Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by the loss of upper and lower motor neurons. Presently, three FDA-approved drugs are available to help slow functional decline for patients with ALS, but no cure yet exists. With an average life expectancy of only two to five years after diagnosis, there is a clear need for biomarkers to improve the care of patients with ALS and to expedite ALS treatment development. Here, we provide a review of the efforts made towards identifying diagnostic, prognostic, susceptibility/risk, and response fluid biomarkers with the intent to facilitate a more rapid and accurate ALS diagnosis, to better predict prognosis, to improve clinical trial design, and to inform interpretation of clinical trial results. Over the course of 20 + years, several promising fluid biomarker candidates for ALS have emerged. These will be discussed, as will the exciting new strategies being explored for ALS biomarker discovery and development.

中文翻译：

---

肌萎缩侧索硬化症的液体生物标志物：综述

肌萎缩侧索硬化症（ALS）是一种致命的神经退行性疾病，其特征是上、下运动神经元丧失。目前，FDA 批准的三种药物可帮助减缓 ALS 患者的功能衰退，但尚无治愈方法。由于诊断后的平均预期寿命仅为两到五年，因此显然需要生物标志物来改善 ALS 患者的护理并加快 ALS 治疗的开发。在这里，我们回顾了在识别诊断、预后、易感性/风险和反应液体生物标志物方面所做的努力，旨在促进更快速、更准确的 ALS 诊断，更好地预测预后，改进临床试验设计，并告知临床试验结果的解释。在 20 多年的时间里，一些有前途的 ALS 液体生物标志物候选物已经出现。我们将讨论这些问题，以及正在探索的 ALS 生物标志物发现和开发的令人兴奋的新策略。