Department of Neurology, Medical University of Innsbruck, Innsbruck, Austria.
The Multiple System Atrophy Coalition, Inc., McLean, VA, USA.
J Parkinsons Dis. 2022;12(5):1369-1387. doi: 10.3233/JPD-223183.
Multiple system atrophy is a rapidly progressive and fatal neurodegenerative disorder. While numerous preclinical studies suggested efficacy of potentially disease modifying agents, none of those were proven to be effective in large-scale clinical trials. Three major strategies are currently pursued in preclinical and clinical studies attempting to slow down disease progression. These target α-synuclein, neuroinflammation, and restoration of neurotrophic support. This review provides a comprehensive overview on ongoing preclinical and clinical developments of disease modifying therapies. Furthermore, we will focus on potential shortcomings of previous studies that can be avoided to improve data quality in future studies of this rare disease.
多系统萎缩是一种快速进展且致命的神经退行性疾病。虽然许多临床前研究表明潜在的疾病修饰药物具有疗效，但没有一种药物在大规模临床试验中被证明是有效的。目前，在试图减缓疾病进展的临床前和临床研究中，主要有三种策略。这些靶点是α-突触核蛋白、神经炎症和神经营养支持的恢复。这篇综述全面概述了疾病修饰疗法的临床前和临床进展。此外，我们还将重点讨论以前研究中可能存在的缺陷，以便在未来对这种罕见疾病的研究中提高数据质量。
