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治疗靶点为帕金森病患者线粒体的干预措施的临床获益。

Clinical Benefits of Therapeutic Interventions Targeting Mitochondria in Parkinson's Disease Patients.

机构信息

Department of Brain Sciences, Imperial College London, London, UK.

Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.

出版信息

CNS Neurol Disord Drug Targets. 2024;23(5):554-561. doi: 10.2174/1871527322666230330122444.

Abstract

Parkinson's disease is the second most common neurodegenerative disease. Mitochondrial dysfunction has been associated with neurodegeneration in Parkinson's disease, and several treatments targeting mitochondria have been tested in these patients to delay disease progression and tackle disease symptoms. Herein, we review available data from randomised, double-blind clinical studies that have investigated the role of compounds targeting mitochondria in idiopathic Parkinson's disease patients, with a view of providing patients and clinicians with a comprehensive and practical paper that can inform therapeutic interventions in this group of people. A total of 9 compounds have been tested in randomized clinical trials, but only exenatide has shown some promising neuroprotective and symptomatic effects. However, whether this evidence can be translated into daily clinical practice still needs to be confirmed. In conclusion, targeting mitochondrial dysfunction in Parkinson's disease is a promising therapeutic approach, although only one compound has shown a positive effect on Parkinson's disease progression and symptoms. New compounds have been investigated in animal models, and their efficacy needs to be confirmed in humans through robust, randomised, double-blind clinical trials.

摘要

帕金森病是第二常见的神经退行性疾病。线粒体功能障碍与帕金森病的神经退行性变有关,已经在这些患者中测试了几种针对线粒体的治疗方法,以延缓疾病进展和解决疾病症状。本文综述了随机、双盲临床试验中有关针对特发性帕金森病患者的靶向线粒体化合物的现有数据,旨在为患者和临床医生提供一份全面实用的论文,为这组人群的治疗干预提供信息。已经在随机临床试验中测试了 9 种化合物,但只有 exenatide 显示出一些有希望的神经保护和症状缓解作用。然而,这些证据是否可以转化为日常临床实践仍需要确认。总之,靶向帕金森病中的线粒体功能障碍是一种很有前途的治疗方法,尽管只有一种化合物对帕金森病的进展和症状有积极影响。已经在动物模型中研究了新的化合物,需要通过严格的、随机的、双盲临床试验在人体中证实其疗效。

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