Ari Benedé was 13 years old when she was diagnosed with acute lymphoblastic leukemia (ALL), one of the four main types of leukemia. It was detected after she returned from a trip to England, when she noticed she wasn't feeling entirely well. They started treating her with chemotherapy for a year and a half. Years later, she had a relapse and was admitted again, undergoing more invasive treatments. Her family began searching for possible treatments for the disease worldwide and found that CAR-T existed in the United States, a cell and gene therapy that modifies lymphocytes to make them capable of destroying cancer cells.
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This is how the ARI project emerged, which aimed to raise the necessary funds to bring this treatment to Barcelona, as it was too expensive. Numerous personalities, such as Leo Messi, Judit Mascó, Mercedes Milà, and Joan Manuel Serrat, supported her, and Ari, along with two friends, organized a Love of Lesbian concert at Luz de Gas, which sold out all tickets. In total, the ARI project managed to raise nearly 1.8 million euros.
Ari died in September 2016 at the age of 18, but her efforts have allowed the Clínic to develop CAR-T ARI-0001. The Spanish Agency of Medicines and Medical Devices (AEMPS) has just approved its use as a medicine for adult patients with this type of leukemia for whom conventional treatments no longer work. In this way, it becomes the first CAR-T developed entirely in Europe to be approved by a regulatory agency and, moreover, is public, a fact that will allow it to have a more affordable cost for the healthcare system. With the approval, Hospital Clínic expects to be able to treat 25 patients a year and has launched a new clinical trial to extend the use of this therapy to other hospitals.
"With the ARI project, everything began. Not only the necessary economic resources, but also the motivation, the enthusiasm, the teamwork. We will always be indebted to Ari," stated the director of the Institut Clínic de Malalties Hemato-Oncològiques, Dr. Álvaro Urbano-Ispizua, at the presentation of the authorization obtained by the AEMPS, which was also attended by Ari's mother, Ángela Jove, who has continued to promote the project launched by her daughter. "Today is a tribute to Ángela, but above all to Ariana, who truly deserves it and deserved it. Today would be a very special day for her," added the general director of Clínic, Dr. Josep M. Campistol.
What is CAR-T?
In this type of cell and gene therapy, the patient becomes their own donor. It is based on modifying the patient's T lymphocytes so that they have the ability to attack tumor cells. In the case of CAR-T ARI-0001, Hospital Clínic has developed it from its own antibody created in the hospital more than 30 years ago, as explained by the head of the Immunology service at the Centre de Diagnòstic Mèdic (CDB) of Clínic and head of the joint platforms of Hospital Sant Joan de Déu and Banc de Sangs i Teixits, Dr. Manel Juan. Through apheresis, a technique that allows the separation of blood components, T lymphocytes are obtained, a type of white blood cell responsible for the immune response. T lymphocytes are genetically reprogrammed so that they can recognize tumor cells and attack them, once they have been transfused back into the patient.
The clinical trial to evaluate this CAR-T was launched in July 2017. Led by Dr. Julio Delgado, from the Hematology Service of Hospital Clínic, and Dr. Susana Rives, from the Hematology Service of Hospital Sant Joan de Déu de Barcelona, the study included 58 patients, adults and children, of whom 38 had acute lymphoblastic leukemia (ALL) and were treated with the CAR-T developed in the Barcelona hospital. All patients had received several treatments and the vast majority had undergone bone marrow transplants and, therefore, had no more therapeutic alternatives left.
The CAR-T ARI-0001 has been tested in a clinical trial with 58 patients, of whom 47% have been free of the diseaseThe results of the trial indicate that 69% of participating patients, who had a life expectancy of weeks, are still alive one year later and 47% have become disease-free"Our CAR-T is safe and very effective, with results comparable to those described by other CAR-T therapies," explains Dr. Urbano-Ispizua. "This CAR-T covers an unmet need for the treatment of patients with ALL, specifically for those adult patients refractory to available treatments, for whom there was no other option," he emphasizes.
Likewise, Dr. Josep M. Campistol has highlighted the importance of this medical advance, which he has described as an "important milestone." "It is a relevant moment for our country, research, and public healthcare," defended Campistol, who stressed that, having been fully developed by research centers and not by private companies, it ensures that many patients will be able to access the treatment.