Med, 25 February, 2026, DOI：https://doi.org/10.1016/j.medj.2026.101026

Single-cell profiling of immune reset in patients with refractory generalized myasthenia gravis receiving autologous CD19/BCMA CAR-T cell therapy

Zhe Ruan, Fan Ning, Wenyan Zhang, Shuang Li, Yue Su, Yonglan Tang, Xiangqi Cao, Xiaoxi Huang, Na Song, Zhuyi Li, Hang Li, Xiao Luan, Naibo Yang, Ying Li, Friedemann Paul, Qiang Liu, Ting Chang

Abstract

Patients with refractory generalized myasthenia gravis (gMG) respond poorly to conventional immunotherapy and experience severely compromised quality of life. In this phase 1 trial, Ruan et al. investigated the safety and preliminary efficacy of CD19/BCMA dual-targeting CAR-T cells in gMG without lymphodepletion. CD19/BCMA CAR-T cells are safe in all six enrolled patients with gMG who achieved clinical improvement. Five patients with gMG reached minimal symptom expression. Longitudinal flow cytometry and single-cell profiling revealed depletion of antibody-producing cells and reconstitution of B cell lineage, along with reduced memory B and plasma cells. The safety and efficacy of CD19/BCMA CAR-T cells warrant future investigations in larger clinical trials.

Article link：https://www.sciencedirect.com/science/article/pii/S2666634026000292?via%3Dihub