-708,163 is considered to be a promising drug candidate because of its reported Notch-sparing activity for the inhibition of Aβ production over Notch cleavage. We now report that BMS-708,163 binds directly to the presenilin-1 N-terminal fragment and that binding can be challenged by other pan-GSIs, but not by γ-secretase modulators. Furthermore, BMS-708,163 blocks the binding of four different active site-directed GSI photoaffinity probes. We therefore report that this compound acts as a nonselective γ-secretase inhibitor.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alzheimer Disease / drug therapy
  • Alzheimer Disease / genetics
  • Alzheimer Disease / metabolism
  • Amyloid Precursor Protein Secretases / antagonists & inhibitors
  • Amyloid Precursor Protein Secretases / genetics
  • Amyloid Precursor Protein Secretases / metabolism
  • Amyloid beta-Protein Precursor / genetics
  • Amyloid beta-Protein Precursor / metabolism*
  • Catalytic Domain
  • Clinical Trials, Phase II as Topic
  • HeLa Cells
  • Humans
  • Oxadiazoles / pharmacology*
  • Presenilins / antagonists & inhibitors*
  • Presenilins / genetics
  • Presenilins / metabolism
  • Protein Binding / drug effects
  • Receptors, Notch / genetics
  • Receptors, Notch / metabolism*
  • Sulfonamides / pharmacology*

Substances

  • APP protein, human
  • Amyloid beta-Protein Precursor
  • BMS 708163
  • Oxadiazoles
  • Presenilins
  • Receptors, Notch
  • Sulfonamides
  • Amyloid Precursor Protein Secretases