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神经疾病生物疗法的发展:从前人的失败中吸取教训。

The development of biological therapies for neurological diseases: moving on from previous failures.

机构信息

a Division of Applied Regulatory Science, Office of Clinical Pharmacology, Office of Translational Sciences , Center for Drug Evaluation and Research, Food and Drug Administration , Silver Spring , MD , USA.

出版信息

Expert Opin Drug Discov. 2018 Apr;13(4):283-293. doi: 10.1080/17460441.2018.1437142. Epub 2018 Feb 7.

Abstract

Although years of research have expanded the use of biologics for several clinical conditions, such development has not yet occurred in the treatment of neurological diseases. With the advancement of biologic technologies, there is promise for these therapeutics as novel therapeutic approaches for neurological diseases. Areas covered: In this article, the authors review the therapeutic potential of different types of biologics for the treatment of neurological diseases. Preclinical and clinical studies that investigate the efficacy and safety of biologics in the treatment of neurological diseases, namely Alzheimer's disease, amyotrophic lateral sclerosis, Parkinson disease, multiple sclerosis, and stroke, were reviewed. Moreover, the authors describe the key challenges in the development of therapeutically safe and effective biologics for the treatment of neurological diseases. Expert opinion: Several biologics have shown promise in the treatment of neurological diseases. However, the complexity of the CNS, as well as a limited understanding of disease progression, and restricted access of biologics to the CNS has limited successful development. Therefore, more research needs to be conducted to overcome these hurdles before developing effective and safe biologics for neurological diseases. The emergence of new technologies for the design, production and delivery of biologics will accelerate translating biologics to the clinic.

摘要

尽管多年的研究已经扩大了生物制剂在几种临床病症中的应用,但在治疗神经疾病方面,这种发展尚未发生。随着生物技术的进步,这些疗法有望成为治疗神经疾病的新疗法。

涵盖领域

在本文中,作者综述了不同类型的生物制剂治疗神经疾病的治疗潜力。综述了探讨生物制剂在治疗神经疾病(如阿尔茨海默病、肌萎缩侧索硬化症、帕金森病、多发性硬化症和中风)中的疗效和安全性的临床前和临床研究。此外,作者还描述了治疗神经疾病的治疗安全有效的生物制剂开发中的关键挑战。

专家意见

几种生物制剂在治疗神经疾病方面显示出了希望。然而,中枢神经系统的复杂性、对疾病进展的有限了解以及生物制剂进入中枢神经系统的受限,限制了其成功开发。因此,在开发治疗神经疾病的有效和安全的生物制剂之前,需要进行更多的研究来克服这些障碍。用于生物制剂设计、生产和输送的新技术的出现将加速将生物制剂推向临床。

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