Overview

• CAR-T therapy was used for the first time globally in a pediatric patient to treat dermatomyositis anti-MDA5, a rare and often fatal autoimmune disease.
• The patient, critically ill with severe pulmonary complications, received the treatment under compassionate use when no other options remained.
• CAR-T ARI-0001 therapy reprogrammed B lymphocytes, key immune cells in the disease, to reset the immune system and halt its progression.
• One year post-treatment, the patient has achieved full remission, no longer requires immunosuppressive drugs, and has resumed normal activities, including attending school.
• This pioneering intervention, conducted by a multidisciplinary team at Hospital La Paz, has been documented in the scientific journal Med.