Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease resulting in increasing disability, being uniformly fatal. Since its approval in the 1990s, riluzole remained for long time the unique treatment, offering modest survival benefit. Most recently a second drug has been approved by the US Food and Drug Administration for treatment of ALS: edaravone. Significant advances have been made in the symptomatic management of the disease but more effective drug therapy targeting disease progression is still dreadfully needed, the success appearing almost a miracle. Recent discoveries related to genetics indicate divergent mechanisms of disease encouraging precision medicine leading to molecularly tailored interventions. The search for effective therapy still faces important challenges in the areas of both basic science and animal research, adequate translation of results into human clinical trials, inherent bias in human studies, and issues related to delays in clinical diagnosis. It is interesting to point out that ALS research may speed up drug development not only for this disease, but also for other more prevalent neurodegenerative diseases: the reverse is also conceivable.

中文翻译：

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肌萎缩性侧索硬化症（ALS）的治疗：意想不到的发展情况。

肌萎缩性侧索硬化症（ALS）是一种进行性神经退行性疾病，会导致残疾增加，并致命。自1990年代批准以来，利鲁唑长期以来一直是独特的治疗方法，可提供适度的生存益处。最近，第二种药物已被美国食品和药物管理局批准用于治疗ALS：依达拉奉。在对症治疗方面已取得了重大进展，但仍急切需要针对疾病进展的更有效的药物治疗，这一成功几乎是奇迹。与遗传学有关的最新发现表明，疾病的不同机制促进了精密医学的发展，从而导致了分子定制的干预措施。在基础科学和动物研究，将结果充分转化为人类临床试验，人类研究中固有的偏见以及与临床诊断延误有关的领域中，寻求有效疗法仍然面临着重大挑战。有趣的是，ALS研究不仅可以加快这种疾病的药物开发速度，而且还可以加速其他更普遍的神经退行性疾病的药物开发：相反的情况也是可以想象的。