a Parkinson's disease and Movement Disorders Department , Hygeia Hospital , Athens , Greece.
b Neurology Clinic , Philipps-University , Marburg , Germany.
Expert Opin Emerg Drugs. 2019 Jun;24(2):83-92. doi: 10.1080/14728214.2019.1609450. Epub 2019 May 20.
: Progressive supranuclear palsy (PSP) is a common cause of atypical parkinsonism and a rapidly progressive disease that greatly burdens both patients and caregivers. Drugs with disease-modifying potential, targeting mechanisms implicated in the disease's pathogenesis are currently tested in Phase 1 and 2 trials. If proven efficacious, these compounds might provide substantial benefits not only to patients with PSP but to patients with other tauopathies as well. : Drugs in Phase 1 and 2 trials in PSP, and Phase 2 trials in other tauopathies (Alzheimer's disease) are reviewed. : The rationale behind the currently tested compounds as well as the tools available to document a treatment effect offer hope for a therapeutic breakthrough in PSP. The current lack of sufficiently validated biomarkers remains a hurdle that needs to be overcome, in order to facilitate both clinical trials and the accurate prescription of future treatments.
进行性核上性麻痹（PSP）是一种常见的非典型帕金森病病因，也是一种快速进展性疾病，给患者和照护者都带来了沉重负担。目前，针对疾病发病机制中涉及的机制，具有疾病修饰潜力的药物正在进行 1 期和 2 期临床试验。如果这些化合物被证明有效，不仅对 PSP 患者，而且对其他 tau 病患者（如阿尔茨海默病）也可能带来实质性益处。
对 PSP 的 1 期和 2 期临床试验中的药物以及其他 tau 病（阿尔茨海默病）的 2 期临床试验进行综述。
目前正在测试的化合物背后的原理以及记录治疗效果的可用工具为 PSP 治疗方面的突破带来了希望。目前，缺乏足够验证的生物标志物仍然是一个需要克服的障碍，这既有利于临床试验，也有利于未来治疗的准确处方。
