Safety, Efficacy, and Regulation of Mesenchymal Stromal/Stem Cells

  • Mimmi PatrikoskiEmail author
  • Kristiina Rajala
  • Susanna Miettinen


The scientific progress during the last decades has led to the development of cell therapy and tissue-engineered products that are moving rapidly into clinical application. Cranio-maxillofacial applications have paved the way for cell therapy and especially for tissue-engineered products. Cell-based medicinal products (CBMP) differ from conventional drug products, as they are often more complex and variable and less comprehensively characterizable. In addition, they often have complex mechanisms of action that remain incompletely understood. The overall risk/benefit to the proposed indication in certain patient population is evaluated with respect to the available data on quality, safety, and efficacy on a case-by-case basis. In this chapter, safety and efficacy of CBMP, especially mesenchymal stromal/stem cells (MSC)-based products, are discussed. The demonstration of quality, safety, efficacy, and comparability between production batches may be demanding for CBMP. Thus, a well-established and controlled manufacturing process is needed in order to produce a qualified product to guarantee reproducible data from nonclinical and clinical studies. In this chapter, we will also provide an overview of the regulation of CBMP in the European Union (EU) and the United States of America (US).


Mesenchymal stromal/stem cells MSC Cell-based medicinal products Advanced medicinal products Safety Efficacy Cell therapy Clinical trials Regulation 



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Copyright information

© Springer Nature Switzerland AG 2019

Authors and Affiliations

  • Mimmi Patrikoski
    • 1
    • 2
    • 3
    Email author
  • Kristiina Rajala
    • 1
    • 2
  • Susanna Miettinen
    • 1
    • 2
  1. 1.Adult Stem Cell Group, Faculty of Medicine and Health TechnologyTampere UniversityTampereFinland
  2. 2.Research, Development and Innovation CentreTampere University HospitalTampereFinland
  3. 3.Obesity Research Unit, Research Program for Clinical and Molecular Metabolism, Faculty of MedicineUniversity of HelsinkiHelsinkiFinland

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