Concise Review: Developing Best-Practice Models for the Therapeutic Use of Extracellular Vesicles

Agnes T Reiner¹, Kenneth W Witwer^{2

3}, Bas W M van Balkom⁴, Joel de Beer⁵, Chaya Brodie^{6

7}, Randolph L Corteling⁸, Susanne Gabrielsson⁹, Mario Gimona^{10

11}, Ahmed G Ibrahim¹², Dominique de Kleijn^{13

14}, Charles P Lai¹⁵, Jan Lötvall^{16

17}, Hernando A Del Portillo^{18

19}, Ilona G Reischl^{20

21}, Milad Riazifar^{22

23}, Carlos Salomon^{24

25}, Hidetoshi Tahara²⁶, Wei Seong Toh²⁷, Marca H M Wauben²⁸, Vicky K Yang²⁹, Yijun Yang³⁰, Ronne Wee Yeh Yeo³¹, Hang Yin^{32

33}, Bernd Giebel³⁴, Eva Rohde^{10

11}, Sai Kiang Lim³¹

Affiliations

¹ BioSensor Technologies, AIT Austrian Institute of Technology, Vienna, Austria.
² Departments of Molecular and Comparative Pathobiology, The Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
³ Neurology, The Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
⁴ Department of Nephrology and Hypertension, University Medical Center Utrecht, Utrecht, The Netherlands.
⁵ Anjarium Biosciences AG, Zollikon, Switzerland.
⁶ Department of Neurosurgery, Henry Ford Hospital, Detroit, Michigan, USA.
⁷ Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan, Israel.
⁸ ReNeuron Group, Pencoed, United Kingdom.
⁹ Department of Medicine, Unit for Immunology and Allergy, Karolinska Institute, Stockholm, Sweden.
¹⁰ Spinal Cord Injury & Tissue Regeneration Center Salzburg (SCI-TReCS), Paracelsus Medical University (PMU), Salzburg, Austria.
¹¹ Department of Blood Group Serology and Transfusion Medicine, University Hospital, Salzburger Landeskliniken GesmbH (SALK), Salzburg, Austria.
¹² Capricor Therapeutics, Los Angeles, California, USA.
¹³ Dept. of Vascular Surgery & Cardiology, Utrecht University, Utrecht, The Netherlands.
¹⁴ NUS Surgery & A-STAR, Singapore.
¹⁵ Institute of Biomedical Engineering, National Tsing Hua University, Hsinchu, Taiwan, Republic of China.
¹⁶ Krefting Research Centre, Institute of Medicine, The Sahlgrenska Academy, Gothenburg University, Gothenburg, Sweden.
¹⁷ Codiak BioSciences, Woburn, Massachusetts, USA.
¹⁸ ICREA at ISGlobal Barcelona Institute for Global Health, Ctr. Int. Health Res. (CRESIB), Hospital Clínic, University of Barcelona, Barcelona, Spain.
¹⁹ Institut d'Investigació Germans Trias i Pujol (IGTP), Badalona, Spain.
²⁰ Federal Office for Safety in Health Care, Institute Surveillance, Vienna, Austria.
²¹ Austrian Agency for Health and Food Safety, Institute Surveillance, Vienna, Austria.
²² Department of Pharmaceutical Sciences, University of California, Irvine, California, USA.
²³ Sue and Bill Gross Stem Cell Research Center, University of California, Irvine, California, USA.
²⁴ Centre for Clinical Research, Royal Brisbane and Women's Hospital, The University of Queensland, Brisbane, Australia.
²⁵ Department of Obstetrics and Gynecology, Ochsner Clinic Foundation, New Orleans, Louisiana, USA.
²⁶ Department of Cellular and Molecular Biology, Institute of Biomedical & Health Sciences, Hiroshima University, Hiroshima, Japan.
²⁷ Faculty of Dentistry, National University of Singapore, Singapore.
²⁸ Department of Biochemistry and Cell Biology, Faculty of Veterinary Medicine, Utrecht University, Utrecht, The Netherlands.
²⁹ Department of Clinical Sciences, Tufts University Cummings School of Veterinary Medicine, North Grafton, Massachusetts, USA.
³⁰ State Key Laboratory of Respiratory Disease, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, China.
³¹ Institute of Medical Biology, A*STAR, Singapore.
³² Department of Chemistry and Biochemistry and the BioFrontiers Institute, University of Colorado Boulder, Boulder, Colorado, USA.
³³ Center of Basic Molecular Science (CBMS), Department of Chemistry, Tsinghua University, Beijing, China.
³⁴ Institute for Transfusion Medicine, University Hospital Essen, University Duisburg-Essen, Germany.

PMID: 28714557
PMCID: PMC5689784
DOI: 10.1002/sctm.17-0055

Review

Concise Review: Developing Best-Practice Models for the Therapeutic Use of Extracellular Vesicles

Agnes T Reiner et al. Stem Cells Transl Med. 2017 Aug.

. 2017 Aug;6(8):1730-1739.

doi: 10.1002/sctm.17-0055. Epub 2017 Jul 17.

