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

Abstract

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).