The first CAR-T developed entirely in Europe has been authorized for use in patients because its safety and efficacy have been proven in clinical trials. This non-industrially advanced treatment will allow the treatment of acute lymphoblastic leukemia that is resistant to traditional therapies, for which there are no other treatment options.
Spanish Agency for Medicines and Health Products (Energy Management System) It has been approved to be used as non-industrial advanced therapeutic drugs in the following fields: CAR-T ARI-0001, Developed by Clinical hospitalFor Patients over 25 years of age with Lymphocytic leukemia Resistant to conventional treatment.
It is the first CAR-T fully developed in Europe and approved by regulatory agencies.Special authorization requirements Milestones in Spain, Because this is the first treatment method using genetically modified cells.
Cesar HernandezThe head of the human medicine department of AEMPS pointed out that “the granted license must allow patients who have no other treatment options to enter and continue to generate knowledge about the drug, so in the end, it will obtain a centralized marketing authorization throughout Europe.”
This cart (Chimeric antigen receptor T cell) is a type of cell and gene therapy in which the patient becomes his own donor.It includes modification T lymphocytes In this way they have the ability to attack tumor cells. He explained: “In our case, CART was developed based on a proprietary antibody created in the hospital 30 years ago, and we discovered its new application.” Manel Juan, The head of the immunology service of the clinic’s biomedical diagnosis center.
by in love -A technology that can separate blood components-obtained T lymphocytes, a type of white blood cell responsible for the immune response. These are genetically reprogrammed so that when they are returned to the patient, they can specifically recognize tumor cells and attack them. After three weeks, the patient’s response can be observed.
In order to enable patients to receive this treatment, Juan’s team developed a new CAR-T CD19 from their own monoclonal antibody to modify T lymphocytes. Preclinical studies have shown that CAR-T ARI-0001 can completely control the animal’s The disease progression model, its efficacy is similar to other available CAR-T.
Juan said: “The preparation method used to obtain our CAR-T is reliable and reproducible, and can reduce the production cost of the treatment method, so that academic institutions can afford it and be used by all patients.”
Treat patients without treatment
This Acute lymphocytic leukemia (ALL) is one of the four main types of leukemia. It is characterized by excessive production of lymphocytes or immature white blood cells, which rapidly multiply and crowd out normal cells in the bone marrow.
Although chemotherapy or bone marrow transplantation can achieve complete remission in most cases, 10% and 15% Of patients died of resistance to treatment, toxicity or recurrence. When treatment options are exhausted, less toxic and more targeted solutions are needed, in which immunotherapy can play a key role.
Trial reason Julio Delgado, The hematology service department of the hospital clinic, and Susana rivesThe study in the Department of Hematology at the Sant Joan de Déu Hospital in Barcelona was launched in July 2017.Research includes 58 adults and pediatric patientsAmong them, 38 patients with ALL received CAR-T treatment developed in the clinic. All patients have received multiple treatments, and the vast majority of patients have received bone marrow transplantation, which means that they have no other treatment options.
Both hospitals carried out CAR-T infusion and subsequent patient management. The patient was still sent to the inpatient room to control related toxicity. Some patients had to be transferred to the intensive care unit.
The test results show that CAR-T ARI-0001 produced More than 70% of patients have a complete remission, Which means there is no residual disease. Alvaro Urbano EspisuaThe director of the Clinical Research Institute of Hematological Tumor Diseases emphasized that this treatment is “safe and very effective, and its results are comparable to those described by other CAR-T therapies.” The therapy meets the unmet needs of ALL patients, especially for those Adult patients who are powerless with existing therapies have no other choice.
“The fact that it is produced in our own hospital means that we can prepare it in a very short period of time. Considering the vulnerability of many patients, this is a fundamental aspect. In addition, we can adjust the treatment plan according to the characteristics of the patient. On the other hand, if necessary, by putting the product in our hands, we can adjust the amount of CART used or the repeated dose,” Urbano-Ispizua concluded.
ARI project, the engine of CAR-T
This ARI project Born in the dream of AriBenedé, AriBenedé was a girl with this leukemia who died on September 2, 2016. She and her mother, ngela Jover, carried out the project based on the following two aspects: research, focusing on implanting in CAR clinics to help improve the home care of cancer and blood disease patients. It raised approximately 1.8 million euros from companies, foundations, associations and individuals.