UCIBIO - Applied Molecular Biosciences Unit, Laboratory of Toxicology, Department of Biological Sciences, Faculty of Pharmacy, Porto University, 4050-313 Porto, Portugal; Associate Laboratory i4HB - Institute for Health and Bioeconomy, Faculty of Pharmacy, University of Porto, 4050-313 Porto, Portugal.
TOXRUN - Toxicology Research Unit, Department of Sciences, University Institute of Health Sciences, CESPU, CRL, Gandra, Portugal; Instituto de Investigação e Inovação em Saúde (i3S), Universidade do Porto, Porto, Portugal.
Biochem Pharmacol. 2023 May;211:115522. doi: 10.1016/j.bcp.2023.115522. Epub 2023 Mar 28.
Alzheimer's disease (AD) is one of the most prevalent neurodegenerative diseases that affect millions of people worldwide, with both prevalence and incidence increasing with age. It is characterized by cognitive decline associated, specifically, with degeneration of cholinergic neurons. The problem of this disease is even more fundamental as the available therapies remain fairly limited and mainly focused on symptoms' relief. Although the aetiology of the disease remains elusive, two main pathological hallmarks are described: i) presence of neurofibrillary tangles formed by unfolded protein aggregates (hyperphosphorylated Tau protein) and ii) presence of extracellular aggregates of amyloid-beta peptide. Given the complexity surrounding the pathogenesis of the disease, several potential targets have been highlighted and interrelated upon its progression, such as oxidative stress and the accumulation of metal ions. Thus, advances have been made on the development of innovative multitarget therapeutical compounds to delay the disease progression and restore cell function. This review focuses the ongoing research on new insights and emerging disease-modifying drugs for AD treatment. Furthermore, classical and novel potential biomarkers for early diagnosis of the disease, and their role in assisting on the improvement of targeted therapies will also be approached.
阿尔茨海默病(AD)是最常见的神经退行性疾病之一,影响着全球数以百万计的人,其患病率和发病率随着年龄的增长而增加。它的特征是认知能力下降,特别是与胆碱能神经元退化有关。由于现有的治疗方法仍然相当有限,主要集中在症状缓解上,因此该疾病的问题更加严重。尽管该疾病的病因仍然难以捉摸,但已经描述了两个主要的病理特征:i)由未折叠蛋白聚集体(过度磷酸化的 Tau 蛋白)形成的神经原纤维缠结和 ii)淀粉样-β肽的细胞外聚集物的存在。鉴于疾病发病机制的复杂性,已经突出了几个潜在的靶点,并在其进展过程中相互关联,如氧化应激和金属离子的积累。因此,在开发创新的多靶点治疗化合物以延缓疾病进展和恢复细胞功能方面取得了进展。本综述集中研究了用于 AD 治疗的新的、有前景的疾病修饰药物的最新进展。此外,还将探讨用于早期诊断该疾病的经典和新型潜在生物标志物及其在辅助靶向治疗中的作用。