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The therapy called CAR-T ARI-0001 is the first non-industrially manufactured advanced therapy drug to obtain approval in Europe. Now, work is being done to scale production at a continental level
CAR-T therapy (from the acronym T lymphocytes with a chimeric antigen receptor, in English) developed by the Hospital Clínic de Barcelona-Idibaps, called CAR-T ARI-0001, for the treatment of acute lymphoblastic leukemia (ALL). ) represents a double milestone for the centre, Catalan healthcare and the entire National Health System. On the one hand, it achieves potentially curative treatment for patients that, after suffering the failure of other therapies, they have no alternative; and, on the other hand, it obtained PRIME designation from the European Medicines Agency (EMA) in February 2022.
The latter means that this academic and public immunotherapy is a priority for the European agency, something so difficult to achieve that it is the first academic product to receive this designation. For this reason, the Clínic team is hopeful that the EMA will open the way to treat patients from all over Europe with this CAR-T.
A year before that, in February 2021, this CAR-T therapy received approval as a non-industrially manufactured advanced therapy medicine by the Spanish Agency for Medicines and Health Products (Aemps), thus It will go down in the annals as the first therapy of this type developed entirely in Europe to receive approval from a regulatory agency.. The approval in Spain is for use in patients over 25 years of age with ALL resistant to conventional treatments, a gap in the current indications for industrial CAR-Ts marketed in Spain.
Promote own and public manufacturing
Authorized sources from the Ministry of Health have told this newspaper that "promoting the own and public manufacture of these medicines in the academic sphere of the SNS, under conditions that guarantee standards of quality, safety and effectiveness is one of the main objectives of the Plan to address advanced therapies in the SNS".
They remember that, on February 1, 2021, the AEMPS authorized the first CAR-T of non-industrial manufacture; On May 17, its price was approved, and was included in the pharmaceutical benefit on June 1, 2021, "so that all patients could access it with guarantees of equity in the access to all Spanish citizens, regardless of where they reside".
What does the therapy consist of and how did it get the green light?
The CAR-T ARI-0001 is directed against the CD19 antigen of tumor cells. After extracting the patient's T lymphocytes through leukapheresis, they are genetically modified in the hospital's own clean room by inserting a gene that codes for the chimeric antigen receptor (CAR), which enhances its action against cancer cells. Those modified cells are then infused back into the patient. For the structure of this receiver, the CD19 recognition sequences, an antibody also created in the same hospital more than 30 years ago who found this new application. and for the genetic change, a virus is used as a vector, also produced in the Barcelona acute care center.
To assess the effectiveness of this CAR-T, a clinical trial between 2017 and 2019, directed by Julio Delgado, hematologist at the Clínic, and Susana Rives, hematologist at the Sant Joan de Déu Maternal and Child Hospital in Barcelona. The trial results showed that there was a complete response in more than 70% of patients, meaning there was no residual disease. And before, preclinical studies already demonstrated that CART ARI-0001 was capable of controlling disease progression in animal models and that its efficacy was very similar to that of other CAR-T available.
La clinical research continues, while the same team develops the CAR-T ARI-0002, which changes the target of tumor cells from CD19 to BCMA to treat patients with multiple myeloma, also resistant to other treatments. This second CAR-T, in a clinical trial led by specialist Carlos Fernández, has already shown that, after one year of follow-up, 75% of patients are still alive and without disease progression.
Source: The World
