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Design and development of drugs for Alzheimer’s dementia as a protein misfolding disorder

Published online by Cambridge University Press:  03 June 2015

C Barden
Affiliation:
(Toronto)
F Meier-Stephenson
Affiliation:
(Toronto)
MD Carter
Affiliation:
(Halifax)
S Banfield
Affiliation:
(Halifax)
EC Diez
Affiliation:
(Toronto)
B Kelly
Affiliation:
(Toronto)
E Lu
Affiliation:
(Toronto)
A Meek
Affiliation:
(Toronto)
V Meier-Stephenson
Affiliation:
(Halifax)
L Pan
Affiliation:
(Toronto)
GA Simms
Affiliation:
(Halifax)
B Sweeting
Affiliation:
(Toronto)
Y Wang
Affiliation:
(Toronto)
F Wu
Affiliation:
(Toronto)
A Yadav
Affiliation:
(Toronto)
S Yang
Affiliation:
(Toronto)
M Taylor
Affiliation:
(Toronto)
M Reed
Affiliation:
(Toronto)
DF Weaver
Affiliation:
(Toronto)
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Abstract

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Background: There are no disease modifying agents for the treatment of Alzheimer’s disease (AD). Pathologically, AD is associated with the misfolding of two peptides: beta-amyloid (plaques) and tau (tangles). Methods: Using large-scale computer simulations, we modelled the misfolding of both beta-amyloid and tau, identifying a common conformational motif (CCM; i.e. an abnormal peptide shape), present in both beta-amyloid and tau, that promotes their misfolding. We screened a library of 11.8 million compounds against this in silico model of protein misfolding, identifying three novel molecular classes of putative therapeutics as anti-protein misfolding agents. We synthesized approximately 400 new chemical entity drug-like molecules in each of these three classes (i.e. 1200 potential drug candidates). These were comprehensively screened in a battery of five in vitro protein oligomerization assays. Selected compounds were next evaluated in the APP/PS1 doubly transgenic mouse model of AD. Results: Two new classes of molecules were identified with the ability to block the oligomerization of both beta-amyloid and tau. These compounds are drug-like with good pharmacokinetic properties and are brain-penetrant. They exhibit excellent efficacy in transgenic mouse models. Conclusion: Computer aided drug design has enabled the discovery of novel drug-like molecules able to inhibit both tau and beta-amyloid misfolding.

Type
CNS / CSCN Platform Presentations
Copyright
Copyright © The Canadian Journal of Neurological Sciences Inc. 2015