The safety committee of the European Medicines Agency (EMA) recently initiated a signaling procedure to review data on secondary malignancies related to T cells, including T cell lymphoma and leukemia, for the six T cell drugs with chimeric antigen receptors (CARs) approved in the European Union. In the last decade, as a result of scientific progress in the research and development of cellular and molecular biotechnology, the first ATMPs were launched on the EU market and, to date, a total of 24 ATMPs have been approved; specifically, 10 gene therapies based on T cells with chimeric antigen receptors (CAR) and 3 TEP (Osservatorio Terapie Avanzate, 202). Spain is the first country to approve CAR T-cell therapy developed entirely in Europe. Using a monoclonal antibody created at the Hospital Clínic de Barcelona more than 30 years ago, it is approved for patients over 25 who suffer from acute lymphoblastic leukemia (a type of blood and bone marrow cancer), and is pending approval for other European countries.
The first two CAR-T cell drugs recommended for approval in European Union. While potential applications of cell-based therapies include the treatment of cancer and neurodegenerative, autoimmune and infectious diseases, the reconstruction of damaged cartilage in the joints, and the repair of spinal cord injuries, most of these advanced therapies are still in an early phase of clinical development. CAR-T cell therapy is classified as cell-based gene therapy, since it involves the genetic modification of a patient's T cells to express a gene for a receptor (called a chimeric antigen receptor or CAR) specific for a tumor antigen, followed by ex vivo cell expansion and reinfusion to the patient. Within the European Medicines Agency (EMA), an Advanced Therapies Committee (CAT) was created, composed of experts in specific scientific fields related to advanced therapies.
The objective of this review is to report on current evidence on cell-based therapies authorized in the EU, including somatic cell therapies, tissue engineering products and cell-based gene therapy products in the form of T cells with chimeric antigen receptors (CARs), focusing on the evaluation of efficacy and safety in clinical trials and real environments.
