单位：[1]Department of Neurology,Tongji Hospital,Tongji Medical College,Huazhong University of Science and Technology,430030 Wuhan,China神经内科神经科华中科技大学同济医学院附属同济医院[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内科学系血液内科华中科技大学同济医学院附属同济医院[4]Department of Radiology,Tongji Hospital,Tongji Medical College,Huazhong University of Science and Technology,430030 Wuhan,China放射科华中科技大学同济医学院附属同济医院[5]Nanjing IASO Biotherapeutics Ltd, Nanjing, China
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.
基金：
This study
was funded by Nanjing IASO Therapeutics Ltd.
第一作者单位：[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
共同第一作者：
通讯作者：
通讯机构：[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
推荐引用方式(GB/T 7714)：
Qin Chuan,Tian Dai-Shi,Zhou Luo-Qi,et al.Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders: phase 1 trial interim results[J].SIGNAL TRANSDUCTION AND TARGETED THERAPY.2023,8(1):doi:10.1038/s41392-022-01278-3.
APA：
Qin,Chuan,Tian,Dai-Shi,Zhou,Luo-Qi,Shang,Ke,Huang,Liang...&Wang,Wei.(2023).Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders: phase 1 trial interim results.SIGNAL TRANSDUCTION AND TARGETED THERAPY,8,(1)
MLA：
Qin,Chuan,et al."Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders: phase 1 trial interim results".SIGNAL TRANSDUCTION AND TARGETED THERAPY 8..1(2023)
医学