Amylovis-201 is a new dual-target ligand, acting as an anti-amyloidogenic compound and a potent agonist of the σ (1) chaperone protein

Amylovis-201 是一种新型双靶点配体,既具有抗淀粉样蛋白生成作用,又是 σ(1) 分子伴侣蛋白的强效激动剂。

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Abstract

The aggregation of Amyloid-β (Aβ) peptides is associated with neurodegeneration in Alzheimer's disease (AD). We previously identified novel naphtalene derivatives, including the lead compound Amylovis-201, able to form thermodynamically stable complexes with Aβ species, peptides and fibrils. As the drug showed a chemical scaffold coherent for an effective interaction with the σ (1) receptor chaperone and as σ (1) agonists are currently developed as potent neuroprotectants in AD, we investigated the pharmacological action of Amylovis-201 on the σ (1) receptor. We report that Amylovis-201 is a potent σ (1) agonist by several in silico, in vitro and in vivo assays and that its anti-amnesic and neuroprotective effects involve a pharmacological action at σ (1) receptors. Furthermore, we show for the first time that classical σ (1) receptor agonist (PRE-084), and antagonist (NE-100) are able to interact and disaggregate Aβ (25-35) fibrils. Interestingly, Amylovis-201 was the only compound inhibiting Aβ (25-35) aggregates formation. Our results therefore highlight a dual action of Amylovis-201 as anti-aggregating agent and σ (1) receptor agonist that could be highly effective in long-term treatment against neurodegeneration in AD.

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