Dunning Elise E, Decourt Boris, Zawia Nasser H, Shill Holly A, Sabbagh Marwan N
Creighton University School of Medicine - Phoenix, Phoenix, AZ, USA.
Department of Pharmacology and Neuroscience School of Medicine, Texas Tech University Health Sciences Center, Lubbock, TX, USA.
Neurol Ther. 2024 Aug;13(4):975-1013. doi: 10.1007/s40120-024-00614-9. Epub 2024 May 14.
Progressive supranuclear palsy (PSP) is a neurodegenerative disorder resulting from the deposition of misfolded and neurotoxic forms of tau protein in specific areas of the midbrain, basal ganglia, and cortex. It is one of the most representative forms of tauopathy. PSP presents in several different phenotypic variations and is often accompanied by the development of concurrent neurodegenerative disorders. PSP is universally fatal, and effective disease-modifying therapies for PSP have not yet been identified. Several tau-targeting treatment modalities, including vaccines, monoclonal antibodies, and microtubule-stabilizing agents, have been investigated and have had no efficacy. The need to treat PSP and other tauopathies is critical, and many clinical trials investigating tau-targeted treatments are underway. In this review, the PubMed database was queried to collect information about preclinical and clinical research on PSP treatment. Additionally, the US National Library of Medicine's ClinicalTrials.gov website was queried to identify past and ongoing clinical trials relevant to PSP treatment. This narrative review summarizes our findings regarding these reports, which include potential disease-modifying drug trials, modifiable risk factor management, and symptom treatments.
进行性核上性麻痹(PSP)是一种神经退行性疾病,由错误折叠且具有神经毒性的tau蛋白在中脑、基底神经节和皮质的特定区域沉积所致。它是tau蛋白病最具代表性的形式之一。PSP呈现出几种不同的表型变异,且常伴有并发神经退行性疾病的发展。PSP是普遍致命的,尚未确定有效的疾病修饰疗法。包括疫苗、单克隆抗体和微管稳定剂在内的几种针对tau蛋白的治疗方式已被研究,但均无疗效。治疗PSP和其他tau蛋白病的需求至关重要,许多针对tau蛋白靶向治疗的临床试验正在进行。在本综述中,查询了PubMed数据库以收集有关PSP治疗的临床前和临床研究信息。此外,查询了美国国立医学图书馆的ClinicalTrials.gov网站,以识别与PSP治疗相关的既往和正在进行的临床试验。本叙述性综述总结了我们关于这些报告的研究结果,其中包括潜在的疾病修饰药物试验、可改变危险因素的管理和症状治疗。