ABSTRACT
This chapter discusses European requirements for development of Advanced Therapy Medicinal Products (ATMP) based on stem cells, describing the framework for clinical trials and for marketing authorization, as well as the critical issues and challenges for developing stem cell-based ATMP. ATMP are covered by the European legal frame for medicinal products through the EU Regulation 1394/2007. Stem Cell (SC) therapy holds the promise to treat degenerative diseases, cancer and repair of damaged tissues for which there are currently no or limited therapeutic options. SC-based ATMP can be obtained from adult stem cells or pluripotent stem cells, such as mesenchymal/stromal stem cells, hematopoietic stem cells, tissue-specific progenitor cells. Cell plasticity and product differentiation might also affect results generated during development, therefore it is expected that nonclinical and clinical studies are performed with well defined and characterized product so that results can be better interpreted.
