Institute of Pharmacy, University of Regensburg, Regensburg, Germany; Department of Medicinal Chemistry, Institute for Therapeutics Discovery and Development, University of Minnesota, Minneapolis, Minnesota; Department of Neurology, University of Minnesota, Minneapolis, Minnesota.
Department of Medicinal Chemistry, Institute for Therapeutics Discovery and Development, University of Minnesota, Minneapolis, Minnesota.
Transl Res. 2023 Apr;254:34-40. doi: 10.1016/j.trsl.2022.10.009. Epub 2022 Nov 4.
Targeting amyloid-β plaques and tau tangles has failed to provide effective treatments for Alzheimer's disease and related dementias (ADRD). A more fruitful pathway to ADRD therapeutics may be the development of therapies that target common signaling pathways that disrupt synaptic connections and impede communication between neurons. In this review, we present our characterization of a signaling pathway common to several neurological diseases featuring dementia including Alzheimer's disease, frontotemporal dementia, Lewy body dementia, and Huntington's disease. This signaling pathway features the cleavage of tau by caspase-2 (Casp2) yielding Δtau314 (Casp2/tau/Δtau314). Through a not yet fully delineated mechanism, Δtau314 catalyzes the mislocalization and accumulation of tau to dendritic spines leading to the internalization of AMPA receptors and the concomitant weakening of synaptic transmission. Here, we review the accumulated evidence supporting Casp2 as a druggable target and its importance in ADRD. Additionally, we provide a brief overview of our initial medicinal chemistry explorations aimed at the preparation of novel, brain penetrant Casp2 inhibitors. We anticipate that this review will spark broader interest in Casp2 as a target for restoring synaptic dysfunction in ADRD.
针对淀粉样蛋白-β斑块和 tau 缠结的治疗方法未能为阿尔茨海默病和相关痴呆症 (ADRD) 提供有效治疗。针对干扰突触连接和阻碍神经元之间通讯的常见信号通路开发治疗方法,可能是治疗 ADRD 的更有成效的途径。在这篇综述中,我们介绍了我们对几种以痴呆为特征的神经疾病(包括阿尔茨海默病、额颞叶痴呆、路易体痴呆和亨廷顿病)中常见信号通路的特征描述。该信号通路的特征是 caspase-2(Casp2)切割 tau 产生 Δtau314(Casp2/tau/Δtau314)。通过一个尚未完全阐明的机制,Δtau314 催化 tau 向树突棘的错误定位和积累,导致 AMPA 受体内化和随之而来的突触传递减弱。在这里,我们回顾了支持 Casp2 作为可药物靶标的累积证据及其在 ADRD 中的重要性。此外,我们还简要介绍了我们最初的药物化学探索,旨在制备新型、可穿透大脑的 Casp2 抑制剂。我们预计,这篇综述将引发人们对 Casp2 作为恢复 ADRD 中突触功能障碍的靶点的更广泛兴趣。