Almeida Zaida L, Vaz Daniela C, Brito Rui M M
Chemistry Department and Coimbra Chemistry Centre - Institute of Molecular Sciences (CQC-IMS), University of Coimbra, 3004-535, Coimbra, Portugal.
School of Health Sciences, Polytechnic Institute of Leiria, 2411-901, Leiria, Portugal.
Mol Neurobiol. 2025 Apr;62(4):4391-4419. doi: 10.1007/s12035-024-04543-4. Epub 2024 Oct 24.
Alzheimer's disease (AD) is the most common form of dementia around the world (~ 65%). Here, we portray the neuropathology of AD, biomarkers, and classification of amyloid plaques (diffuse, non-cored, dense core, compact). Tau pathology and its involvement with Aβ plaques and cell death are discussed. Amyloid cascade hypotheses, aggregation mechanisms, and molecular species formed in vitro and in vivo (on- and off-pathways) are described. Aβ42/Aβ40 monomers, dimers, trimers, Aβ-derived diffusible ligands, globulomers, dodecamers, amylospheroids, amorphous aggregates, protofibrils, fibrils, and plaques are characterized (structure, size, morphology, solubility, toxicity, mechanistic steps). An update on AD-approved drugs by regulatory agencies, along with new Aβ-based therapies, is presented. Beyond prescribing Aβ plaque disruptors, cholinergic agonists, or NMDA receptor antagonists, other therapeutic strategies (RNAi, glutaminyl cyclase inhibitors, monoclonal antibodies, secretase modulators, Aβ aggregation inhibitors, and anti-amyloid vaccines) are already under clinical trials. New drug discovery approaches based on "designed multiple ligands", "hybrid molecules", or "multitarget-directed ligands" are also being put forward and may contribute to tackling this highly debilitating and fatal form of human dementia.
阿尔茨海默病(AD)是全球最常见的痴呆形式(约占65%)。在此,我们阐述了AD的神经病理学、生物标志物以及淀粉样斑块的分类(弥漫性、无核心、致密核心、紧密型)。讨论了tau病理学及其与Aβ斑块和细胞死亡的关系。描述了淀粉样蛋白级联假说、聚集机制以及在体外和体内形成的分子种类(在途径上和非途径上)。对Aβ42/Aβ40单体、二聚体、三聚体、Aβ衍生的可扩散配体、球状体、十二聚体、淀粉样球体、无定形聚集体、原纤维、纤维和斑块进行了表征(结构、大小、形态、溶解性、毒性、作用机制步骤)。介绍了监管机构批准的AD药物以及新的基于Aβ的疗法。除了开处方Aβ斑块破坏剂、胆碱能激动剂或NMDA受体拮抗剂外,其他治疗策略(RNA干扰、谷氨酰胺环化酶抑制剂、单克隆抗体、分泌酶调节剂、Aβ聚集抑制剂和抗淀粉样疫苗)已在临床试验中。基于“设计的多配体”“杂合分子”或“多靶点导向配体”的新药发现方法也在被提出,可能有助于应对这种极具衰弱性和致命性的人类痴呆形式。