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脊髓损伤药物治疗:截至2015年的当前研究与开发及竞争激烈的商业格局。

Spinal cord injury pharmacotherapy: Current research & development and competitive commercial landscape as of 2015.

作者信息

Guercio Jason R, Kralic Jason E, Marrotte Eric J, James Michael L

机构信息

a North American Partners in Anesthesiology , New Britain, Connecticuit , USA.

b Innervate BD Solutions, LLC , Hillsborough, North Carolina , USA.

出版信息

J Spinal Cord Med. 2019 Jan;42(1):102-122. doi: 10.1080/10790268.2018.1439803. Epub 2018 Feb 27.

Abstract

CONTEXT

Current treatment of spinal cord injury (SCI) focuses on cord stabilization to prevent further injury, rehabilitation, management of non-motor symptoms, and prevention of complications. Currently, no approved treatments are available, and limited treatment options exist for symptoms and complications associated with chronic SCI. This review describes the pharmacotherapy landscape in SCI from both commercial and research and development (R&D) standpoints through March 2015.

METHODS

Information about specific compounds has been obtained through drug pipeline monographs in the Pharmaprojects® (Citeline, Inc., New York, New York, USA) drug database (current as of a search on May 30, 2014), websites of individual companies with compounds in development for SCI (current as of March 24, 2015), and a literature search of published R&D studies to validate the Pharmaprojects® source for selected compounds (current as of March 24, 2015).

RESULTS

Types of studies conducted and outcomes measured in earlier phases of development are described for compounds in clinical development Currently four primary mechanisms are under investigation and may yield promising therapeutic targets: 1) neuronal regeneration; 2) neuroprotection (including anti-inflammation); 3) axonal reconnection; and 4) neuromodulation and signal enhancement. Many other compounds are no longer under investigation for SCI are mentioned; however, in most cases, the reason for terminating their development is not clear.

CONCLUSION

There is urgent need to develop disease-modifying therapy for SCI, yet the commercial landscape remains small and highly fragmented with a paucity of novel late-stage compounds in R&D.

摘要

背景

目前脊髓损伤(SCI)的治疗重点在于脊髓稳定以防止进一步损伤、康复治疗、非运动症状的管理以及并发症的预防。目前尚无获批的治疗方法,对于慢性SCI相关的症状和并发症,治疗选择有限。本综述从商业和研发(R&D)的角度描述了截至2015年3月脊髓损伤的药物治疗情况。

方法

关于特定化合物的信息通过《药物项目》(美国纽约市Citeline公司)药物数据库中的药物研发专论(截至2014年5月30日的搜索结果)、有正在研发用于脊髓损伤化合物的各公司网站(截至2015年3月24日)以及对已发表的研发研究进行文献检索以验证所选化合物的《药物项目》来源(截至2015年3月24日)获得。

结果

描述了处于临床研发阶段的化合物在早期研发阶段所进行的研究类型和测量的结果。目前正在研究四种主要机制,可能会产生有前景的治疗靶点:1)神经元再生;2)神经保护(包括抗炎);3)轴突重新连接;4)神经调节和信号增强。还提到了许多其他不再针对脊髓损伤进行研究的化合物;然而,在大多数情况下,终止其研发的原因尚不清楚。

结论

迫切需要开发针对脊髓损伤的疾病修饰疗法,但商业前景仍然狭小且高度分散,研发中缺乏新型晚期化合物。

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本文引用的文献

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Development of a database for translational spinal cord injury research.用于转化性脊髓损伤研究的数据库开发。
J Neurotrauma. 2014 Nov 1;31(21):1789-99. doi: 10.1089/neu.2014.3399. Epub 2014 Jul 31.
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Spinal cord injury models: a review.脊髓损伤模型:综述
Spinal Cord. 2014 Aug;52(8):588-95. doi: 10.1038/sc.2014.91. Epub 2014 Jun 10.
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Nanovector-mediated drug delivery for spinal cord injury treatment.用于脊髓损伤治疗的纳米载体介导的药物递送
Wiley Interdiscip Rev Nanomed Nanobiotechnol. 2014 Sep-Oct;6(5):506-15. doi: 10.1002/wnan.1276. Epub 2014 May 21.

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