肌萎缩侧索硬化细胞治疗研发现状和研发策略及评价标准关键要素的考量
Current status,research strategies,and evaluation criteria of cell therapy for amyotrophic lateral sclerosis:considerations of key elements
李嘉桐 1孙搏 2曹钰然 3王亚宁 4马璟 5张菁 3陈向军1
作者信息
- 1. 复旦大学附属华山医院神经内科/复旦大学神经病学研究所,上海 200040;国家神经疾病医学中心,上海 200040
- 2. 上海药品审评核查中心,上海 201210
- 3. 复旦大学附属华山医院临床药理研究中心,上海 200040
- 4. 朗来科技发展有限公司,上海 201700
- 5. 上海泰楚生物技术有限公司,上海 201413
- 折叠
摘要
肌萎缩侧索硬化(ALS)是一种以上、下运动神经元退行性变为特征的神经系统罕见病,临床表现为进行性发展的肌肉无力、萎缩,最终导致死亡,给患者家庭及社会带来沉重的负担.因ALS发病机制不明,目前现有ALS治疗药物均无法显著延长生存期或改变生存结局.近年来,多种不同种类干细胞的细胞治疗已被开发用于ALS的治疗,并处于临床前研究和临床试验阶段.本文通过概述细胞治疗在ALS中的应用情况,并详细介绍代表性细胞治疗NurOwn在ALS中的研究和发展历程,分析了目前ALS细胞治疗取得的成果和存在的问题,建议在未来ALS细胞治疗的相关研究中进一步优化细胞类型选择和疗效评价指标,以指导ALS细胞治疗研发和获批上市,惠及患者及其家庭.
Abstract
Amyotrophic lateral sclerosis(ALS)is a rare neurodegenerative disease characterized by the degeneration of upper and lower motor neurons.Clinically,it manifests as progressive muscle weakness and atrophy,ultimately leading to death,imposing a significant burden on patients'families and society.The pathogenesis of ALS remains unclear.Currently,several ALS treatment drugs have failed to significantly extend survival or alter the outcome.In recent years,cell therapy has been increasingly applied to refractory or incurable diseases.Various types of stem cell therapies have been developed for ALS treatment,undergoing preclinical research and clinical trials.This article provides an overview of the application of cell therapy in ALS,with a detailed examination of the research and development history of the representative cell therapy NurOwn in ALS.And the achievements and challenges of current ALS cell therapy,suggesting the optimization of cell type selection and efficacy evaluation metrics in future ALS cell therapy research were further discussed.This optimization aims to guide the development,approval,and market entry of ALS cell therapies,benefiting patients and their families.
关键词
神经变性疾病/肌萎缩侧索硬化/细胞疗法Key words
neurodegenerative diseases/amyotrophic lateral sclerosis/cell therapy引用本文复制引用
基金项目
国家自然科学基金面上项目(82371431)
医疗服务与保障能力提升(医疗卫生机构能力建设)项目(2020)()
出版年
2024
NETL
NSTL
万方数据