针对帕金森病患者线粒体的治疗干预的临床疗效。

IF 2.7 4区 医学 Q3 NEUROSCIENCES CNS & neurological disorders drug targets Pub Date : 2024-01-01 DOI:10.2174/1871527322666230330122444
Matteo Ciocca, Chiara Pizzamiglio
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引用次数: 0

摘要

帕金森病是第二大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,针对线粒体的几种治疗方法已在帕金森病患者中进行了测试,以延缓疾病进展和解决疾病症状。在此,我们回顾了现有的随机双盲临床研究数据,这些研究调查了针对线粒体的化合物在特发性帕金森病患者中的作用,以期为患者和临床医生提供一份全面而实用的文件,为这类患者的治疗干预提供参考。在随机临床试验中,共有 9 种化合物接受了测试,但只有艾塞那肽显示出了一些有希望的神经保护和症状治疗效果。不过,这些证据能否转化为日常临床实践仍有待证实。总之,针对帕金森病的线粒体功能障碍是一种很有前景的治疗方法,尽管只有一种化合物显示出对帕金森病进展和症状的积极影响。新化合物已在动物模型中进行了研究,其疗效需要通过可靠的随机双盲临床试验在人体中得到证实。
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Clinical Benefits of Therapeutic Interventions Targeting Mitochondria in Parkinson's Disease Patients.

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.

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来源期刊
CiteScore
5.10
自引率
3.30%
发文量
158
审稿时长
6-12 weeks
期刊介绍: Aims & Scope CNS & Neurological Disorders - Drug Targets aims to cover all the latest and outstanding developments on the medicinal chemistry, pharmacology, molecular biology, genomics and biochemistry of contemporary molecular targets involved in neurological and central nervous system (CNS) disorders e.g. disease specific proteins, receptors, enzymes, genes. CNS & Neurological Disorders - Drug Targets publishes guest edited thematic issues written by leaders in the field covering a range of current topics of CNS & neurological drug targets. The journal also accepts for publication original research articles, letters, reviews and drug clinical trial studies. As the discovery, identification, characterization and validation of novel human drug targets for neurological and CNS drug discovery continues to grow; this journal is essential reading for all pharmaceutical scientists involved in drug discovery and development.
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