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子痫前期——孤儿药地位会促进创新性生物疗法的发展吗?

Preeclampsia - will orphan drug status facilitate innovative biological therapies?

作者信息

Hahn Sinuhe

机构信息

Laboratory for Prenatal Medicine, Department of Biomedicine, University Hospital Basel , Basel , Switzerland.

出版信息

Front Surg. 2015 Feb 26;2:7. doi: 10.3389/fsurg.2015.00007. eCollection 2015.

DOI:10.3389/fsurg.2015.00007
PMID:25767802
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4341571/
Abstract

It is generally accepted that the development of novel therapies to treat pregnancy-related disorders, such as preeclampsia, is hampered by the paucity of research funding. Hence, it is with great interest to become aware of at least three novel therapeutic approaches for the treatment of this disorder: exploiting either the anticoagulant activity of antithrombin, the free radical scavenging activity of alpha-1-microglobulin, or the regenerative capacity of placenta-derived mesenchymal stem cells. As these projects are being carried out by small biotech enterprises, the question arises of how they are able to fund such undertakings. A novel strategy adopted by two of these companies is that they successfully petitioned US and EU agencies in order that preeclampsia is accepted in the register of rare or orphan diseases. This provides a number of benefits including market exclusivity, assistance with clinical trials, and dedicated funding schemes. Other strategies to supplement meager research funds, especially to test novel approaches, could be crowdfunding, a venture that relies on intimate interaction with advocacy groups. In other words, preeclampsia meets Facebook. Perhaps similar strategies can be adopted to examine novel therapies targeting either the imbalance in pro- or anti-angiogenic growth factors, complement activation, reduced levels of placenta protein 13, or excessive neutrophil activation evident in preeclampsia.

摘要

人们普遍认为,用于治疗与妊娠相关疾病(如先兆子痫)的新型疗法的开发受到研究资金匮乏的阻碍。因此,了解至少三种治疗该疾病的新型治疗方法会让人颇感兴趣:利用抗凝血酶的抗凝活性、α-1-微球蛋白的自由基清除活性或胎盘来源间充质干细胞的再生能力。由于这些项目是由小型生物技术企业开展的,因此就出现了它们如何为这些事业提供资金的问题。其中两家公司采用的一种新策略是,它们成功地向美国和欧盟机构请愿,以使先兆子痫被纳入罕见病或孤儿病登记册。这带来了许多好处,包括市场独占权、临床试验援助和专项资助计划。补充微薄研究资金的其他策略,尤其是用于测试新方法的策略,可以是众筹,这是一种依赖与倡导团体密切互动的活动。换句话说,先兆子痫遇上了脸书。或许可以采用类似策略来研究针对先兆子痫中促血管生成或抗血管生成生长因子失衡、补体激活、胎盘蛋白13水平降低或中性粒细胞过度激活的新型疗法。

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