关键词: Clinical trials Microtubule dysfunction Modifiable risk factor Monoclonal antibody Progressive supranuclear palsy Tau Tau phosphorylation

来  源:   DOI:10.1007/s40120-024-00614-9   PDF(Pubmed)

Abstract:
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.
摘要:
进行性核上性麻痹(PSP)是一种神经退行性疾病,是由于中脑特定区域中tau蛋白的错误折叠和神经毒性形式沉积而引起的。基底神经节,和皮质。它是Tau蛋白病变的最具代表性的形式之一。PSP表现出几种不同的表型变异,并且通常伴随着并发神经退行性疾病的发展。PSP普遍是致命的,和PSP的有效疾病改善疗法尚未确定。几种tau靶向治疗方式,包括疫苗,单克隆抗体,和微管稳定剂,已经被调查过,没有疗效。治疗PSP和其他tau蛋白病的必要性至关重要,许多研究tau靶向治疗的临床试验正在进行中。在这次审查中,我们查询PubMed数据库,以收集有关PSP治疗的临床前和临床研究的信息.此外,查询了美国国家医学图书馆的ClinicalTrials.gov网站,以确定与PSP治疗相关的过去和正在进行的临床试验。这篇叙述性综述总结了我们关于这些报告的发现,其中包括潜在的改善疾病的药物试验,可修改的风险因素管理,和症状治疗。
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