Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders:phase 1 trial interim results  

作　　者：Chuan Qin Dai-Shi Tian Luo-Qi Zhou Ke Shang Liang Huang Ming-Hao Dong Yun-Fan You Jun Xiao Ying Xiong Wen Wang Hao Pang Jing-Jing Guo Song-Bai Cai Di Wang Chun-Rui Li Min Zhang Bi-Tao Bu Wei Wang 

机构地区：[1]Department of Neurology,Tongji Hospital,Tongji Medical College,Huazhong University of Science and Technology,430030 Wuhan,China [2]Hubei Key Laboratory of Neural Injury and Functional Reconstruction,Huazhong University of Science and Technology,430030 Wuhan,China [3]Department of Hematology,Tongji Hospital,Tongji Medical College,Huazhong University of Science and Technology,430030 Wuhan,China [4]Department of Radiology,Tongji Hospital,Tongji Medical College,Huazhong University of Science and Technology,430030 Wuhan,China [5]Nanjing IASO Biotherapeutics Ltd,Nanjing,China

出　　处：《Signal Transduction and Targeted Therapy》2023年第2期683-691,共9页信号转导与靶向治疗（英文）

摘　　要：Chimeric antigen receptor(CAR)T-cell therapy that targets B-cell maturation antigen(BCMA)have great potentials in autoimmune diseases and could be novel therapeutics for relapsed/refractory neuromyelitis optica spectrum disorder(NMOSD).To evaluate the safety and efficacy of the CT103A,a self-developed BCMA-targeting CAR construct against BCMA,in patients with AQP4-IgG seropositive NMOSD,an ongoing,investigator-initiated,open-label,single-arm,phase 1 clinical trial is conducted at our center.In total,12 patients were administered with a CAR-BCMA infusion.Ten of the 12 patients dosed were women(83.3%),with a median age of 49.5 years(range,30-67).were The most common events of grade 3 or higher were hematologic toxic effects.Seven patients(58%)developed infections,but no grade 4 infections occurred.Cytokine release syndrome was reported in all patients with only events of grade 1 or 2 observed.During the follow-up of a median 5.5 months,11 patients had no relapse;all patients generally reported improvement in disabilities and quality-of-life outcomes;11 patients’AQP-4 antibodies in serum showed a downward trend by the cutoff date.CAR T-cell expansion was associated with responses,and persisted more than 6 months post-infusion in 17%of the patients.In summary,CAR T-cell therapy shows a manageable safety profile and therapeutic potentials for patients with relapsed/refractory AQP4-IgG seropositive NMOSD.Another expansion phase is currently underway to determine the safety and efficacy of CAR T-BCMA infusion in patients with other neuro-inflammatory diseases.

关 键 词：AQP4 BCMA INFUSION 

分 类 号：R744.3[医药卫生—神经病学与精神病学]