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多发性硬化症药物发现中动物模型置信度评估的反向翻译。

Reverse Translation for Assessment of Confidence in Animal Models of Multiple Sclerosis for Drug Discovery.

机构信息

Department Immunobiology, Biomedical Primate Research Centre, Rijswijk, The Netherlands.

University of Groningen, University Medical Centre, Dept. Neuroscience, Groningen, The Netherlands.

出版信息

Clin Pharmacol Ther. 2018 Feb;103(2):262-270. doi: 10.1002/cpt.801. Epub 2017 Aug 29.

DOI:10.1002/cpt.801
PMID:28748631
Abstract

The poor predictive quality of currently used animal models in preclinical research is an important cause of the high attrition of promising drug candidates for human autoimmune disease in clinical trials. Examples from own work in a primate multiple sclerosis (MS) model illustrate that important lessons can be learned from a critical reassessment of failed drugs in the animal model, which can help improve the animal model and better understand the targeted disease.

摘要

目前在临床前研究中使用的动物模型预测质量较差,是导致在临床试验中具有前景的治疗人类自身免疫性疾病候选药物大量淘汰的一个重要原因。从我们在灵长类多发性硬化症 (MS) 模型中的工作中举例说明,从对动物模型中失败药物的严格重新评估中可以吸取重要的经验教训,这有助于改进动物模型并更好地了解目标疾病。

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Reverse Translation for Assessment of Confidence in Animal Models of Multiple Sclerosis for Drug Discovery.多发性硬化症药物发现中动物模型置信度评估的反向翻译。
Clin Pharmacol Ther. 2018 Feb;103(2):262-270. doi: 10.1002/cpt.801. Epub 2017 Aug 29.
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Front Ophthalmol (Lausanne). 2025 Jun 4;5:1610215. doi: 10.3389/fopht.2025.1610215. eCollection 2025.
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Multiple sclerosis and drug discovery: A work of translation.多发性硬化症与药物研发:一次转化之旅。
EBioMedicine. 2021 Jun;68:103392. doi: 10.1016/j.ebiom.2021.103392. Epub 2021 May 24.
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Experimental autoimmune encephalomyelitis in the common marmoset: a translationally relevant model for the cause and course of multiple sclerosis.
普通狨猴实验性自身免疫性脑脊髓炎:一种与多发性硬化病因及病程相关的转化模型
Primate Biol. 2019 May 10;6(1):17-58. doi: 10.5194/pb-6-17-2019. eCollection 2019.
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When Enough Is Enough: Decision Criteria for Moving a Known Drug into Clinical Testing for a New Indication in the Absence of Preclinical Efficacy Data.适可而止:在缺乏临床前疗效数据的情况下,将已知药物用于新适应症临床测试的决策标准。
Assay Drug Dev Technol. 2017 Dec;15(8):354-361. doi: 10.1089/adt.2017.821. Epub 2017 Dec 1.