Applying extracellular vesicles based therapeutics in clinical trials - an ISEV position paper.
Applying extracellular vesicles based therapeutics in clinical trials - an ISEV position paper.
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DOI：
10.3402/jev.v4.30087
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发表时间：
2015
影响因子：
16
通讯作者：
Giebel B
中科院分区：
文献类型：
--
作者：
Lener T;Gimona M;Aigner L;Börger V;Buzas E;Camussi G;Chaput N;Chatterjee D;Court FA;Del Portillo HA;O'Driscoll L;Fais S;Falcon-Perez JM;Felderhoff-Mueser U;Fraile L;Gho YS;Görgens A;Gupta RC;Hendrix A;Hermann DM;Hill AF;Hochberg F;Horn PA;de Kleijn D;Kordelas L;Kramer BW;Krämer-Albers EM;Laner-Plamberger S;Laitinen S;Leonardi T;Lorenowicz MJ;Lim SK;Lötvall J;Maguire CA;Marcilla A;Nazarenko I;Ochiya T;Patel T;Pedersen S;Pocsfalvi G;Pluchino S;Quesenberry P;Reischl IG;Rivera FJ;Sanzenbacher R;Schallmoser K;Slaper-Cortenbach I;Strunk D;Tonn T;Vader P;van Balkom BW;Wauben M;Andaloussi SE;Théry C;Rohde E;Giebel B
细胞外囊泡（EV），如外泌体和微泡，由不同类型的细胞释放，并参与生理和病理生理过程。EV作为细胞来源的细胞外信号细胞器介导细胞间通讯，将特定信息从其来源细胞传递到其靶细胞。由于这些特性，具有确定细胞类型的EV可以用作各种治疗方法的新工具，包括（a）抗肿瘤治疗，（B）病原体疫苗接种，（c）免疫调节和再生治疗以及（d）药物递送。将EV转化为临床治疗需要根据现有监管框架对基于EV的治疗进行分类。由于该分类定义了生产、质量控制和临床研究的后续要求，因此定义EV是否被视为活性药物组分或主要用作药物递送载体至关重要。为了将基于EV的治疗有效且特别安全地转化为临床实践，研究人员，临床医生和主管当局之间的高水平合作至关重要。在本立场声明中，基础和临床科学家作为国际细胞外囊泡学会（ISEV）和欧盟欧洲科学技术合作（COST）计划的成员，即欧洲微囊泡和外泌体健康与疾病网络（ME-HaD），总结了最近的发展和基于EV的治疗的当前知识。强调了药品生产和临床应用必须考虑的安全性和法规要求。讨论了生产和质量控制过程。在未来的临床研究中，促进EV的治疗应用的策略进行了讨论。
Extracellular vesicles (EVs), such as exosomes and microvesicles, are released by different cell types and participate in physiological and pathophysiological processes. EVs mediate intercellular communication as cell-derived extracellular signalling organelles that transmit specific information from their cell of origin to their target cells. As a result of these properties, EVs of defined cell types may serve as novel tools for various therapeutic approaches, including (a) anti-tumour therapy, (b) pathogen vaccination, (c) immune-modulatory and regenerative therapies and (d) drug delivery. The translation of EVs into clinical therapies requires the categorization of EV-based therapeutics in compliance with existing regulatory frameworks. As the classification defines subsequent requirements for manufacturing, quality control and clinical investigation, it is of major importance to define whether EVs are considered the active drug components or primarily serve as drug delivery vehicles. For an effective and particularly safe translation of EV-based therapies into clinical practice, a high level of cooperation between researchers, clinicians and competent authorities is essential. In this position statement, basic and clinical scientists, as members of the International Society for Extracellular Vesicles (ISEV) and of the European Cooperation in Science and Technology (COST) program of the European Union, namely European Network on Microvesicles and Exosomes in Health and Disease (ME-HaD), summarize recent developments and the current knowledge of EV-based therapies. Aspects of safety and regulatory requirements that must be considered for pharmaceutical manufacturing and clinical application are highlighted. Production and quality control processes are discussed. Strategies to promote the therapeutic application of EVs in future clinical studies are addressed.