Applying extracellular vesicles based therapeutics in clinical trials - an ISEV position paper

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2015
Author
Lener, Thomas
Gimona, Mario
Aigner, Ludwig
Börger, Verena
Buzas, Edit
Camussi, Giovanni
Chaput, Nathalie
Court, Felipe A.
del Portillo, Hernando A.
O'Driscoll, Lorraine
Fais, Stefano
Falcon-Perez, Juan M.
Felderhoff-Mueser, Ursula
Fraile Sauce, Lorenzo José
Gho, Yong Song
Görgens, André
Gupta, Ramesh C.
Hendrix, An
Hermann, Dirk
Hill, Andrew F.
Hochberg, Fred
Horn, Peter A.
de kleijn, Dominique
Kordelas, Lambros
Kramer, Boris W.
Krämer-Albers, Eva-Maria
Laner-Plamberger, Sandra
Laitien, Saara
Leonardi, Tommaso
Lorenowicz, Magdalena J.
Lim, Sai Kiang
Lötvall, Jan
Maguire, Casey A.
Marcilla, Antonio
Nazarenko, Irina
Ochiya, Takahiro
Patel, Tushar
Pedersen, Shona
Pocsfalvi, Gabriella
Pluchino, Stefano
Quesenberry, Peter
Reischl, Ilona G.
Rivera, Francisco J.
Sanzenbacher, Ralf
Schallmoser, Katharina
Slaper-Cortenbach, Ineke
Strunk, Dirk
Tonn, Torsten
Vader, Pieter
Van Balkom, Bas W. M.
Wauben, Marca
El Andaloussi, Samir
Théry, Clotilde
Rohde, Eva
Giebel, Bernd
Suggested citation
Lener, Thomas; Gimona, Mario; Aigner, Ludwig; Börger, Verena; Buzas, Edit; Camussi, Giovanni; ... Giebel, Bernd. (2015) . Applying extracellular vesicles based therapeutics in clinical trials - an ISEV position paper. Journal of Extracellular Vesicles, 2015, vol. 4, p. 1-31. https://doi.org/10.3402/jev.v4.30087.
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Abstract
xtracellular 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.
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http://hdl.handle.net/10459.1/64670
DOI
https://doi.org/10.3402/jev.v4.30087
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Journal of Extracellular Vesicles, 2015, vol. 4, p. 1-31
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cc-by-nc, (c) Lener et at., 2015
Except where otherwise noted, this item's license is described as cc-by-nc, (c) Lener et at., 2015