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欧洲调节性T细胞疗法的障碍:从生产到监管。

Barriers to Treg therapy in Europe: From production to regulation.

作者信息

Hennessy Conor, Deptula Milena, Hester Joanna, Issa Fadi

机构信息

Transplantation Research and Immunology Group, Nuffield Department of Surgical Sciences, University of Oxford, Oxford, United Kingdom.

Laboratory of Tissue Engineering and Regenerative Medicine, Division of Embryology, Medical University of Gdańsk, Gdańsk, Poland.

出版信息

Front Med (Lausanne). 2023 Jan 19;10:1090721. doi: 10.3389/fmed.2023.1090721. eCollection 2023.

Abstract

There has been an increased interest in cell based therapies for a range of medical conditions in the last decade. This explosion in novel therapeutics research has led to the development of legislation specifically focused on cell and gene based therapies. In Europe, the European medicines agency (EMA) designates any medicines for human use which are based on genes, tissues, or cells as advanced therapy medicinal products or advanced therapy medicinal products (ATMPs). In this article we discuss the hurdles to widespread adoption of ATMPs in Europe, with a focus on regulatory T cells (Tregs). There are numerous barriers which must be overcome before mainstream adoption of Treg therapy becomes a reality. The source of the cells, whether to use autologous or allogenic cells, and the methods through which they are isolated and expanded, must all meet strict good manufacturing practice (GMP) standards to allow use of the products in humans. GMP compliance is costly, with the equipment and reagents providing a significant cost barrier and requiring specialized facilities and personnel. Conforming to the regulations set centrally by the EMA is difficult, and the different interpretations of the regulations across the various member states further complicates the regulatory approval process. The end products then require a complex and robust distribution network to ensure timely delivery of potentially life saving treatments to patients. In a European market whose logistics networks have been hammered by COVID and Brexit, ensuring rapid and reliable delivery systems is a more complex task than ever. In this article we will examine the impact of these barriers on the development and adoption of Tregs in Europe, and potential approaches which could facilitate more widespread use of Tregs, instead of its current concentration in a few very specialized centers.

摘要

在过去十年中,人们对基于细胞的疗法治疗一系列医疗病症的兴趣与日俱增。新型治疗方法研究的激增导致了专门针对细胞和基因疗法的立法的出台。在欧洲,欧洲药品管理局(EMA)将任何基于基因、组织或细胞的人类用药品指定为高级治疗用药品或高级治疗用药品(ATMPs)。在本文中,我们将讨论欧洲广泛采用ATMPs的障碍,重点是调节性T细胞(Tregs)。在Treg疗法成为主流疗法之前,有许多障碍必须克服。细胞的来源,无论是使用自体细胞还是异体细胞,以及分离和扩增细胞的方法,都必须符合严格的良好生产规范(GMP)标准,才能将产品用于人体。符合GMP要求成本高昂,设备和试剂构成了巨大的成本障碍,并且需要专门的设施和人员。遵守EMA集中制定的法规很困难,而且各成员国对法规的不同解释进一步使监管审批过程复杂化。最终产品还需要一个复杂而强大的分销网络,以确保将潜在的救命治疗及时提供给患者。在一个物流网络受到新冠疫情和英国脱欧冲击的欧洲市场,确保快速可靠的配送系统比以往任何时候都更加复杂。在本文中,我们将研究这些障碍对欧洲Tregs的开发和采用的影响,以及可能促进Tregs更广泛使用的潜在方法,而不是目前集中在少数非常专业的中心。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4b96/9892909/6d06973910b7/fmed-10-1090721-g001.jpg

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