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NEJM:诱导白血病缓解的新型免疫疗法

  1. 免疫治疗
  2. 白血病

来源:生物谷 2014-10-22 10:34

2014年10月22日讯 /生物谷BIOON/ --根据发表在New England Journal of Medicine杂志上的一项研究证实:一种实验性免疫系统治疗可导致晚期急性淋巴细胞白血病(ALL)患者得到完全缓解,通常晚期急性淋巴细胞白血病(A

2014年10月19日讯 /生物谷BIOON/ --根据发表在New England Journal of Medicine杂志上的一项研究证实:一种实验性免疫系统治疗可使晚期急性淋巴细胞白血病(ALL, acute lymphoblastic leukemia)患者得到完全缓解,通常晚期急性淋巴细胞白血病(ALL)患者已经没有其他治疗选择。

新疗法帮助免疫系统靶向ALL细胞的特异性蛋白质。病人的T细胞被基因工程改造后表达嵌合抗原受体,后者允许T细胞识别并摧毁ALL细胞。经修饰的T细胞注入回患者的血液中,在那里它们进行增殖。

研究人员发现,30名儿童和成人白血病患者接受经过基因改造的自体免疫系统细胞后,其中27人症状都充分缓解。经过至少六个月的随访,有的长达两年的随访。在此期间,19名患者仍处于在缓解期,而7名由于癌症复发或恶化而去世。

费城儿童医院和宾夕法尼亚大学高级研究员Stephan Grupp医学博士表示:此疗法的主要短期风险是细胞因子释放综合征。在当前的实验中,所有的病人在接受治疗后很快会发生细胞因子释放综合征,病人也会感到恶心,但GRUPP说:这是一个可控的副作用。(生物谷Bioon.com)

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Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia

Shannon L. Maude, M.D., Ph.D., Noelle Frey, M.D., Pamela A. Shaw, Ph.D., Richard Aplenc, M.D., Ph.D., David M. Barrett, M.D., Ph.D., Nancy J. Bunin, M.D., Anne Chew, Ph.D., Vanessa E. Gonzalez, M.B.A., Zhaohui Zheng, M.S., Simon F. Lacey, Ph.D., Yolanda D. Mahnke, Ph.D., Jan J. Melenhorst, Ph.D., Susan R. Rheingold, M.D., Angela Shen, M.D., David T. Teachey, M.D., Bruce L. Levine, Ph.D., Carl H. June, M.D., David L. Porter, M.D., and Stephan A. Grupp, M.D., Ph.D.

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 0.76×106 to 20.6×106 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.

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