A research team led by Professors Wang Wei, Tian Daishi, and Qin Chuan at Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology (HUST), published groundbreaking results in the top-tier journal Cell on Oct 15. For the first time globally, the researchers demonstrated that a CAR-T therapy targeting B cell maturation antigen (BCMA) is both safe and effective in treating progressive multiple sclerosis (MS).

Progressive MS is a leading cause of non-traumatic disability among young adults worldwide. This inflammatory demyelinating disease causes the immune system to attack the central nervous system (CNS), damaging myelin and disrupting nerve signal transmission.

Patients often experience motor deficits, vision problems, sensory disturbances, and cognitive decline. Until now, progressive MS has remained largely untreatable, as conventional drugs cannot penetrate central nervous system lesions where compartmentalized neuroinflammation drives neurodegeneration.

The study challenges traditional treatment paradigms and reveals a new regulatory mechanism within the CNS immune microenvironment. The team found that CAR-T therapy is safe and effective, with patients showing no immune effector cell-related neurotoxicity or serious infections, and significantly improves neurological function.

The CAR-T cells efficiently migrate to CNS compartments and expand within the cerebrospinal fluid, eliminating disease-causing B cell lineages across the CNS and halting pathological intrathecal immunoglobulin production. By removing these pathogenic B cells, chronic neuroinflammation, driven by interactions between B cells and microglia-like cells, is markedly reduced, resulting in lower levels of inflammatory cytokines in the cerebrospinal fluid.

The HUST team has long focused on understanding the mechanisms of major neurological diseases and developing innovative treatments. Their work has been published in top international journals, including The Lancet and The BMJ. The team was the first globally to apply BCMA-targeted CAR-T therapy to treat refractory neuroimmune disorders, achieving long-term, drug-free remission in 83 percent of patients over two years.

This latest breakthrough extends CAR-T therapy to progressive MS, expanding its application in neuroimmune diseases and laying the foundation for personalized treatment strategies.

Professor Wang Wei emphasized that this study introduces the first BCMA-targeted CAR-T treatment for progressive MS and uses single-cell multiomics to uncover the mechanisms of central immune microenvironment remodeling, opening new avenues for immunotherapy in chronic neuroinflammatory disorders.

Source: Tongji Hospital, Tongji Medical College of HUST