A major breakthrough is offering new hope for children suffering from severe autoimmune diseases. Eight pediatric patients with aggressive conditions that had failed to respond to conventional treatments were able to completely discontinue immunosuppressive therapy following treatment with CAR-T cells. More than two years later, seven are in complete remission, showing no signs of disease, while the eighth patient has experienced significant clinical improvement.
The final results of the study, with follow-up exceeding 24 months, were published in Nature Medicine. The research was coordinated by the Bambino Gesù Children’s Hospital in Rome in collaboration with the University of Erlangen, marking a milestone in pediatric medicine.
Autoimmune diseases arise when the immune system malfunctions and attacks the body’s own tissues instead of protecting it from pathogens. This abnormal immune response leads to chronic inflammation that can affect virtually any part of the body, including vital organs such as the kidneys and lungs, as well as the skin, joints and blood vessels.
The patients - seven girls and one boy aged between five and 17 - were affected by particularly severe forms of early-onset autoimmune disease. Four had systemic lupus erythematosus, three suffered from dermatomyositis, and one from juvenile systemic sclerosis. Five of the children were treated with CAR-T cells at the Bambino Gesù Hospital.
All had complex medical histories marked by only partial or temporary responses to multiple immunosuppressive therapies, including biologic drugs targeting B lymphocytes, and several had experienced life-threatening organ involvement.
Originally developed for oncology, CAR-T therapy appears to have “reset” the immune system, enabling long-lasting disease control without ongoing treatment. The findings represent a potential paradigm shift in the management of rare and severe pediatric autoimmune disorders.