At Bambino Gesù in Rome, new applications of CAR-T cell therapy, which involves the laboratory manipulation of the patient's T lymphocytes to make them capable of recognizing and attacking tumor cells through the introduction of a particular DNA sequence. The first 3 patients with autoimmune disease were treated at the pediatric hospital: 2 Italian girls and a 12-year-old Ukrainian boy who fled the war. They are the first pediatric patients with serious autoimmune diseases treated with Car-T capable of putting their disease into remission, announced by Bambino Gesù. This is an innovative application of gene therapy based on the manipulation of the patient's T lymphocytes, tested for the first time in the pediatric field on this type of pathology. The results of the treatment, performed on Bambino Gesù, were recently presented in Padua as part of the work of the National Center 3 for the development of gene therapy foreseen by the Pnrr, and again in Rotterdam, the Netherlands, on the occasion of the last European Congress of pediatric rheumatology.
Autoimmune diseases – recalls a note from the Capitoline IRCCS – are pathologies characterized by an aggression of the immune system which, instead of defending the organism from pathogenic agents such as bacteria and viruses, attacks and destroys an individual's own healthy tissues, exchanging them for strangers and dangerous people. This malfunction can cause an inflammatory process and the formation of antibodies that mistakenly attack healthy cells, potentially affecting any part of the body, including vital organs such as the kidney and lungs, joints, skin, blood vessels and other tissues. The 3 patients treated with Car-T cells were affected in particular by very severe forms of systemic lupus erythematosus, a chronic disease that can attack the kidneys, lungs and central nervous system, and dermatomyositis, a rare autoimmune inflammatory disease that affects the skin and muscles skeletal.
Recent scientific literature describes 5 cases of adult patients with lupus erythematosus successfully treated thanks to Car-T cell therapy, most commonly used in the context of neoplastic diseases, such as leukemia, lymphoma and myeloma. From this precedent was born the idea of the Bambino Gesù researchers to test the same solution for the first time also in the pediatric field, using the 'construct' that had worked with adults suffering from lupus, i.e. the gene therapy product developed in this case from the biotech company Miltenyi. Hence the request to AIFA for non-repetitive use (hospital exemption) of the Car-T treatment for 3 patients with particularly serious forms of autoimmune disease that are refractory to conventional treatments.
Car-T therapy – continues the note – involves the laboratory manipulation of the patient's T lymphocytes to make them capable of recognizing diseased cells, through the introduction of a DNA sequence that codes for a protein called chimeric receptor (Car, Chimeric antigen receptor). In acute lymphoblastic leukemias and non-Hodgkin lymphomas this protein recognizes a target represented by the CD19 antigen, expressed by tumor cells, which are thus recognized and attacked. The same CD19 antigen is also expressed by the B lymphocytes of the immune system, which in the case of lupus erythematosus and dermatomyositis play a crucial role in determining the disease. “By using the same target – explains Franco Locatelli, head of the area of Oncohematology and Cellular and Gene Therapy of the Bambino Gesù and full professor of Paediatrics at the Catholic University of Rome – we translate the same gene therapy approach from a context of neoplastic disease ( leukemias and lymphomas) to a context of non-neoplastic pathology, but where the elements that produce the damage are the B-lymphocytes that express CD19”.
All 3 treated patients found “relevant and sustained benefits over time. Several months after treatment with Car-T cells, consistently with what was found in adult patients described in the literature, they are in disease remission and are no longer taking drugs immunosuppressants”, they report from the IRCCS.
The first patient, a 17-year-old girl from Messina suffering from lupus – the note details – is now almost 9 months after the infusion of Car-T cells. The second patient, a 12-year-old Ukrainian suffering from dermatomyositis, is 7 months after treatment. He was followed in the Ukrainian capital before the war, then transferred to Hungary and finally to Italy, to Bambino Gesù, where he was able to benefit from Car-T therapy. The third patient, an 18-year-old Roman girl also suffering from lupus (a pathology much more frequent in females than males), is approximately 2 months after treatment. She had been hospitalized for 6 months in a row, dependent on oxygen, assisted in intensive care several times, with significant side effects due to cortisone therapies. She is at home today in good general health.
“These are absolutely relevant data”, states Fabrizio De Benedetti, head of the research area of Immunology, Rheumatology and Infectious Diseases of the Bambino Gesù. “All 3 patients had responded unsatisfactorily to aggressive immunosuppressive therapies, necessary due to the severity of the their disease, and at the same time they had developed important side effects. The results obtained with Car-T cells – the specialist points out – encourage us to continue in the direction of a clinical trial that can include a larger number of pediatric patients suffering from various diseases autoimmune in which a fundamental role in development is played by B lymphocytes”.
“Gene therapy – declares the president of the Bambino Gesù hospital, Tiziano Onesti – represents a challenge and a unique opportunity for global healthcare systems. 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 the management of chronic diseases. This medical revolution It not only offers hope and healing, therefore, but also the possibility of strengthening the sustainability of health systems, freeing up resources to improve overall health and promote further medical discoveries.”
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