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

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Abstract
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.
Keywords
immunology, neurobiology, haematology, stem cells, tissue regeneration, tumour vaccination, regulation, MESENCHYMAL STEM-CELLS, ENDOTHELIAL PROGENITOR CELLS, OUTER-MEMBRANE VESICLES, REGULATORY T-CELLS, VERSUS-HOST-DISEASE, TUMOR-DERIVED EXOSOMES, UMBILICAL-CORD BLOOD, NANOPARTICLE TRACKING ANALYSIS, ISCHEMIA-REPERFUSION INJURY, FIELD-FLOW FRACTIONATION

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MLA
Lener, Thomas, Mario Gimona, Ludwig Aigner, et al. “Applying Extracellular Vesicles Based Therapeutics in Clinical Trials : an ISEV Position Paper.” JOURNAL OF EXTRACELLULAR VESICLES 4 (2015): n. pag. Print.
APA
Lener, T., Gimona, M., Aigner, L., Börger, V., Buzas, E., Camussi, G., Chaput, N., et al. (2015). Applying extracellular vesicles based therapeutics in clinical trials : an ISEV position paper. JOURNAL OF EXTRACELLULAR VESICLES, 4.
Chicago author-date
Lener, Thomas, Mario Gimona, Ludwig Aigner, Verena Börger, Edit Buzas, Giovanni Camussi, Nathalie Chaput, et al. 2015. “Applying Extracellular Vesicles Based Therapeutics in Clinical Trials : an ISEV Position Paper.” Journal of Extracellular Vesicles 4.
Chicago author-date (all authors)
Lener, Thomas, Mario Gimona, Ludwig Aigner, Verena Börger, Edit Buzas, Giovanni Camussi, Nathalie Chaput, Devasis Chatterjee, Felipe A Court, Hernando A del Portillo, Lorraine O’Driscoll, Stefano Fais, Juan M Falcon-Perez, Ursula Felderhoff-Mueser, Lorenzo Fraile, Yong Song Gho, André Görgens, Ramesh C Gupta, An Hendrix, Dirk M Hermann, Andrew F Hill, Fred Hochberg, Peter A Horn, Dominique de Kleijn, Lambros Kordelas, Boris W Kramer, Eva-Maria Krämer-Albers, Sandra Laner-Plamberger, Saara Laitinen, Tommaso Leonardi, Magdalena J Lorenowicz, Sai Kiang Lim, Jan Lötvall, Casey A Maguire, Antonio Marcilla, Irina Nazarenko, Takahiro Ochiya, Tushar Patel, Shona Pedersen, Gabriella Pocsfalvi, Stefano Pluchino, Peter Quesenberry, Ilona G Reischl, Francisco J Rivera, Ralf Sanzenbacher, Katharina Schallmoser, Ineke Slaper-Cortenbach, Dirk Strunk, Torsten Tonn, Pieter Vader, Bas WM van Balkom, Marca Wauben, Samir El Andaloussi, Clotilde Théry, Eva Rohde, and Bernd Giebel. 2015. “Applying Extracellular Vesicles Based Therapeutics in Clinical Trials : an ISEV Position Paper.” Journal of Extracellular Vesicles 4.
Vancouver
1.
Lener T, Gimona M, Aigner L, Börger V, Buzas E, Camussi G, et al. Applying extracellular vesicles based therapeutics in clinical trials : an ISEV position paper. JOURNAL OF EXTRACELLULAR VESICLES. 2015;4.
IEEE
[1]
T. Lener et al., “Applying extracellular vesicles based therapeutics in clinical trials : an ISEV position paper,” JOURNAL OF EXTRACELLULAR VESICLES, vol. 4, 2015.
@article{7094476,
  abstract     = {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.},
  articleno    = {30087},
  author       = {Lener, Thomas and Gimona, Mario and Aigner, Ludwig and Börger, Verena and Buzas, Edit and Camussi, Giovanni and Chaput, Nathalie and Chatterjee, Devasis and Court, Felipe A and del Portillo, Hernando A and O'Driscoll, Lorraine and Fais, Stefano and Falcon-Perez, Juan M and Felderhoff-Mueser, Ursula and Fraile, Lorenzo and Gho, Yong Song and Görgens, André and Gupta, Ramesh C and Hendrix, An and Hermann, Dirk M and Hill, Andrew F and Hochberg, Fred and Horn, Peter A and de Kleijn, Dominique and Kordelas, Lambros and Kramer, Boris W and Krämer-Albers, Eva-Maria and Laner-Plamberger, Sandra and Laitinen, Saara and Leonardi, Tommaso and Lorenowicz, Magdalena J and Lim, Sai Kiang and Lötvall, Jan and Maguire, Casey A and Marcilla, Antonio and Nazarenko, Irina and Ochiya, Takahiro and Patel, Tushar and Pedersen, Shona and Pocsfalvi, Gabriella and Pluchino, Stefano and Quesenberry, Peter and Reischl, Ilona G and Rivera, Francisco J and Sanzenbacher, Ralf and Schallmoser, Katharina and Slaper-Cortenbach, Ineke and Strunk, Dirk and Tonn, Torsten and Vader, Pieter and van Balkom, Bas WM and Wauben, Marca and El Andaloussi, Samir and Théry, Clotilde and Rohde, Eva and Giebel, Bernd},
  issn         = {2001-3078},
  journal      = {JOURNAL OF EXTRACELLULAR VESICLES},
  keywords     = {immunology,neurobiology,haematology,stem cells,tissue regeneration,tumour vaccination,regulation,MESENCHYMAL STEM-CELLS,ENDOTHELIAL PROGENITOR CELLS,OUTER-MEMBRANE VESICLES,REGULATORY T-CELLS,VERSUS-HOST-DISEASE,TUMOR-DERIVED EXOSOMES,UMBILICAL-CORD BLOOD,NANOPARTICLE TRACKING ANALYSIS,ISCHEMIA-REPERFUSION INJURY,FIELD-FLOW FRACTIONATION},
  language     = {eng},
  pages        = {31},
  title        = {Applying extracellular vesicles based therapeutics in clinical trials : an ISEV position paper},
  url          = {http://dx.doi.org/10.3402/jev.v4.30087},
  volume       = {4},
  year         = {2015},
}

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