Authors

Affiliations

¹ BioSensor Technologies, AIT Austrian Institute of Technology, Vienna, Austria.
² Departments of Molecular and Comparative Pathobiology, The Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
³ Neurology, The Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
⁴ Department of Nephrology and Hypertension, University Medical Center Utrecht, Utrecht, The Netherlands.
⁵ Anjarium Biosciences AG, Zollikon, Switzerland.
⁶ Department of Neurosurgery, Henry Ford Hospital, Detroit, Michigan, USA.
⁷ Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan, Israel.
⁸ ReNeuron Group, Pencoed, United Kingdom.
⁹ Department of Medicine, Unit for Immunology and Allergy, Karolinska Institute, Stockholm, Sweden.
¹⁰ Spinal Cord Injury & Tissue Regeneration Center Salzburg (SCI-TReCS), Paracelsus Medical University (PMU), Salzburg, Austria.
¹¹ Department of Blood Group Serology and Transfusion Medicine, University Hospital, Salzburger Landeskliniken GesmbH (SALK), Salzburg, Austria.
¹² Capricor Therapeutics, Los Angeles, California, USA.
¹³ Dept. of Vascular Surgery & Cardiology, Utrecht University, Utrecht, The Netherlands.
¹⁴ NUS Surgery & A-STAR, Singapore.
¹⁵ Institute of Biomedical Engineering, National Tsing Hua University, Hsinchu, Taiwan, Republic of China.
¹⁶ Krefting Research Centre, Institute of Medicine, The Sahlgrenska Academy, Gothenburg University, Gothenburg, Sweden.
¹⁷ Codiak BioSciences, Woburn, Massachusetts, USA.
¹⁸ ICREA at ISGlobal Barcelona Institute for Global Health, Ctr. Int. Health Res. (CRESIB), Hospital Clínic, University of Barcelona, Barcelona, Spain.
¹⁹ Institut d'Investigació Germans Trias i Pujol (IGTP), Badalona, Spain.
²⁰ Federal Office for Safety in Health Care, Institute Surveillance, Vienna, Austria.
²¹ Austrian Agency for Health and Food Safety, Institute Surveillance, Vienna, Austria.
²² Department of Pharmaceutical Sciences, University of California, Irvine, California, USA.
²³ Sue and Bill Gross Stem Cell Research Center, University of California, Irvine, California, USA.
²⁴ Centre for Clinical Research, Royal Brisbane and Women's Hospital, The University of Queensland, Brisbane, Australia.
²⁵ Department of Obstetrics and Gynecology, Ochsner Clinic Foundation, New Orleans, Louisiana, USA.
²⁶ Department of Cellular and Molecular Biology, Institute of Biomedical & Health Sciences, Hiroshima University, Hiroshima, Japan.
²⁷ Faculty of Dentistry, National University of Singapore, Singapore.
²⁸ Department of Biochemistry and Cell Biology, Faculty of Veterinary Medicine, Utrecht University, Utrecht, The Netherlands.
²⁹ Department of Clinical Sciences, Tufts University Cummings School of Veterinary Medicine, North Grafton, Massachusetts, USA.
³⁰ State Key Laboratory of Respiratory Disease, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, China.
³¹ Institute of Medical Biology, A*STAR, Singapore.
³² Department of Chemistry and Biochemistry and the BioFrontiers Institute, University of Colorado Boulder, Boulder, Colorado, USA.
³³ Center of Basic Molecular Science (CBMS), Department of Chemistry, Tsinghua University, Beijing, China.
³⁴ Institute for Transfusion Medicine, University Hospital Essen, University Duisburg-Essen, Germany.

PMID: 28714557
PMCID: PMC5689784
DOI: 10.1002/sctm.17-0055

Abstract

Growing interest in extracellular vesicles (EVs, including exosomes and microvesicles) as therapeutic entities, particularly in stem cell-related approaches, has underlined the need for standardization and coordination of development efforts. Members of the International Society for Extracellular Vesicles and the Society for Clinical Research and Translation of Extracellular Vesicles Singapore convened a Workshop on this topic to discuss the opportunities and challenges associated with development of EV-based therapeutics at the preclinical and clinical levels. This review outlines topic-specific action items that, if addressed, will enhance the development of best-practice models for EV therapies. Stem Cells Translational Medicine 2017;6:1730-1739.

Keywords: Cellular therapy; Clinical translation; Clinical trials; Exosomes; Extracellular vesicles; Microvesicles; Stem cells; Therapeutics.

PubMed Disclaimer

Figures

**Figure 1**
Preclinical studies toward Investigational New Drug filings.

See this image and copyright information in PMC

References

1. Yáñez‐ Mó M, Siljander PR‐M, Andreu Z et al. Biological properties of extracellular vesicles and their physiological functions. J Extracell Vesicles 2015;4:27066. - PMC - PubMed
1. Simons M, Raposo G. Exosomes–vesicular carriers for intercellular communication. Curr Opin Cell Biol 2009;21:575–581. - PubMed
1. Tkach M, Théry C. Communication by extracellular vesicles: Where we are and where we need to go. Cell 2016;164:1226–1232. - PubMed
1. Lener T, Gimona M, Aigner L et al. Applying extracellular vesicles based therapeutics in clinical trials ‐ an ISEV position paper. J Extracell Vesicles 2015;4:30087. - PMC - PubMed
1. Raposo G, Stoorvogel W. Extracellular vesicles: Exosomes, microvesicles, and friends. J Cell Biol 2013;200:373–383. - PMC - PubMed

Publication types

Actions

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

Grants and funding

R01 DA040385/DA/NIDA NIH HHS/United States

LinkOut - more resources

Full Text Sources
Other Literature Sources
- scite Smart Citations

Save citation to file

Email citation

Add to Collections

Add to My Bibliography

Your saved search

Create a file for external citation management software

Your RSS Feed

Concise Review: Developing Best-Practice Models for the Therapeutic Use of Extracellular Vesicles

Affiliations

Concise Review: Developing Best-Practice Models for the Therapeutic Use of Extracellular Vesicles

Authors

Affiliations

Abstract

Figures

References

Publication types

MeSH terms

Grants and funding

LinkOut - more resources

Full Text Sources

Other Literature Sources