Discovery and Biological Evaluation of a Novel Highly Potent Selective Butyrylcholinsterase Inhibitor

Journal of Medicinal Chemistry
2020.0

Abstract

To discover novel BChE inhibitors, a hierarchical virtual screening protocol followed by biochemical evaluation was applied. The most potent compound <b>8012-9656</b> (<i>eq</i>BChE IC<sub>50</sub> = 0.18 ± 0.03 μM, <i>h</i>BChE IC<sub>50</sub> = 0.32 ± 0.07 μM) was purchased and synthesized. It inhibited BChE in a noncompetitive manner and could occupy the binding pocket forming diverse interactions with the target. <b>8012-9656</b> was proven to be safe in vivo and in vitro and showed comparable performance in ameliorating the scopolamine-induced cognition impairment to tacrine. Additionally, treatment with <b>8012-9656</b> could almost entirely recover the Aβ<sub>1-42</sub> (icv)-impaired cognitive function to the normal level and showed better behavioral performance than donepezil. The evaluation of the Aβ<sub>1-42</sub> total amount confirmed its anti-amyloidogenic profile. Moreover, <b>8012-9656</b> possessed blood-brain barrier (BBB) penetrating ability, a long <i>T</i><sub>1/2</sub>, and low intrinsic clearance. Hence, the novel potential BChE inhibitor <b>8012-9656</b> can be considered as a promising lead compound for further investigation of anti-AD agents.

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