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Developing BACE-1 inhibitors for FXS

Cara J Westmark1, Elizabeth M Berry-Kravis, Chrysanthy Ikonomidou

  • 1Department of Neurology, University of Wisconsin Madison, WI, USA.

Frontiers in Cellular Neuroscience
|June 12, 2013
PubMed

Insights

Fragile X syndrome (FXS) may benefit from Alzheimer's disease (AD) drugs. Targeting amyloid precursor protein (APP) and BACE-1 may restore synaptic balance in FXS patients.

Area of Science:

  • Neuroscience
  • Genetics
  • Pharmacology

Background:

  • Fragile X syndrome (FXS) is a genetic disorder with limited treatment options.
  • FXS involves overactive metabotropic glutamate receptor 5 signaling, leading to synaptic protein overexpression.
  • Amyloid precursor protein (APP) and amyloid-beta are overexpressed in FXS, similar to Alzheimer's disease (AD).

Purpose of the Study:

  • To explore the potential of AD therapeutics for FXS treatment.
  • To investigate if modulating APP and its metabolites can be a viable strategy for FXS.

Main Methods:

  • Reviewing existing research on FXS and AD.
  • Identifying potential drug targets common to both diseases, specifically APP and BACE-1.
  • Considering the use of acetyltransferase inhibitors to reduce BACE-1 activity.

Main Results:

  • Excessive APP and amyloid-beta are implicated in FXS pathophysiology.
  • Pharmaceuticals targeting APP and BACE-1 in AD research show promise for FXS.

Conclusions:

  • Modulating APP catabolites via BACE-1 inhibition is a potential therapeutic avenue for FXS.
  • Repurposing AD drugs could offer new hope for treating FXS.
Keywords:
Alzheimer’s diseaseBACE-1amyloid-betafragile X syndromemGluR5

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