[CAR-T cells gene therapy medicines: Regulatory status and pharmaceutical circuits in Europe and France]

Romain de Jorna; Isabelle Madelaine; Jérôme Larghero; Miryam Mebarki

doi:10.1016/j.bulcan.2021.05.004

[CAR-T cells gene therapy medicines: Regulatory status and pharmaceutical circuits in Europe and France]

Bull Cancer. 2021 Oct;108(10S):S162-S167. doi: 10.1016/j.bulcan.2021.05.004.

[Article in French]

Authors

Romain de Jorna¹, Isabelle Madelaine¹, Jérôme Larghero², Miryam Mebarki³

Affiliations

¹ Université de Paris, AP-HP, hôpital Saint-Louis, pharmacie à Usage Intérieur, 1, avenue Claude Vellefaux, 75010 Paris, France.
² Université de Paris, AP-HP, hôpital Saint-Louis, unité de Thérapie Cellulaire, porte 15A, 1, avenue Claude Vellefaux, 75010 Paris, France; Université de Paris, AP-HP, hôpital Saint-Louis, centre MEARY de thérapie cellulaire et génique, 1, avenue Claude Vellefaux, 75010 Paris, France; INSERM, centre d'investigation clinique de biothérapies CBT501 et U976, 1, avenue Claude Vellefaux, 75010 Paris, France.
³ Université de Paris, AP-HP, hôpital Saint-Louis, unité de Thérapie Cellulaire, porte 15A, 1, avenue Claude Vellefaux, 75010 Paris, France; INSERM, centre d'investigation clinique de biothérapies CBT501 et U976, 1, avenue Claude Vellefaux, 75010 Paris, France. Electronic address: [email protected].

PMID: 34920799
DOI: 10.1016/j.bulcan.2021.05.004

Abstract

CAR-T cells belong to a new class of biological medicines, referred to as Advanced Therapy Medicinal Products (ATMPs). Despite the cellular component, according to the regulatory definition, CAR-T cells are gene therapy medicines, a sub-category of ATMPs, since their therapeutic effect is the result of their genetic modification. The specificity and the complexity of these innovative drugs have required a complete reorganization of the hospital and pharmaceutical circuits, from the cell collection to the drug administration to the patient. Indeed, increased interaction and collaboration between different healthcare professionals is essential in order to guarantee the quality and safety of these innovative medicines.

Keywords: Advanced Therapy Medicinal Products; CAR-T cells; Circuit pharmaceutique; Gene therapy; Médicaments de thérapie innovante; Pharmaceutical circuit; Thérapie génique.

Publication types

Review

MeSH terms

Cell Engineering / legislation & jurisprudence*
Drug Compounding / standards
Drug Industry / legislation & jurisprudence
Drug Industry / standards
Europe
France
Genetic Therapy / legislation & jurisprudence*
Genetic Therapy / methods
Humans
Immunotherapy, Adoptive / legislation & jurisprudence*
Immunotherapy, Adoptive / methods
Receptors, Chimeric Antigen*
T-Lymphocytes* / immunology
T-Lymphocytes* / transplantation

Substances

Receptors, Chimeric Antigen