标示外治疗肌萎缩侧索硬化症的科学和治疗原理。

IF 8.1 1区医学 Q1 CLINICAL NEUROLOGY Annals of Neurology Pub Date : 2024-11-06 DOI:10.1002/ana.27126

Richard Bedlack MD, PhD, Xiaoyan Li MD, PhD, Baggio Angelo Evangelista PhD, Maria E. Panzetta PhD, Justin Kwan MD, Lauren M. Gittings PhD, Rita Sattler PhD

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引用次数: 0

摘要

肌萎缩性脊髓侧索硬化症是一种具有多种潜在机制的复杂疾病，如神经炎症、氧化应激、线粒体功能障碍、微生物组改变和抗逆转录病毒活性。我们筛选了一个名为 ALSUntangled 的小组 15 年来的综述，找出了 8 种针对其中≥1 种机制、且有≥1 项人体实验表明有显著疗效的替代疗法和标签外疗法。鉴于这些重点产品的病理机制相互重叠，我们认为这些针对不同机制的治疗方法的组合在未来肌萎缩性脊髓侧索硬化症的治疗开发中可能会很有价值。ann neurol 2024.

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The Scientific and Therapeutic Rationale for Off-Label Treatments in Amyotrophic Lateral Sclerosis

There are no dramatically effective pharmacological treatments for most patients with amyotrophic lateral sclerosis, a complex disease with multiple underlying mechanisms, such as neuroinflammation, oxidative stress, mitochondrial dysfunction, microbiome alteration, and antiretroviral activity. We sifted through 15 years of reviews by a group called ALSUntangled to identify 8 alternative and off-label treatments that target ≥1 of these mechanisms, and have ≥1 human trial suggesting meaningful benefits. Given the overlapping pathological mechanisms of the highlighted products, we suggest that combinations of these treatments targeting diverse mechanisms might be worthwhile for future amyotrophic lateral sclerosis therapy development. ANN NEUROL 2025;97:15–27

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来源期刊

Annals of Neurology 医学-临床神经学

CiteScore

18.00

自引率

1.80%

发文量

270

审稿时长

3-8 weeks

期刊介绍： Annals of Neurology publishes original articles with potential for high impact in understanding the pathogenesis, clinical and laboratory features, diagnosis, treatment, outcomes and science underlying diseases of the human nervous system. Articles should ideally be of broad interest to the academic neurological community rather than solely to subspecialists in a particular field. Studies involving experimental model system, including those in cell and organ cultures and animals, of direct translational relevance to the understanding of neurological disease are also encouraged.