a Department of Nursing , Catholic University San Vicente Mártir , Valencia , Spain.
b Department of Health and Functional Valorization , Catholic University San Vicente Martir , Valencia , Spain.
Amyotroph Lateral Scler Frontotemporal Degener. 2019 Feb;20(1-2):115-122. doi: 10.1080/21678421.2018.1536152. Epub 2019 Jan 22.
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease, characterized by progressive loss of spinal and cortical motor neurons, leading to muscular atrophy, respiratory failure, and ultimately death. There is no known cure, and the clinical benefit of the two drugs approved to treat ALS remains unclear. Novel disease-modifying therapeutics that are able to modulate the disease course are desperately needed. Our objective was to evaluate the efficacy and tolerability of Elysium Health's candidate drug EH301 in people with ALS (PALS).
This was a single-center, prospective, double-blind, randomized, placebo-controlled pilot study. Thirty-two PALS were recruited thanks to the collaboration of the Spanish Foundation for ALS Research (FUNDELA). Study participants were randomized to receive either EH301 or placebo and underwent evaluation for 4 months. Differences between EH301 and placebo-treated participants were evaluated based on standard clinical endpoints, including the revised ALS functional rating scale (ALSFRS-R), forced vital capacity (FVC), and the Medical Research Council (MRC) grading scale.
Compared to placebo, participants treated with EH301 demonstrated significant improvements in the ALSFRS-R score, pulmonary function, muscular strength, and in skeletal muscle/fat weight ratio. EH301 was shown to significantly slow the progression of ALS relative to placebo, and even showed improvements in several key outcome measures compared with baseline.
This study provides evidence in support of the disease-modifying effects of EH301 for the treatment of ALS.
肌萎缩侧索硬化症（ALS）是一种破坏性的神经退行性疾病，其特征是脊髓和皮质运动神经元进行性丧失，导致肌肉萎缩、呼吸衰竭，最终导致死亡。目前尚无已知的治愈方法，两种已批准用于治疗 ALS 的药物的临床获益仍不清楚。迫切需要能够调节疾病进程的新型疾病修饰治疗药物。我们的目的是评估 Elysium Health 的候选药物 EH301 在肌萎缩侧索硬化症患者（PALS）中的疗效和耐受性。
这是一项单中心、前瞻性、双盲、随机、安慰剂对照的初步研究。由于西班牙肌萎缩侧索硬化症研究基金会（FUNDELA）的合作，招募了 32 名 PALS。研究参与者随机接受 EH301 或安慰剂治疗，并接受了 4 个月的评估。根据标准临床终点评估 EH301 与安慰剂治疗参与者之间的差异，包括修订后的肌萎缩侧索硬化症功能评定量表（ALSFRS-R）、用力肺活量（FVC）和医学研究委员会（MRC）分级量表。
与安慰剂相比，接受 EH301 治疗的参与者在 ALSFRS-R 评分、肺功能、肌肉力量以及骨骼肌/脂肪重量比方面均有显著改善。EH301 显示可显著减缓 ALS 的进展，与安慰剂相比，甚至在几个关键终点测量上也有所改善。
这项研究为 EH301 治疗 ALS 的疾病修饰作用提供了证据支持。
