Drug Regulators, EMA (EMEA), Publishes a Reflection Paper on Stem Cell-Based Medicinal Products
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Stem cells hold the promise for a limitless source of cells for therapeutic applications in various conditions, including metabolic, degenerative and inflammatory diseases, cancer and for repair and regeneration of damaged or lost tissue. Various stem cell types can be isolated from different tissues of the human body, expanded and/or differentiated in in vitro culture conditions, and subsequently administered to patients. Existing guidance on cell-based medicinal products (Guideline on human cell-based medicinal products (EMEA/CHMP/410869/2006)) covers the general aspects of all cell-based products including stem cell advanced therapy medicinal products. In addition, in case of genetic modification of stem cells, the future guideline for genetically modified cells should be consulted (see Draft guideline on the quality, preclinical and clinical aspects of medicinal products containing genetically modified cells (EMEA/CHMP/GTWP/671639/2010)).
The aim of this reflection paper is to cover specific aspects related to stem cells based medicinal products as defined below.
This reflection paper shall apply to all types of stem cells regardless of their differentiation status at the time of administration. Stem cells that are not substantially manipulated and intended to be used for the same essential function in the recipient as in the donor as referred to in Article. 2 (1 (c)) of Regulation EC (No) 1394/2007 are out of the scope of this reflection paper. For a list of manipulations that are not considered substantially manipulated see Annex I of Regulation EC (No) 1394/2007.
Although the stem cells share the same principal characteristics of self-renewing potential and differentiation, stem-cell-based medicinal products do not constitute a homogeneous class. Instead, they represent a spectrum of different cell-based products for which there is a variable degree of scientific knowledge and clinical experience available. For example, while HSCs have been used for therapeutic purposes, this is not the case for human embryonic stem cells or induced pluripotent cells.
In addition, varying levels of risks are associated with specific types of stem cells. A risk-based approach according to Annex I, part IV of Dir 2001/83/EC is recommended for stem cell containing products.
This reflection paper is relevant to all medicinal products using stem cells as starting material. The final products may constitute of terminally differentiated cells derived from stem-cells, from pluripotent stem cells or even from a mixture of cells with varying differentiation profile.