在Charcot-Marie-Tooth疾病的临床试验:回顾性和临床前评估
Malavika A Nair1, Zhiyv Niu2,3, Nicholas N Madigan4
1Department of Graduate Education, Alix School of Medicine, Rochester, MN, United States.
Frontiers in neurology
|October 9, 2023
概括
针对Charcot-Marie-Tooth (CMT) 疾病的临床试验显著增加,重点是新型基因疗法和程序治疗. 虽然许多有前途的分子疗法仍处于临床前阶段,但人类试验正在推进,显示整个行业的参与.
科学领域:
- 神经学 神经学
- 遗传学 遗传学 是一个
- 临床试验 临床试验
背景情况:
- 查尔科-玛丽-牙 (CMT) 历史上通过症状治疗和遗传咨询来管理.
- 分子和病理学理解的进步为基因向疗法提供了希望.
研究的目的:
- 评估查科-玛丽- (CMT) 疾病临床和临床前试验的进展情况.
- 分析治疗方法和行业参与的趋势.
主要方法:
- 从临床试验.gov (1999-2022) 中提取的数据用于CMT临床试验.
- 在PubMed搜索CMT中进行的临床前动物基因治疗试验.
主要成果:
- 活跃的CMT试验从1999年的1个跃升到2022年的286个,其中学术参与率为91%,制药参与率为9%.
- 治疗试验 (86%) 包括程序 (50%),试验药物 (23%),设备 (15%) 和物理治疗 (11%).
- 第一阶段的人体试验重点是小分子和基因疗法;临床前研究探索11种CMT形式的多种基因疗法方法.
结论:
- 目前的CMT试验探讨了过程性和分子疗法,全球制药行业参与其中.
- 新兴的分子向药物疗法正在人类试验中取得进展,尽管大多数仍在动物研究中.
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