Functional assays to assess the therapeutic potential of extracellular vesicles

J Extracell Vesicles, 2020 · DOI: 10.1002/jev2.12033 · Published: October 26, 2020

Pharmacology Genetics Research Methodology & Design

Simple Explanation

Extracellular vesicles (EVs) are important for cell communication and can be used for therapies, but it's crucial to identify and measure their key characteristics to ensure consistent therapeutic effects. Before clinical trials, EV features should be tested using standardized assays to predict their therapeutic potential and allow comparison between different EV preparations. There are challenges in setting up reliable assays for EVs due to their complexity, so this paper discusses in vitro and in vivo testing, the need for standardization, and new developments for functional testing.

Study Duration

Not specified

Participants

Not specified

Evidence Level

Review Article

Key Findings

1
Functional assays are crucial for assessing the molecular and physiological effects of EV preparations on target cells, organoids, organs, or organisms.
2
Formal potency assays, as defined by regulatory guidelines, are needed to indicate the specific ability of EVs to achieve a therapeutic result based on a defined biological effect.
3
Harmonized functional assays are essential for global comparison of EV studies, describing a quantifiable effect of a certain dose of EVs based on reproducible and uniformly implemented tests.

Research Summary

The development of EV therapeutics hinges on the ability to consistently produce EVs with reliable therapeutic efficacy, necessitating well-standardized assays. Functional assays, which evaluate the effects of EVs on target cells or organisms, are distinct from formal potency assays, which regulatory bodies define as directly indicative of a product's ability to achieve a specific therapeutic result. Standardizing functional assays and defining functional units for EVs are critical steps toward enabling comparisons between EV preparations and facilitating clinical translation, though significant challenges remain in addressing EV heterogeneity and assay variability.

Practical Implications

Standardized Potency Assays

The development of standardized potency assays will allow for better prediction of therapeutic efficacy and facilitate regulatory approval of EV-based therapies.

Improved Comparability

Harmonized functional assays will enable researchers to compare results across different EV preparations, batches, and laboratories, accelerating the advancement of the field.

Clinical Translation

Robust functional and safety testing, including in vitro and in vivo assays, are essential for translating EV-based therapeutics to clinical applications and ensuring patient safety.

Study Limitations

1
Heterogeneity in EV-preparation procedures.
2
Absence of uniform quality control (QC) criteria.
3
Necessity of alignment with national and international regulatory guidelines to reach clinical testing.

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