Cell子刊：CAR-T细胞疗法又攻克一种自身免疫疾病

Core Viewpoint - The article discusses the promising application of CD19 CAR-T cell therapy in treating MDA5+ dermatomyositis with rapidly progressive interstitial lung disease (RPILD), highlighting its effectiveness and safety in a pediatric patient [2][3][7]. Group 1: Disease Overview - Dermatomyositis (DM) is an autoimmune disease primarily affecting the skin and muscles, with MDA5+ DM being a severe subtype associated with high mortality due to rapidly progressive interstitial lung disease [2]. - MDA5+ DM-RPILD is considered one of the most challenging conditions in the medical field, with no curative therapies currently available [2]. Group 2: Research Findings - The study reports the first use of second-generation CD19 CAR-T cell therapy in a child with severe MDA5+ DM-RPILD, demonstrating both efficacy and safety [5][7]. - Prior to CAR-T cell injection, conventional treatment stabilized the patient's condition, and CAR-T cell expansion indicated the presence of CD19+ B lymphocytes in deep tissues [6]. - Over 11 months, the patient showed gradual improvement in lung function, skin, motor, respiratory, and neurological functions without the use of immunosuppressants [6][7][9]. Group 3: Implications - The results suggest that CD19 CAR-T cell therapy may provide a new treatment avenue for complex autoimmune diseases that are resistant to traditional immunosuppressive therapies [2][9].