For the first time in the world, three young patients suffering from serious autoimmune diseases were treated with Car T, genetically modified immune system cells that are usually used in the treatment of oncological diseases. The doctors of the Bambino Gesù Hospital in Rome experimented with this innovative application of gene therapy in the pediatric field and recently presented their results in Padua, as part of the work of the National Center 3 for the development of gene therapy planned from the Pnrr.
Car-T against lymphomas improves survival by Anna Lisa Bonfranceschi 23 June 2023
The little patients
The three patients involved in the trial were suffering from severe forms of autoimmune diseases, pathologies in which the immune system mistakenly attacks the tissues of the patient’s body, potentially affecting any part of the body, including vital organs such as the kidney and lungs, the joints, the skin, blood vessels and other tissues. In particular, 2 patients are young Italian girls suffering from systemic lupus erythematosus, a chronic disease that can attack the kidneys, lungs and central nervous system, which in both cases was refractory to immunosuppressive therapies. The third is a 12-year-old Ukrainian child who suffers from dermatomyositis, a rare autoimmune inflammatory disease that affects the skin and skeletal muscles.
Systemic lupus erythematosus, with the new therapy symptoms under control and less cortisone by Irma D’Aria 10 May 2023
The Car T therapy
Car T cell therapy, as we said, is usually reserved for the treatment of neoplastic pathologies. It consists of genetically modifying T lymphocytes taken from the patient, so that they attack a specific antigen present on tumor cells more effectively. In the case of acute lymphoblastic leukemias and non-Hodgkin lymphomas, the target is represented by the CD19 antigen, expressed by the cells of these tumors. However, the same antigen is also present on the B lymphocytes of the immune system. For this reason, in recent years it has been thought of experimenting with CAR Ts also in the treatment of autoimmune diseases such as lupus and dermatomyositis, in which B lymphocytes play a crucial role in the onset of the disease.
In recent scientific literature, 5 cases of adult patients with Lupus erythematosus successfully treated thanks to CAR-T cell therapy are described. In the pediatric field, however, the therapy had never been tested until now. That of the Child Jesus is therefore a first on a global level, and the results – assure the doctors involved – have so far proved to be absolutely satisfactory.
Tumors, the EMA launches a review of Car-T: “Risk of secondary neoplasms” 12 January 2024
The results
In fact, all three patients treated experienced significant and sustained benefits over time. Several months after treatment with CAR-T cells, consistently with what has been found in adult patients described in the literature, they are all in disease remission and are no longer taking immunosuppressive drugs. “These are absolutely relevant data – he underlines Fabrizio De Benedetti, head of the Immunology, Rheumatology and Infectious Diseases research area of the Bambino Gesù Children’s Hospital – All 3 patients had responded unsatisfactorily to aggressive immunosuppressive therapies, necessary due to the severity of their disease, and at the same time had developed important side effects. The results obtained with CAR-T cells encourage us to continue in the direction of a clinical trial that could include a larger number of pediatric patients suffering from various autoimmune diseases in which B lymphocytes play a fundamental role in development.”
The results of the treatment of these first patients had already been presented in Rotterdam, on the occasion of the last European congress of pediatric rheumatology, and more recently they were discussed in Padua, as part of the work of the National Center 3 for the development of gene therapy provided for by the Pnrr. “Gene therapy represents a unique challenge and opportunity for global health systems – he underlines Tiziano Onesti, president of Bambino Gesù – It allows us to offer concrete answers to patients who until recently were without hope, tackling genetic diseases and serious clinical conditions in a personalized and targeted way. Furthermore, gene therapy promises to free patients from chronic conditions, improving their quality of life and reducing the long-term costs associated with chronic disease management. This medical revolution not only offers hope and healing, therefore, but also the possibility of strengthening the sustainability of healthcare systems, freeing up resources to improve overall health and promote further medical discoveries.”