首个白血病治疗基因疗法在美获批

US regulators on Wednesday approved the first gene therapy against cancer - a treatment that uses a patient's own immune cells to fight leukemia - opening a new era in the fight against one of the world's top killers.
美国监管机构于8月30日批准了首个对抗癌症的基因治疗手段——使用患者的免疫细胞对抗白血病。白血病是世界上致死率最高的疾病之一，该治疗手段开启了对抗白血病的新纪元。

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.
这种对抗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）被人们熟知。

"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：“这是全球首次允许使用CAR-T细胞疗法。”

"It uses a new approach that is wholly personalized by using a patient's own T-cells."
“这种新疗法使用患者自己的T细胞，是完全个性化的。”

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获得美国食品药品监督管理局批准，用于治疗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.
只有患有难治型B细胞急性淋巴细胞白血病的患者或复发两次以上的患者才能获得治疗资格。

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.
一份声明指出，美国食品药品监督管理局将这次批准形容为“一次历史性的举动”和“治疗癌症及其他严重或致命疾病的新途径”。

如何治疗

The treatment is not a pill or a form of chemotherapy, which can weaken the body's natural defenses.
这种治疗手段并不使用药剂或化疗，不会削弱人体自身的抵抗力。

Instead, it harnesses a patient's own immune cells, called T-cells and white blood cells, and trains them to recognise and fight cancer.
该疗法使用患者自身的免疫细胞——T细胞和白细胞，并训练细胞识别并对抗癌症。

The patient's 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.
经过特殊血液过滤程序筛选后，患者的免疫细胞被送往实验室，之后医疗人员将细胞重新进行基因编码后，赋予其捕获癌细胞的能力。

These re-engineered T-cells are then transfused back into the patient, where they can begin attacking leukemia.
这些改造过的T细胞会被输回患者体内，对抗白血病。

Studies have shown that 83 percent of patients responded to the treatment, achieving remission within three months, Novartis said.
诺华称，研究发现，治疗对83%的患者有效，患者的病情在三个月内成功得到了缓解。

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。

价格：每次47.5万美元

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，Kymriah的价格是每次治疗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，如果治疗一个月内对患者无效，那么患者无需付款。

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，白血病更常见的治疗方法是骨髓移植法，第一年骨髓移植的价格在美国通常介于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，与此相比，外界分析认为，Kymriah的价格介于60万美元到75万美元较为合理。

"Recognising our responsibility we set the price below that level," said Strigini.
“我们深知责任在身，所以没将价格定得那么高，” 斯蒂格尼说。

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.
诺华的发言人表示，大多数符合条件的患者很可能会用保险支付费用，因为他们都未满25岁，所以会使用父母的保险或者政府资助的医疗补助。

突破性的疗法

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•琼开创。

Its most high-profile patient is Emily Whitehead, now 12.
今年12岁的艾米丽•怀特海是该疗法最为人熟知的一位患者。

Six years ago, she was the first child to receive what was widely considered a risky treatment.
6年前，艾米丽成为首个接受当时社会普遍认为具有风险的Kymriah治疗的儿童。

She has been cancer-free ever since.
不过，Kymriah治好了她的癌症。

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，美国监管机构认定CTL019为“突破性的疗法”，并迅速将这种实验性的免疫疗法推向市场准入阶段。

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.
这是首个被称为“突破性”的癌症免疫疗法。随着免疫疗法领域的发展，我们有希望在未来看到更多突破性的疗法诞生。