Recently, a clinical study on “Targeted CD19 Chimeric Antigen Receptor T-Cell Therapy for Refractory Pediatric Systemic Lupus Erythematosus” has been successfully completed with the 20th patient undergoing CAR-T cell infusion.
“This is the largest global study to date on CAR-T cell therapy for refractory systemic lupus erythematosus in children, which strengthens our confidence in using this therapy for other autoimmune diseases,” said the vice president and director of the Kidney and Urology Center. According to current results, none of the 20 patients experienced infusion-related allergic reactions.
T lymphocytes, or T cells, are one of the key cell types in the human immune system. As an emerging therapeutic technology, Chimeric Antigen Receptor T-Cell (CAR-T) therapy involves collecting and modifying a patient’s T cells, then reintroducing them into the body, enabling these cells to recognize and destroy disease cells that carry specific target antigens. These modified T cells can survive and proliferate in the body, thereby achieving long-term therapeutic effects.
Currently, CAR-T cell therapy is mainly used for treating B-cell related hematologic malignancies, such as relapsed or refractory acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma. In recent years, this therapy has gradually been applied to the treatment of solid tumors and autoimmune diseases.
Clinically, CAR-T therapy faces challenges such as insufficient T cell collection, T cell functional defects, severe side effects, and high treatment costs. Some patients, due to physical conditions or other treatment needs, may find it difficult to undergo autologous T-cell collection. These patients can consider using allogeneic CAR-T therapy, which involves collecting T cells from healthy donors and preparing ready-to-use cell medications. Allogeneic CAR-T therapy is not affected by the patient’s own T cell quantity and quality, has a higher success rate in preparation, and is expected to reduce treatment costs.
The hospital is planning to develop a universal CAR-T cell therapy and expand its research to other autoimmune diseases, such as anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, steroid-resistant nephrotic syndrome, and systemic sclerosis. These studies have already passed ethical review.
In recent years, significant progress has been made in CAR-T therapy research. According to incomplete statistics, over 20 clinical studies worldwide are currently using CAR-T therapy to treat autoimmune diseases. Several CAR-T cell therapy products have been approved in China for treating adult relapsed or refractory mantle cell lymphoma, relapsed or refractory multiple myeloma, and other conditions.
In July of this year, an article published in the international journal Cell featured a study led by Professor Xu Huji from the Second Affiliated Hospital of the Naval Medical University, in collaboration with East China Normal University and Zhejiang University School of Medicine. The team used CRISPR-Cas9 gene-editing technology to modify CAR-T cells targeting CD19 from healthy donor sources, developing a new generation of allogeneic universal CAR-T therapy, which successfully alleviated symptoms in three patients with autoimmune diseases. This research highlights the significant potential of allogeneic universal CAR-T therapy in terms of both efficacy and safety.
Although CAR-T therapy has shown remarkable efficacy in some areas, it is not a universal cure. Its effectiveness is influenced by factors such as the patient’s condition, treatment plan, and individual differences. Moving forward, it is crucial to combine clinical and basic research to continuously improve treatment methods, benefiting more patients.
