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

STEM CELLS TRANSLATIONAL MEDICINE, 2017 · DOI: 10.1002/sctm.17-0055 · Published: July 17, 2017

Regenerative Medicine Genetics Research Methodology & Design

Simple Explanation

Extracellular vesicles (EVs) are gaining attention as therapeutic tools, particularly in stem cell treatments. The International Society for Extracellular Vesicles and the Society for Clinical Research and Translation of Extracellular Vesicles Singapore convened a workshop to discuss the development of EV-based therapeutics. The workshop aimed to create best-practice models for using EVs in therapy, addressing the challenges and opportunities at preclinical and clinical stages. The review outlines specific actions that, if taken, will improve EV therapy development. EVs transmit signals between cells and interact with target cells through various mechanisms, making them potential candidates for therapeutics. They have shown therapeutic properties in areas like wound healing, inflammation, and cardiovascular disease.

Study Duration

Not specified

Participants

Not specified

Evidence Level

Level 5, Review

Key Findings

1
EVs, especially from mesenchymal stem cells (MSCs), have therapeutic properties for wound healing, inflammation, cardiovascular disease, brain injury, and cancers.
2
EVs can have immunomodulatory effects, leading to the development of EV vaccine platforms and potential treatments for immune diseases like graft-versus-host disease (GVHD).
3
The manufacture, storage, transport and end-use of nonviable stem cell EV therapeutics, as opposed to viable stem cell therapeutics, are less complex.

Research Summary

The review addresses the growing interest in EVs as therapeutic entities, particularly in stem cell-related approaches, and the need for standardization and coordination of development efforts. It outlines topic-specific action items to enhance the development of best-practice models for EV therapies, covering EV production, isolation, preclinical and clinical studies, and the role of animal models. The review also discusses challenges in the clinical use of EV-based therapeutics, including regulatory considerations, risk assessment, manufacturing processes, and maximizing research participation.

Practical Implications

Standardization of EV Production

Establish standardized cell culture conditions and optimize scalable purification systems to ensure reproducible EV function.

Development of Assays

Develop reliable fingerprinting, potency, and mechanistic assays to evaluate EV-based products effectively.

Clinical Trial Design

Incorporate noninferiority and superiority testing parameters to compare EV-based therapeutics with current standards of care.

Study Limitations

1
Lack of standardized assays for safety testing of EV-based therapeutics.
2
Challenges in defining the active ingredient in EV-based therapeutics for regulatory purposes.
3
Uncertainties regarding the long-term risks associated with EV therapies, such as potential oncogenic effects.

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