Overview of Current and Emerging Therapies for Amyotrophic Lateral Sclerosis
Overview of Current and Emerging Therapies for Amyotrophic Lateral Sclerosis
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DOI：
10.37765/ajmc.2020.88483
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发表时间：
2020-08-01
影响因子：
3.2
通讯作者：
Chen, Jack J.
中科院分区：
文献类型：
--
作者：
Chen, Jack J.
肌萎缩侧索硬化症（ALS）是一种毁灭性的，致命的神经肌肉疾病。大多数患者在诊断后2至5年内死亡。这种疾病源于上、下运动神经元的死亡，导致运动通路的退化和疾病的麻痹作用。这种疾病的经济成本尚不清楚，估计每年约为64 000美元至200 000美元。两种药物，利鲁唑和依达拉奉，目前被FDA批准用于治疗ALS，并且每种药物在死亡率和/或功能方面都提供了适度的益处。对导致ALS的潜在病理生理过程的理解的最新进展导致了许多研究性疗法的开发，其中一些现在处于3期试验中。本文重点介绍了口服酪氨酸激酶抑制剂马赛替尼;反义药物tofersen;人源化单克隆抗体C5补体抑制剂ravulizumab-cwvz;和间充质干细胞（MSC）-神经营养因子（NTF）细胞，一种诱导自体骨髓来源的MSC分泌高水平NTF的专有平台。
Amyotrophic lateral sclerosis (ALS) is a devastating, fatal neuromuscular disease. Most patients die within 2 to 5 years of diagnosis. The disease stems from death of upper and lower motor neurons leading to degeneration of motor pathways and the paralytic effects of the disease. The economic cost of the disease is not clear, with estimates ranging from about $64,000 per year to $200,000. Two drugs, riluzole and edaravone, are currently FDA approved for the treatment of ALS, and each provides modest benefits in mortality and/or function. Recent developments in the understanding of the underlying pathophysiologic processes that contribute to ALS have led to the development of numerous investigational therapies, with several now in phase 3 trials. This article highlights the oral tyrosine kinase inhibitor masitinib; the antisense drug tofersen; the humanized monoclonal antibody C5 complement inhibitor ravulizumab-cwvz; and mesenchymal stem cell (MSC)-neurotrophic factor (NTF) cells, a proprietary platform that induces autologous bone marrow-derived MSCs to secrete high levels of NTFs.