Somatic Cell Therapy—Navigating the Regulatory Landscape


The diversified sources of the somatic cells call for additional oversight to prevent the introduction, transmission, and spread of communicable disease.

One of the most promising research fields in medicine is the somatic cell therapy area and their use in regenerative medicine. Advancement in understanding the pathways involved in tissue damage and repair and the biology of adult stem cells offers promise to restore functioning through cell replacement in those diseases and conditions where defects are largely cell autonomous and entail the loss or dysfunction of a single class of cells, such as cancer, Parkinson´s disease, and diabetes, among many others.

However, the regenerative potential of somatic cells awakes desires for rejuvenating cures, too. Some actors seized on this promise making hallow claims to patients while offering often unproven treatment—knowingly or unknowingly. The field of regenerative medicine, because of the very nature of its biology and the rapid advancement of clinical development, often swings around the line separating individualized cell therapies from those therapies which are medicinal products subject to regulatory oversight.

In the past years, authorities refined their regulatory framework for somatic cell therapies to ensure the safe collection, manufacture and use of human cells linking the degree of risk of the therapy to the appropriate regulatory and clinical development plan.

To determine the required regulatory oversight, regulators classify somatic cell therapy products based on their level of manipulation—substantial vs. minimal; their intended use—homologous vs. non-homologous, and their source—autologous vs. allogeneic. Specifically, somatic cells used in human application that have been subject to substantial manipulation, and/or cells that are used for an essential function or different functions as in the donor, are regulated as medicinal products in the investigated jurisdictions (Table 1). Substantial manipulation is defined as any manipulation other than cutting, grinding, shaping, centrifugation, soaking in antibiotic or antimicrobial solutions, sterilization, irradiation, cell separation, concentration or purification, filtering, lyophilization, freezing, cryopreservation and vitrification. Except for South Korea, minimal manipulated autologous cells intended for homologous use are not regulated as medicinal products. In addition, the US legislation accepts also allogeneic, homologous use in a first-degree or second-degree blood relatives for qualifying as non-medicinal product.

The diversified sources of the somatic cells call for additional oversight to prevent the introduction, transmission and spread of communicable disease. Applicable also for the category of somatic cell therapy medicinal products, all cells of allogenic origin must be tested for infectious agents before administration to the patient. Such testing is recommended for cells of autologous use.

The European Union authorizes the use of custom-made somatic cell therapies prepared on a non-routine basis in the absence of a centralized marketing authorization, provided that the product is used for individual patients in a hospital and under the professional responsibility of a medical practitioner. But also, this ‘hospital exemption’ requires respecting the requirements on quality and safety of those applied to medicinal products. Similar regulations are established in the US and Australia.

In developing the tiered, risk-based approach the regulators enable an efficient and least burdensome framework that does not become a barrier for development of new therapies and best treatment for patients.

Karoline Hahn is a Senior Consultant for KCR

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