New applications of CAR-T cell therapy are arriving, 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. In fact, the first three patients with autoimmune disease were treated at the Bambino Gesù Children’s Hospital in Rome. They are 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. It is an innovative application.
It is therefore 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, on the occasion of the last European Congress of Rheumatology pediatric.
“We have significant and sustained benefits over time – says the president of the pediatric hospital, Tiziano OnestiOnesti -. Gene therapy provides concrete answers to hopeless patients. A challenge for our systems”.
Autoimmune diseases are pathologies characterized by an aggression of the immune system which, instead of defending the body from pathogens such as bacteria and viruses, attacks and destroys an individual’s own healthy tissues, mistaking them for foreign and dangerous ones. The three patients were affected in particular by very serious 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 skeletal muscles.
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