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Spotlight Article

Regulatory viewpoints on the development of advanced stem cell–based medicinal products in light of the first EU-approved stem cell product

Navigating the Regulatory Landscape

Egbert Flory, Paolo Gasparini, Veronika Jekerle, Tiina Palomäki, Patrick Celis, Tomáš Boráň, James W McBlane, John Joseph Borg, Jan Kyselovic, Metoda Lipnik–Stangelj, Toivo Maimets, Margarida Menezes-Ferreira, Guido Pante, Stefanie Prilla, Una Riekstina, Christian K Schneider, Asterios Tsiftsoglou and Paula Salmikangas


Recent years have seen a dramatic increase in research activity and investment into therapeutic applications of stem-cell-based medicinal products. In April 2015, the first stem cell product, ex vivo autologous corneal epithelial cells including stem cells (Holoclar®, Holostem Advanced Therapies), was granted a Marketing Authorization in the European Union (EU). This product is based on limbal stem cells and is intended for the treatment of limbal stem cell deficiency caused by ocular burns. Whilst only one stem cell product has been approved, there are many others being studied for various indications. Approximately 166 clinical trials are testing the use of adult stem cells in regeneration of lost or damaged tissue and in hematological or solid-organ malignancies in the EU. Embryonic stem cells and induced pluripotent stem cells are mainly being explored in nonclinical studies, although early-stage clinical studies with these pluripotent cells have also been reported. Unfortunately, there are also reports of patients travelling outside the EU for unregulated treatments with stem cells. In order to meet patient’s demands for authorized, safe and effective treatments and to foster innovation in this dynamic area, in 2011 the Committee for Advanced Therapies of the European Medicines Agency developed and published regulatory guidance with contributions from European and international experts. It has become clear that a multidisciplinary point of view has to be applied to stem-cell-based products in order to regulate them adequately, thus spanning the bridge from quality criteria and impact of starting materials, animal models, biodistribution and niche, to safety issues including tumorigenicity, and clinical aspects. In this article, we report on the regulatory and scientific framework for stem-cell-based products in Europe and how this can be successfully applied in the development and evaluation of novel stem-cell-based products.

DOI: 10.18609/cgti.2015.010
Submitted for review: June 30, 2015
Open access
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