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European Biopharmaceutical Review

Revamping Regulations

Stem cell products hold great promise for multiple clinical indications and are actively studied in clinical trials (1). However, many related safety features raise concerns and currently generate extensive and costly testing of the final product, if intended for clinical use. The regulatory expectations for stem cell testing for clinical studies, as well as marketing authorisation applications, are not entirely clear, up-to-date or detailed. Furthermore, differences between US and EU guidance, in this respect, hamper the current development of products for EU markets.

Risk Profiling

Stem cells used for therapeutic purposes include a wide variety of cells with different type differentiation capacities, spanning from restricted multipotent up to pluripotent stem cells (2). Hematopoietic stem cells have been widely used for ex vivo gene therapy of monogenic inherited diseases, and mesenchymal stem cells have been utilised eg in treatment of Graft versus Host Disease for more than a decade due to their immunomodulatory properties (3,4). Pluripotent stem cells, ie embryonic stem cells and induced pluripotent stem cells (iPSCs), are still in early clinical development, yet some clinical data are already available (5,6).

When considering possible risks of stem cells, several issues need to be considered, such as the donor (autologous versus allogeneic), characteristics of the cells, extent of manipulation, design of vectors for genetic modification, dose and the existing knowledge of the cell type/product to be used. One of the main concerns related to stem cell products is tumourigenicity. For pluripotent stem cells, teratoma formation is part of their natural characteristics, which calls for thorough control of the cell differentiation status before clinical use.

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Dr Paula Salmikangas, Director for Biopharmaceuticals and ATMPs, NDA Advisory Board, is a clinical biochemist by original training with a PhD in muscle cell biology. Since 2006, she has been an Adjunct Professor of Biochemistry for the University of Helsinki, Finland. Paula joined NDA in 2017, has served as a member of the EMA Committee for Advanced Therapies (CAT) from 2009 to 2017 and has been the Chair of the CAT 2014-2017. Her main areas of expertise are biological medicinal products, especially ATMPs, chemistry, manufacturing and controls aspects of biopharmaceuticals.
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Dr Paula Salmikangas
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