靶向CD19的嵌合抗原受体T细胞治疗白血病获得持续缓解
Chimeric antigen receptor T cells for sustained remissions in leukemia
复发或难治的急性淋巴细胞白血病（ALL，一种白细胞癌）传统治疗效果很差。研究者把患者自己的T细胞用病毒载体改造，使其表达能识别CD19的嵌合抗原受体（CAR，一种人工改造的细胞受体），再回输给30名儿童和成人患者。结果显示27人（90%）达到完全缓解，包括对另一种免疫治疗耐药以及干细胞移植后复发的患者。6个月无事件生存率为67%，总体生存率78%，部分患者缓解持续长达24个月。所有患者都出现细胞因子释放综合征（免疫细胞大量激活引起的发热和多器官损伤），27%为重度，用托珠单抗（抗白介素6受体抗体）可有效控制。这确立了CD19 CAR-T疗法治疗复发难治ALL的疗效。
为什么推荐给您：首个CD19 CAR-T治疗复发难治ALL的大样本阳性结果，开创全新细胞治疗模态。
不需要生物学背景，多打比方
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摘要Abstract
BACKGROUND: Relapsed acute lymphoblastic leukemia (ALL) is difficult to treat despite the availability of aggressive therapies. Chimeric antigen receptor-modified T cells targeting CD19 may overcome many limitations of conventional therapies and induce remission in patients with refractory disease.
METHODS: We infused autologous T cells transduced with a CD19-directed chimeric antigen receptor (CTL019) lentiviral vector in patients with relapsed or refractory ALL at doses of 0.76×10(6) to 20.6×10(6) CTL019 cells per kilogram of body weight. Patients were monitored for a response, toxic effects, and the expansion and persistence of circulating CTL019 T cells.
RESULTS: A total of 30 children and adults received CTL019. Complete remission was achieved in 27 patients (90%), including 2 patients with blinatumomab-refractory disease and 15 who had undergone stem-cell transplantation. CTL019 cells proliferated in vivo and were detectable in the blood, bone marrow, and cerebrospinal fluid of patients who had a response. Sustained remission was achieved with a 6-month event-free survival rate of 67% (95% confidence interval [CI], 51 to 88) and an overall survival rate of 78% (95% CI, 65 to 95). At 6 months, the probability that a patient would have persistence of CTL019 was 68% (95% CI, 50 to 92) and the probability that a patient would have relapse-free B-cell aplasia was 73% (95% CI, 57 to 94). All the patients had the cytokine-release syndrome. Severe cytokine-release syndrome, which developed in 27% of the patients, was associated with a higher disease burden before infusion and was effectively treated with the anti-interleukin-6 receptor antibody tocilizumab.
CONCLUSIONS: Chimeric antigen receptor-modified T-cell therapy against CD19 was effective in treating relapsed and refractory ALL. CTL019 was associated with a high remission rate, even among patients for whom stem-cell transplantation had failed, and durable remissions up to 24 months were observed. (Funded by Novartis and others; CART19 ClinicalTrials.gov numbers, NCT01626495 and NCT01029366.).