Manufacturing of Human Extracellular Vesicle-Based Therapeutics for Clinical Use
Open Access
- 3 June 2017
- journal article
- editorial
- Published by MDPI AG in International Journal of Molecular Sciences
- Vol. 18 (6), 1190
- https://doi.org/10.3390/ijms18061190
Abstract
Extracellular vesicles (EVs) derived from stem and progenitor cells may have therapeutic effects comparable to their parental cells and are considered promising agents for the treatment of a variety of diseases. To this end, strategies must be designed to successfully translate EV research and to develop safe and efficacious therapies, whilst taking into account the applicable regulations. Here, we discuss the requirements for manufacturing, safety, and efficacy testing of EVs along their path from the laboratory to the patient. Development of EV-therapeutics is influenced by the source cell types and the target diseases. In this article, we express our view based on our experience in manufacturing biological therapeutics for routine use or clinical testing, and focus on strategies for advancing mesenchymal stromal cell (MSC)-derived EV-based therapies. We also discuss the rationale for testing MSC-EVs in selected diseases with an unmet clinical need such as critical size bone defects, epidermolysis bullosa and spinal cord injury. While the scientific community, pharmaceutical companies and clinicians are at the point of entering into clinical trials for testing the therapeutic potential of various EV-based products, the identification of the mode of action underlying the suggested potency in each therapeutic approach remains a major challenge to the translational path.Keywords
This publication has 93 references indexed in Scilit:
- Stem Cell Extracellular Vesicles: Extended Messages of RegenerationAnnual Review of Pharmacology and Toxicology, 2017
- Dendritic cell-derived exosomes as maintenance immunotherapy after first line chemotherapy in NSCLCOncoImmunology, 2015
- Applying extracellular vesicles based therapeutics in clinical trials – an ISEV position paperJournal of Extracellular Vesicles, 2015
- Therapeutic MSC exosomes are derived from lipid raft microdomains in the plasma membraneJournal of Extracellular Vesicles, 2013
- Membrane vesicles, current state-of-the-art: emerging role of extracellular vesiclesCellular and Molecular Life Sciences, 2011
- Enabling a robust scalable manufacturing process for therapeutic exosomes through oncogenic immortalization of human ESC-derived MSCsJournal of Translational Medicine, 2011
- Exosome secreted by MSC reduces myocardial ischemia/reperfusion injuryStem Cell Research, 2010
- A phase I study of dexosome immunotherapy in patients with advanced non-small cell lung cancerJournal of Translational Medicine, 2005
- Vaccination of metastatic melanoma patients with autologous dendritic cell (DC) derived-exosomes: results of thefirst phase I clinical trialJournal of Translational Medicine, 2005
- Eradication of established murine tumors using a novel cell-free vaccine: dendritic cell derived exosomesNature Medicine, 1998