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阿尔茨海默病:临床试验与药物研发。

Alzheimer's disease: clinical trials and drug development.

机构信息

Aging Research Center, Karolinska Institutet, Stockholm, Sweden.

出版信息

Lancet Neurol. 2010 Jul;9(7):702-16. doi: 10.1016/S1474-4422(10)70119-8.

Abstract

Alzheimer's disease is the most common cause of dementia in elderly people. Research into Alzheimer's disease therapy has been at least partly successful in terms of developing symptomatic treatments, but has also had several failures in terms of developing disease-modifying therapies. These successes and failures have led to debate about the potential deficiencies in our understanding of the pathogenesis of Alzheimer's disease and potential pitfalls in diagnosis, choice of therapeutic targets, development of drug candidates, and design of clinical trials. Many clinical and experimental studies are ongoing, but we need to acknowledge that a single cure for Alzheimer's disease is unlikely to be found and that the approach to drug development for this disorder needs to be reconsidered. Preclinical research is constantly providing us with new information on pieces of the complex Alzheimer's disease puzzle, and an analysis of this information might reveal patterns of pharmacological interactions instead of single potential drug targets. Several promising randomised controlled trials are ongoing, and the increased collaboration between pharmaceutical companies, basic researchers, and clinical researchers has the potential to bring us closer to developing an optimum pharmaceutical approach for the treatment of Alzheimer's disease.

摘要

阿尔茨海默病是老年人中最常见的痴呆症病因。在开发针对症状的治疗方法方面,阿尔茨海默病治疗的研究至少在一定程度上取得了成功,但在开发能够改变疾病进程的治疗方法方面也遭遇了多次失败。这些成功和失败引发了关于我们对阿尔茨海默病发病机制的理解存在潜在缺陷以及在诊断、治疗靶点选择、候选药物开发和临床试验设计方面存在潜在陷阱的争论。目前有许多临床和实验研究正在进行,但我们需要承认,不太可能找到治疗阿尔茨海默病的单一方法,需要重新考虑针对这种疾病的药物开发方法。临床前研究不断为我们提供有关阿尔茨海默病这一复杂谜题的各个部分的新信息,对这些信息进行分析可能揭示出药物相互作用的模式,而不是单一的潜在药物靶点。目前正在进行几项有前途的随机对照试验,制药公司、基础研究人员和临床研究人员之间的合作增加,有可能使我们更接近开发治疗阿尔茨海默病的最佳药物治疗方法。

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