细胞疗法:治愈白血病的新希望

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1、Cell Therapy Shows Promise for Acute Type of Leukemia细胞疗法:治愈白血病的新希望细胞疗法:治愈白血病的新希望A treatment that genetically alters a patients own immune cells to fight cancer has, for the first time, produced remissions in adults with an acute leukemia that is usually lethal, researchers are reporting. In one pat

2、ient who was severely ill, all traces of leukemia vanished in eight days. 研究人员发布报告称,一种抗癌疗法首次缓解了急性白血病(通常是致命的)患者的病情, 该疗法从基因上改变患者自身的免疫细胞,从而达到了抗击癌症的效果。一名重症患者在 接受治疗后,其白血病症状在八天内完全消失。 “We had hoped, but couldnt have predicted that the response would be so profound and rapid,” said Dr. Renier J. Brentjens,

3、the first author of a new study of the therapy and a specialist in leukemia at Memorial Sloan-Kettering Cancer Center. “我们曾希望过,但从未料到患者对该疗法的反应会如此强烈和快速, ”该疗法最新研究报告 的第一作者、纪念斯隆-凯特琳癌症中心(Memorial Sloan-Kettering Cancer Center)的白血病 专家雷尼尔J布伦蒂安斯(Renier J. Brentjens)说。 The treatment is experimental, has been

4、used in only a small number of patients and did not work in all of them. But experts consider it a highly promising approach for a variety of malignancies, including other blood cancers and tumors in organs like the prostate gland. The new study, in five adults with acute leukemia, was published on

5、Wednesday in the journal Science Translational Medicine. The treatment is similar to one that pulled a 7-year-old girl, Emma Whitehead, from deaths door into remission nearly a year ago, and that has had astounding success in several adults with chronic leukemia in whom chemotherapy had failed. The

6、treatment regimen that saved Emma and those adults was developed at the University of Pennsylvania. Related studies have also been done at the National Cancer Institute. 该疗法是实验性的,只被用于少数患者,而且不是对所有人都有效。但专家认为,该疗法 对一些不同类型的恶性肿瘤来说是非常有前景的,包括其他类型的血癌以及器官肿瘤,如 前列腺癌等。新研究的治疗对象是五名急性白血病成人患者,研究报告于周三发表在科 学转化医学(Scien

7、ce Translational Medicine)上。该疗法和另一种疗法有相似之处,后者在 近一年前缓解了7岁女孩埃玛怀特黑德(Emma Whitehead)的病情,把她从死亡线上拉了回 来,而且该疗法在数名患有慢性白血病而化疗又不起作用的成人患者身上也获得了巨大成 功,它的研发机构是宾夕法尼亚大学(University of Pennsylvania)。美国国家癌症研究所 (National Cancer Institute)也参与了相关研究。 But this cell-therapy approach had not been tried before in adults with t

8、he disease that Emma had, acute lymphoblastic leukemia. This type of blood cancer is worse in adults than in children, with a cure rate in adults of only about 40 percent, compared with 80 percent to 90 percent in children. The disease is not common. Each year in the United States, it affects about

9、2,400 people older than 20, and 3,600 younger. Though there are fewer cases in adults, there are more deaths: about 1,170 adults die each year compared with 270 deaths in people under 20. 但这种细胞疗法之前从未被用于急性淋巴细胞性白血病(即埃玛所患的白血病)的成人患 者。此种白血病对成人患者的危险性高于儿童,成人患者的治愈率只有40%,而儿童的治 愈率为80%到90%。这种疾病并不常见。在美国,每年大约有24

10、00名20岁以上的人患病和 3600名20岁以下的人患病。尽管成人病例相对更少,但成人患者的死亡率更高:每年大约有1170名成人患者死于该疾病,而20岁以下的患者中只有270名死于该疾病。 In adults, this type of leukemia is a “devastating, galloping disease,” said Dr. Michel Sadelain, the senior author of the new study and director of the Center for Cell Engineering and the Gene Transfer an

11、d Gene Expression Laboratory at Memorial Sloan-Kettering Cancer Center in Manhattan. 对成人患者来说,此类白血病是一种“破坏力强、发展迅速的疾病”,该研究的高级作者米 歇尔萨德莱恩博士(Michel Sadelain)说。他是曼哈顿纪念斯隆-凯特琳癌症中心细胞工程中 心(Center for Cell Engineering)以及基因转移和基因表达实验室(Gene Transfer and Gene Expression Laboratory)的负责人。 Patients like the ones in th

12、e study, who relapse after chemotherapy, usually have only a few months left, Dr. Sadelain said. But now, three of the five have been in remission for 5 to 24 months. Two others died: one was in remission but died from a blood clot, and the other relapsed. The survivors have gone on to have bone-mar

13、row transplants. Their prognosis is good, but relapse is still possible, and only time will tell. 萨德莱恩博士说,研究中的患者通常只有几个月的生命,他们的病情在化疗后会复发。但 如今,五名患者中有三名的缓解期已长达5到24个月。另外两名已经去世:其中一名患者处 于缓解状态,但死于血栓,另外一名则病情复发。生还者随即接受了骨髓移植。他们有望 康复,但是也有可能复发,只有时间能给出答案。 Experts not connected with the study said it was an impor

14、tant advance in an emerging field. Dr. Carl June of the University of Pennsylvania, who led the team that treated Emma and the other patients, said, “This is the first report showing some real, clinically beneficial activity in adult acute lymphoblastic leukemia.” He said his team was also starting

15、to test its version of the cell therapy on patients with the disease. 没有参与该研究的专家称,该研究是这一新生领域的重要进展。宾夕法尼亚大学的卡尔琼 博士(Carl June)曾带领治疗埃玛和其他患者的小组,他说, “这份报告首次显示了针对成人急 性淋巴细胞性白血病进行的一些真正有临床效益的研究活动。 ”他表示,他的团队也开始针 对这类病人测试自身研发的细胞疗法。 Dr. Richard M. Stone, the program director for adult leukemia at the Dana-Farber

16、Cancer Institute in Boston, called the research exciting and said he hoped to begin collaborating with the team at Sloan-Kettering. He has already sent them a patient. The treatment uses patients own T-cells, a type of white blood cell that normally fights viruses and cancer. The patients blood is run through a machine that extracts T-cells and returns the rest of the blood to the body. Researchers then do some genetic engineering: they use a disable

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