殷明. 阿尔茨海默病治疗药物研发全面受挫及其思考J. 药学学报, 2014,49(6): 757-763.
引用本文: 殷明. 阿尔茨海默病治疗药物研发全面受挫及其思考J. 药学学报, 2014,49(6): 757-763.
YIN Ming. Full-scale setback and thinking in R&D of Alzheimer’s disease drugJ. Acta Pharmaceutica Sinica, 2014,49(6): 757-763.
Citation: YIN Ming. Full-scale setback and thinking in R&D of Alzheimer’s disease drugJ. Acta Pharmaceutica Sinica, 2014,49(6): 757-763.

阿尔茨海默病治疗药物研发全面受挫及其思考

Full-scale setback and thinking in R&D of Alzheimer’s disease drug

  • 摘要: 以往30年中针对众多靶点的阿尔茨海默病(AD)治疗药物的研发,虽然全球药业公司、政府、研究机构、大学和风险投资机构等都投入了大量的人力物力资源,但2003年以来FDA没有批准一个AD药物上市。究其原因,固然与无法早期诊断、治疗策略欠佳及患者的遗传多态性带来药物反应的多样性等因素有关,更主要的原因可能是对包括AD在内的许多复杂性疾病发病机制的了解还很肤浅。继续按照目前思路针对单个靶点的AD药物研发可能不是明智的做法。系统生物医学等发展为认知疾病网络机制及基于系统药理学的多靶点药物研发带来了希望;建立在深入的基础研究水平上的神经干细胞移植或小分子药物影响神经干细胞再生可能成为AD治疗的新途径;生物标志物的发现为研究AD发病机制及新型药物研发将带来启迪。

     

    Abstract: During the past 30 years, Alzheimer's disease (AD) drug R&D aiming at a variety of potential targets has undergone tremendous setback although a large amount of resources have been invested bypharmaceutical companies, governments, academic institutions and venture capitals globally.There is no doubt that uneasy early diagnosis, unsatisfying therapeutic strategies and genetic polymorphism of AD patientsbringing variety of responses to the drugs contribute to the failure of AD drug therapy, but the most important reason might be that people have insufficient understanding of the very complex diseases including AD.Continuing to make efforts in the previous way to find AD drugs might not be a good way.Development of systems biomedicine opens an avenue to understand the disease networks and pursue multi-target drugs R&D based on systems pharmacology theory; neural stem cell transplantation, and neurogenesis affected by small molecules might bring new hope for AD therapy; discovery of new biomarkers of AD will help the study of the pathogenesis and diagnosis of the disease and finally the discovery of new types of AD drugs.

     

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