本文摘要：US regulators on Wednesday approved the first gene therapy against cancer - a treatment that uses a patients own immune cells to fight leukemia - opening a new era in the fight against one of the worlds top killers.美国监管机构于8月30日批准后了首个对付癌症的基因治疗手段——用于患者的免疫细胞对付白血病。

US regulators on Wednesday approved the first gene therapy against cancer - a treatment that uses a patients own immune cells to fight leukemia - opening a new era in the fight against one of the worlds top killers.美国监管机构于8月30日批准后了首个对付癌症的基因治疗手段——用于患者的免疫细胞对付白血病。白血病是世界上致死率最低的疾病之一，该化疗手段打开了对付白血病的新纪元。

The treatment is made by Novartis and is called Kymriah.该疗法由诺华公司研发，并被命名为Kymriah。This type of anti-cancer immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now.这种对付癌症的免疫疗法也被称作CAR-T细胞疗法，并因CTL019疗法（一种CAR-T细胞疗法）被人们熟悉。

This marks the first-ever CAR-T cell therapy to be approved anywhere in the world, Novartis CEO Joseph Jimenez told reporters on a conference call.诺华CEO江慕忠在一次电话会议上对记者说道：“这是全球首次容许用于CAR-T细胞疗法。”It uses a new approach that is wholly personalized by using a patients own T-cells.“这种新疗法用于患者自己的T细胞，是几乎个性化的。

”Kymriah was approved by the US Food and Drug Administration for children and young adult patients up to age 25 with a form of acute lymphoblastic leukemia (ALL).Kymriah取得美国食品药品监督管理局批准后，用作化疗25岁以下的急性淋巴性白血病患者。To qualify for treatment, patients must have B-cell precursor ALL that is refractory, or the patient has relapsed at least twice.只有患上难治型B细胞急性淋巴细胞白血病的患者或发作两次以上的患者才能取得化疗资格。

The FDA described the approval as a historic action and a new approach to the treatment of cancer and other serious and life-threatening diseases, said a statement.一份声明认为，美国食品药品监督管理局将这次批准后形容为“一次历史性的行径”和“化疗癌症及其他相当严重或可怕疾病的新途径”。The treatment is not a pill or a form of chemotherapy, which can weaken the bodys natural defenses.这种化疗手段并不用于药剂或化疗，会巩固人体自身的抵抗力。Instead, it harnesses a patients own immune cells, called T-cells and white blood cells, and trains them to recognise and fight cancer.该疗法用于患者自身的免疫细胞——T细胞和白细胞，并训练细胞辨识并对付癌症。

The patients immune cells are removed with a special blood filtration process, sent to a lab, and genetically encoded to be able to hunt down cancer cells.经过类似血液过滤器程序检验后，患者的免疫细胞被送到实验室，之后医疗人员将细胞新的展开基因编码后，彰显其捕捉癌细胞的能力。These re-engineered T-cells are then transfused back into the patient, where they can begin attacking leukemia.这些改建过的T细胞不会被输回患者体内，对付白血病。Studies have shown that 83 percent of patients responded to the treatment, achieving remission within three months, Novartis said.诺华称之为，研究找到，化疗对83%的患者有效地，患者的病情在三个月内顺利获得了减轻。

An application with the European Medicines Agency is expected to be filed by the end of the year.诺华预计将于今年年底向欧洲药品管理局提出申请。The price of Kymriah - which is delivered to a patient just once - is $475,000, said Bruno Strigini, CEO of Oncology at Novartis.诺华肿瘤事业部CEO布鲁诺?斯蒂格尼说道，Kymriah的价格是每次化疗47.5万美元。

Patients who do not respond to the treatment within the first month would not be expected to pay, he told reporters.他对记者说道，如果化疗一个月内对患者违宪，那么患者需要缴付。The more common treatment for leukemia - bone marrow transplants - can cost between $540,000 and $800,000 the first year in the United States, Strigini said.斯蒂格尼说道，白血病更加少见的化疗方法是骨髓移植法，第一年骨髓移植的价格在美国一般来说介于54万美元到80万美元之间。

Meanwhile, outside analyses have set a cost-effective price for Kymriah between $600,000 and $750,000, he added.他还说道，与此比起，外界分析指出，Kymriah的价格介于60万美元到75万美元更为合理。Recognising our responsibility we set the price below that level, said Strigini.“我们自知责任在身，所以没有将价格以定得那么低，” 斯蒂格尼说道。Most patients who fit the criteria for treatment would likely be covered by insurance, since they are under 25 and would either be on their parents insurance or covered by government-sponsored Medicaid, a Novartis spokesman said.诺华的发言人回应，大多数符合条件的患者很可能会用保险缴纳费用，因为他们都年满25岁，所以不会用于父母的保险或者政府资助的医疗补助金。

The treatment was pioneered by Carl June at the University of Pennsylvania.该疗法由宾夕法尼亚大学的卡尔?琼首创。Its most high-profile patient is Emily Whitehead, now 12.今年12岁的艾米丽?怀特海是该疗法尤为人熟悉的一位患者。

Six years ago, she was the first child to receive what was widely considered a risky treatment.6年前，艾米丽沦为首个拒绝接受当时社会普遍认为具备风险的Kymriah化疗的儿童。She has been cancer-free ever since.不过，Kymriah医治了她的癌症。In 2014, US regulators designated CTL019 as a breakthrough therapy and put the experimental immunotherapy agent on the fast track to market approval.2014年，美国监管机构确认CTL019为“突破性的疗法”，并很快将这种实验性的免疫疗法推上市场准入阶段。

It was the first cancer immunotherapy to receive the breakthrough designation. More are expected to follow in the coming years as the field of immunotherapy grows.这是首个被称作“突破性”的癌症免疫疗法。随着免疫疗法领域的发展，我们有期望在未来看见更加多突破性的疗法问世。

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