From Bench to Bedside: Cutting-Edge and Emerging Therapies for Alzheimer's Disease.

Chanchal Sharma, Avijit Mazumder
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Abstract

Alzheimer's disease (AD) remains one of the most pressing neurodegenerative disorders worldwide, with increasing prevalence and limited disease-modifying treatments. While recent clinical advances, including monoclonal antibodies like lecanemab and donanemab (early stage), show promise in slowing cognitive decline by targeting amyloid-beta pathology, their use is associated with risks such as amyloid-related imaging abnormalities (ARIA). Alongside these developments, preclinical innovations continue to explore novel mechanisms, including antisense oligonucleotides, TREM2 agonists, siRNA, mRNA-LNP platforms, and CRISPR-based gene editing. These approaches target tau aggregation, neuroinflammation, and genetic risk modifiers like APOE4. This review bridges the gap between preclinical research and clinical application by highlighting the mechanisms, therapeutic potential, and translational challenges of both established and emerging therapies. Emphasis is placed on biomarker-guided trials, model systems (e.g., iPSC organoids), and future directions to improve efficacy, safety, and global accessibility of AD therapeutics.

从实验室到床边:阿尔茨海默病的前沿和新兴疗法。
阿尔茨海默病(AD)仍然是世界范围内最紧迫的神经退行性疾病之一,患病率不断上升,疾病改善治疗有限。虽然最近的临床进展,包括单克隆抗体如lecanemab和donanemab(早期),显示出通过靶向淀粉样蛋白病理减缓认知衰退的希望,但它们的使用与淀粉样蛋白相关成像异常(ARIA)等风险相关。随着这些发展,临床前创新继续探索新的机制,包括反义寡核苷酸、TREM2激动剂、siRNA、mRNA-LNP平台和基于crispr的基因编辑。这些方法的目标是tau聚集,神经炎症和遗传风险修饰因子,如APOE4。这篇综述通过强调机制、治疗潜力和现有和新兴疗法的转化挑战,弥合了临床前研究和临床应用之间的差距。重点放在生物标志物引导的试验,模型系统(例如,iPSC类器官),以及未来的方向,以提高阿尔茨海默病治疗的有效性,安全性和全球可及性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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