Preclinical safety and toxicology testing of cell-based medicinal products

Today’s incurable diseases such as diabetes, congestive heart failure or Parkinson’s disease in the future may be overcome by cell therapy, aimed at restoring the body’s proper function. Introduction of foreign cells into a patient’s body, however, may lead to rejection and other unexpected outcomes. The use of patient-derived cells produced from so-called adult induced pluripotent stem cells could be a way out.

© Fraunhofer ITEM
H&E staining of a humanized mouse liver, showing subtle cellular infiltrates.

The safety and toxicology of such cell-based medicinal products (CBMPs) cannot be assessed relying on the standard repertoire used for classical pharmaceuticals. Such assessment, however, is pivotal for regulatory acceptance and to get approval for initiation of the clinical phase of development. In order to fill this gap and to support the translational development of CBMPs to clinical use, we are aiming to evolve as a competent partner for non-clinical safety and toxicology research on such Advanced Therapy Medicinal Products (ATMPs).

Fraunhofer ITEM is specialized in the development of product-specific testing strategies for preclinical evaluation of biologics and non-biologics and already has a track record in pharmacology research on cell-based therapies. Thus, we are excellently positioned to translate such emerging technologies into clinical application.


Before bringing a new cell-based therapy from basic research to trials in patients, it must first be tested for safety and toxicology. For this class of products, the transition to clinic is a particular challenge, since they represent novel approaches that have never been tried in humans.

With standard protocols applied to classical pharmaceuticals, the safety and toxicology of ATMPs such as cell therapies cannot be assessed. Fraunhofer ITEM with its background in product-specific strategies for preclinical testing and toxicology is uniquely positioned to establish non-standard test systems and subsequently transfer them from the research lab to the institute’s GLP environment.

Requirements for the registration of human CBMPs for the European market

Immunohistochemical detection of hematopoietic stem cells and induced pluripotent stem cells