Regulatory perspective on in vitro potency assays for human mesenchymal stromal cells used in immunotherapy

Charlotte de Wolf, Marja van de Bovenkamp, Marcel C Hoefnagel

    Research output: Contribution to journalArticleAcademicpeer-review

    Abstract

    Mesenchymal stromal cells (MSCs) are multipotent cells derived from various tissues that can differentiate into several cell types. MSCs are able to modulate the response of immune cells of the innate and adaptive immune system. Because of these multimodal properties, the potential use of MSCs for immunotherapies is currently explored in various clinical indications. Due to the diversity of potential MSC medicinal products at the level of cell source, manufacturing process and indication, distinct functionality tests may be needed to ensure the quality for each of the different products. In this review, we focus on in vitro potency assays proposed for characterization and release of different MSC medicinal products. We discuss the most used functional assays, as presented in scientific advices and literature, highlighting specific advantages and limitations of the various assays. Currently, the most proposed and accepted potency assay for release is based on in vitro inhibition of T cell proliferation or other functionalities. However, for some products, assays based on other MSC or responder cell properties may be more appropriate. In all cases, the biological relevance of the proposed assay for the intended clinical activity should be substantiated with appropriate product-specific (non-)clinical data. In case practical considerations prevent the use of the ideal potency assay at release, use of a surrogate marker or test could be considered if correlation with functionality has been demonstrated. Nevertheless, as the field of MSC immunology is evolving, improvements can be expected in relevant assays and consequently in guidance related to potency testing.

    Original languageEnglish
    Pages (from-to)784-797
    Number of pages14
    JournalCytotherapy
    Volume19
    Issue number7
    DOIs
    Publication statusPublished - Jul 2017

    Keywords

    • cell-based therapy
    • government regulation
    • immunomodulation
    • in vitro potency assays
    • mesenchymal stromal cells
    • quality control

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