The Hematology Department team at Union Hospital, affiliated with Tongji Medical College of Huazhong University of Science and Technology (HUST), has published a groundbreaking clinical study titled "Extended follow-up of in vivo BCMA CAR-T therapy in relapsed/refractory multiple myeloma" in the renowned journal Nature Medicine.

The study presents the longest follow-up data to date regarding in vivo CAR-T therapy, setting a new global paradigm for cellular immunotherapy. The first authors are Xu Jia, Liu Lin, Xie Wei, and Su Liman, with corresponding authors Mei Heng and Hu Yu.

The research focuses on relapsed/refractory multiple myeloma and extends follow-up for in vivo BCMA CAR-T therapy to up to 15 months. Traditional ex vivo CAR-T therapy involves complex procedures, long wait times, and high costs. In contrast, in vivo CAR-T technology allows direct generation of CAR-T cells within patients, with the potential for immediate treatment.

The study included four patients, one of whom maintained stringent complete remission for 15 months, with a median progression-free survival of four months. For the three patients with relapsed progression, the overall survival following infusion ranged from 5.5 to 10.9 months. The team also developed a comprehensive in vivo CAR-T clinical application and assessment system, encompassing cellular dynamics monitoring, immunogenicity evaluation, genomic integration site analysis, and full-cycle management of clinical complications.

This research is the first to define acute inflammatory reactions associated with in vivo CAR-T delivery vectors, establish innovative antibody detection methods, confirm the polyclonal integration pattern of lentiviral vectors, and explore strategies for combining in vivo and ex vivo CAR-T therapies.

These findings address critical technological gaps for advancing in vivo CAR-T therapy toward confirmatory clinical stages and provide an evidence-based foundation for standardized implementation.

Source: Union Hospital of Tongji Medical College, HUST