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30 CAR-T: from our cancer experience to other options
  1. Manel Juan
  1. Hospital Clínic de Barcelona, Catalonia, Spain


Cellular immunotherapy (particularly chimeric antigen receptor (CAR) T-cell therapy) has become a consolidated therapeutic reality in hematologic neoplastic diseases, but it is mainly seen as having a promising future. Considered advanced therapy medicinal products, their development has so far originated from proposals made by academic centers and later gaining market authorization by large pharmaceutical companies, which are defining the distribution and implementation of these products in patients from whom the T cells are obtained and subsequently incorporated with the chimeric antigen receptor. This unidirectional model of academic development and industrial implementation has clear advantages but also evident drawbacks.

At the Hospital Clíic of Barcelona, we have developed a second-generation CAR-T (with 4-1BB as a co-stimulatory motif) targeting CD19 (ARI0001 or varnimcabtagene autoleucel), which is fully publicly funded and has received authorization for use under the hospital exemption regulation. It is the first academic and European CAR-T approved worldwide for acute lymphoblastic leukemia B and is currently undergoing evaluation under the Priority Medicines (PRIME) designation by the European Medicines Agency (EMA), trying to obtain centralized marketing authorization. Alongside ARI0001, we are also close to obtaining ‘hospital exemption’ for ARI0002h (cesnicabtagene autoleucel), an anti-BCMA CAR-T for multiple myeloma. Obviously, all these academic products meet the same quality requirements as commercial products and must also demonstrate effectiveness within the ranges of commercial products in clinical trials for approval. Possibly, with these Barcelona products, we are paving the way to enable academic production to provide treatment options in cases where pharmaceutical companies are not interested in developing these products (such as rare diseases like pediatric tumors or even autoimmune diseases to low frequent autoantigens). Beyond cancer, other immune-mediated diseases (such as autoimmune disorders and systemic lupus erythematosus) are probably the next challenges for CAR-T immunotherapies.

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