Neurological Clinical Research Institute, Massachusetts General Hospital, 165 Cambridge Street, Suite 600, Boston, Massachusetts, 02114, USA.
Spaulding Rehabilitation Hospital, Boston, Massachusetts, USA.
Muscle Nerve. 2019 Mar;59(3):303-308. doi: 10.1002/mus.26385. Epub 2018 Dec 26.
RNS60 is a novel immune-modulatory agent that has shown neuroprotective effects in amytrophic lateral sclerosis (ALS) preclinical models. RNS60 is administered by weekly intravenous infusion and daily nebulization. The objective of this pilot open-label trial was to test the feasibility, safety, and tolerability of long-term RNS60 administration in ALS patients.
The planned treatment duration was 23 weeks and the primary outcomes were safety and tolerability. Secondary outcomes included PBR28 positron emission tomography (PET) imaging and plasma biomarkers of inflammation.
Sixteen participants with ALS received RNS60 and 13 (81%) completed 23 weeks of RNS60 treatment. There were no serious adverse events and no participants withdrew from the trial due to drug-related adverse events. There were no significant changes in the biomarkers.
Long-term RNS60 administration was safe and well-tolerated. A large, multicenter, phase II trial of RNS60 is currently enrolling participants to test the effects of RNS60 on ALS biomarkers and disease progression. Muscle Nerve 59:303-308, 2019.
RNS60 是一种新型免疫调节药物，在肌萎缩侧索硬化症（ALS）的临床前模型中显示出神经保护作用。RNS60 通过每周静脉输注和每日雾化给药。本试验的目的是测试 RNS60 在 ALS 患者中的长期给药的可行性、安全性和耐受性。
计划的治疗时间为 23 周，主要终点是安全性和耐受性。次要终点包括 PBR28 正电子发射断层扫描（PET）成像和炎症的血浆生物标志物。
16 名 ALS 患者接受了 RNS60 治疗，其中 13 名（81%）完成了 23 周的 RNS60 治疗。没有严重的不良事件，也没有因药物相关不良事件而退出试验的患者。生物标志物没有明显变化。
长期 RNS60 给药是安全且耐受良好的。一项针对 RNS60 的大型、多中心、二期临床试验目前正在招募参与者，以测试 RNS60 对 ALS 生物标志物和疾病进展的影响。肌肉神经 59:303-308, 2019。
