A clinical trial initiated at the Sant Joan de Déu hospital seeks to extend to pediatric patients an immunotherapy treatment developed by the Clínic against type B lymphoblastic leukemia, which was authorized two years ago for patients over 25 years of age.
A 7-year-old girl is the first to have received this therapy within the framework of a trial that will last two years, in which thirty Spanish patients will participate. The study is led by Susana Rives, head of the Leukemia and Lymphoma unit at the Pediatric Cancer Center in Barcelona.
ARI-0001 is the first European CAR-T for acute infoblastic leukemia. It received the approval of the Spanish Medicines Agency in February 2021. Although the results of a first clinical trial carried out jointly at the Clínic and in Sant Joan de Déu were positive for both adults and children, it was authorized only for the first group. The reason is that a CAR-T from the pharmaceutical industry for pediatric patients had already been authorized in 2018.
The trial aims to test whether the CAR-T ARI can replace other treatments associated with more risks and side effects, such as bone marrow transplantation, in children in less advanced stages of the disease, in the first relapse.
In this sense, the CAR-T included since 2018 in the portfolio of services of the Ministry of Health (Kymriah, from Novartis) is indicated for the treatment of the disease in second relapse.
CAR-T is a precision therapy that trains the immune system so that it can identify, attack, and destroy cancer cells in a targeted manner. The patient undergoes a blood extraction, from which the T lymphocytes, a type of immune system cells, are separated, which are modified in the laboratory using genetic engineering techniques.
The modified immune cells express on their surface a receptor capable of recognizing the tumor antigen expressed by type B leukemias and lymphomas, in order to be able to specifically destroy cancer cells.
On the other hand, the Sant Joan de Déu hospital is finalizing the installation in the Pediatric Cancer Center of four clean rooms to develop and produce advanced therapies. Thus, the center will be able to start the production of CAR-T cell therapy and the investigation of new treatments for diseases that do not have an effective alternative, such as primary immunodeficiencies in pediatric patients.
The PCCB has announced today the imminent start of an investigation to study the efficacy of CAR-T combined with a vaccine created from the patient's drendritic cells (which stimulate the immune response), for the treatment of diffuse glioma of the brain stem, a tumor brain that has no cure.
The vaccine developed by the center's researchers between 2016 and 2018 managed to stimulate the immune system's response, but "did not translate into a significant increase in patient survival," explained the PCCB's care director, Andrés Morales. "We believe, however, that CAR-T can intensify the response and, therefore, we want to study the tolerance and the efficacy of a combination therapy."
