摘 要
神经退行性疾病如阿尔茨海默病、帕金森病等严重影响患者生活质量，给社会带来沉重负担，开发有效治疗药物成为医学领域的重要课题。本研究旨在综述神经退行性疾病治疗药物的开发进展，通过文献检索与分析，聚焦近年来新靶点发现、新型药物研发策略及临床试验成果。在方法上，基于国内外权威数据库，筛选近十年相关高质量文献进行系统评价。结果表明，针对β - 淀粉样蛋白、tau蛋白异常磷酸化等病理机制的新药研发取得突破性进展，单克隆抗体类药物可特异性结合靶标，减少神经细胞损伤；基因治疗为从根源上纠正致病基因表达提供可能；小分子化合物凭借良好穿透血脑屏障能力展现独特优势。同时，多靶点协同作用理念逐渐兴起，将不同机制药物联合应用以期达到更佳疗效。本研究结论是神经退行性疾病治疗药物开发虽面临诸多挑战，但随着对疾病发病机制深入理解以及创新技术不断涌现，已取得一系列重要成果，为未来研发方向提供了有益借鉴，特别是多学科交叉融合加速了药物开发进程，为改善患者预后带来了新的希望。
关键词：神经退行性疾病；阿尔茨海默病；帕金森病
Abstract
Neurodegenerative diseases such as Alzheimer's disease and Parkinson's disease significantly impair patients' quality of life and impose a heavy burden on society, making the development of effective therapeutic agents a critical challenge in the medical field. This study aims to review the progress in drug development for neurodegenerative diseases by analyzing recent discoveries of new targets, novel drug development strategies, and clinical trial outcomes through literature retrieval and analysis. Based on authoritative domestic and international databases, high-quality relevant literature from the past decade was systematically evaluated. The findings indicate that breakthroughs have been achieved in drug development targeting pathological mechanisms such as β-amyloid and tau protein hyperphosphorylation. Monoclonal antibody drugs can specifically bind to targets, reducing neuronal damage; gene therapy offers the potential to correct disease-causing gene ex pression at its root; small molecules demonstrate unique advantages with their excellent ability to penetrate the blood-brain barrier. Moreover, the concept of multi-target synergistic effects is emerging, combining drugs with different mechanisms to achieve better therapeutic outcomes. In conclusion, while the development of therapeutic agents for neurodegenerative diseases faces numerous challenges, significant achievements have been made with a deeper understanding of disease pathogenesis and the emergence of innovative technologies, providing valuable insights for future research directions. Particularly, the integration of interdisciplinary approaches has accelerated the drug development process, bringing new hope for improving patient prognosis.
Keywords：Neurodegenerative Diseases；Alzheimer's Disease；Parkinson's Disease
目 录
摘 要 I
Abstract II
引 言 1
第一章 疾病机制与药物靶点探索 2
1.1 神经退行性疾病的病理特征 2
1.2 关键致病蛋白的发现历程 2
1.3 新兴药物靶点的研究进展 3
第二章 小分子药物研发策略 4
2.1 化学结构优化与设计 4
2.2 血脑屏障穿透技术突破 4
2.3 临床前药效评价体系 5
第三章 生物制剂开发与应用 7
3.1 单克隆抗体治疗原理 7
3.2 基因疗法的技术路径 7
3.3 细胞治疗的最新进展 8
第四章 药物临床转化研究 9
4.1 临床试验设计与实施 9
4.2 不同阶段的疗效评估 9
4.3 安全性监测与风险管理 10
结 论 12
参考文献 13
致 谢 14
