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肌萎缩侧索硬化症治疗的现状与未来方向。

Current State and Future Directions in the Therapy of ALS.

机构信息

Department of Neurology, Rechts der Isar Hospital, Technical University of Munich, 81675 München, Germany.

Department of Neurology, University of Regensburg, 93053 Regensburg, Germany.

出版信息

Cells. 2023 May 31;12(11):1523. doi: 10.3390/cells12111523.

Abstract

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potentially diverse, finding a suitable therapy to slow down or possibly stop disease progression remains challenging. Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. Even though curative treatment options, able to prevent or stop disease progression, are still unknown, recent breakthroughs, especially in the field of targeting genetic disease forms, raise hope for improved care and therapy for ALS patients. In this review, we aim to summarize the current state of ALS therapy, including medication as well as supportive therapy, and discuss the ongoing developments and prospects in the field. Furthermore, we highlight the rationale behind the intense research on biomarkers and genetic testing as a feasible way to improve the classification of ALS patients towards personalized medicine.

摘要

肌萎缩侧索硬化症(ALS)是一种快速进展的神经退行性疾病,影响上下运动神经元,发病后 3 至 5 年内主要因呼吸衰竭而死亡。由于确切的潜在病因病理途径尚不清楚且可能多种多样,因此找到一种合适的治疗方法来减缓或可能阻止疾病进展仍然具有挑战性。根据国家的不同,利鲁唑、依达拉奉和苯丁酸钠/牛磺酸二醇是目前唯一被批准用于治疗 ALS 的药物,因为它们对疾病进展有一定的疗效。尽管尚不清楚是否存在能够预防或阻止疾病进展的治愈性治疗方法,但最近的突破,特别是在靶向遗传疾病形式的领域,为 ALS 患者的治疗和护理带来了新的希望。在这篇综述中,我们旨在总结 ALS 治疗的现状,包括药物治疗和支持性治疗,并讨论该领域正在进行的发展和前景。此外,我们强调了对生物标志物和基因测试进行深入研究的基本原理,因为这是一种改善 ALS 患者分类、迈向个体化医疗的可行方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/43fe/10252394/d1c13676eb10/cells-12-01523-g001.jpg

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