首個白血病治療基因療法在美獲批

美國監(jiān)管機(jī)構(gòu)8月30日批準(zhǔn)一種基于改造患者自身免疫細(xì)胞的療法治療白血病，這是第一種在美國獲得批準(zhǔn)的基因療法。專家認(rèn)為，這開辟了癌癥治療的新篇章。

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.

美國監(jiān)管機(jī)構(gòu)于8月30日批準(zhǔn)了首個對抗癌癥的基因治療手段——使用患者的免疫細(xì)胞對抗白血病。白血病是世界上致死率最高的疾病之一，該治療手段開啟了對抗白血病的新紀(jì)元。

The treatment is made by Novartis and is called Kymriah.

該療法由諾華公司研發(fā)，并被命名為Kymriah。

This type of anti-cancer immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now.

這種對抗癌癥的免疫療法也被稱作CAR-T細(xì)胞療法，并因CTL019療法(一種CAR-T細(xì)胞療法)被人們熟知。

"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.

諾華CEO江慕忠在一次電話會議上對記者說：“這是全球首次允許使用CAR-T細(xì)胞療法。”

"It uses a new approach that is wholly personalized by using a patient's own T-cells."

“這種新療法使用患者自己的T細(xì)胞，是完全個性化的。”

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獲得美國食品藥品監(jiān)督管理局批準(zhǔn)，用于治療25歲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.

只有患有難治型B細(xì)胞急性淋巴細(xì)胞白血病的患者或復(fù)發(fā)兩次以上的患者才能獲得治療資格。

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.

一份聲明指出，美國食品藥品監(jiān)督管理局將這次批準(zhǔn)形容為“一次歷史性的舉動”和“治療癌癥及其他嚴(yán)重或致命疾病的新途徑”。

如何治療

The treatment is not a pill or a form of chemotherapy, which can weaken the body's natural defenses.

這種治療手段并不使用藥劑或化療，不會削弱人體自身的抵抗力。

Instead, it harnesses a patient's own immune cells, called T-cells and white blood cells, and trains them to recognise and fight cancer.

該療法使用患者自身的免疫細(xì)胞——T細(xì)胞和白細(xì)胞，并訓(xùn)練細(xì)胞識別并對抗癌癥。

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.

經(jīng)過特殊血液過濾程序篩選后，患者的免疫細(xì)胞被送往實驗室，之后醫(yī)療人員將細(xì)胞重新進(jìn)行基因編碼后，賦予其捕獲癌細(xì)胞的能力。

These re-engineered T-cells are then transfused back into the patient, where they can begin attacking leukemia.

這些改造過的T細(xì)胞會被輸回患者體內(nèi)，對抗白血病。

Studies have shown that 83 percent of patients responded to the treatment, achieving remission within three months, Novartis said.

諾華稱，研究發(fā)現(xiàn)，治療對83%的患者有效，患者的病情在三個月內(nèi)成功得到了緩解。

An application with the European Medicines Agency is expected to be filed by the end of the year.

諾華預(yù)計將于今年年底向歐洲藥品管理局提出申請。

價格：每次47.5萬美元

The price of Kymriah - which is delivered to a patient just once - is $475,000, said Bruno Strigini, CEO of Oncology at Novartis.

諾華腫瘤事業(yè)部CEO布魯諾•斯蒂格尼說，Kymriah的價格是每次治療47.5萬美元。

Patients who do not respond to the treatment within the first month would not be expected to pay, he told reporters.

他對記者說，如果治療一個月內(nèi)對患者無效，那么患者無需付款。

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.

斯蒂格尼說，白血病更常見的治療方法是骨髓移植法，第一年骨髓移植的價格在美國通常介于54萬美元到80萬美元之間。

Meanwhile, outside analyses have set a cost-effective price for Kymriah between $600,000 and $750,000, he added.

他還說，與此相比，外界分析認(rèn)為，Kymriah的價格介于60萬美元到75萬美元較為合理。

"Recognising our responsibility we set the price below that level," said Strigini.

“我們深知責(zé)任在身，所以沒將價格定得那么高，” 斯蒂格尼說。

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.

諾華的發(fā)言人表示，大多數(shù)符合條件的患者很可能會用保險支付費用，因為他們都未滿25歲，所以會使用父母的保險或者政府資助的醫(yī)療補助。

突破性的療法

The treatment was pioneered by Carl June at the University of Pennsylvania.

該療法由賓夕法尼亞大學(xué)的卡爾•瓊開創(chuàng)。

Its most high-profile patient is Emily Whitehead, now 12.

今年12歲的艾米麗•懷特海是該療法最為人熟知的一位患者。

Six years ago, she was the first child to receive what was widely considered a risky treatment.

6年前，艾米麗成為首個接受當(dāng)時社會普遍認(rèn)為具有風(fēng)險的Kymriah治療的兒童。

She has been cancer-free ever since.

不過，Kymriah治好了她的癌癥。

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，美國監(jiān)管機(jī)構(gòu)認(rèn)定CTL019為“突破性的療法”，并迅速將這種實驗性的免疫療法推向市場準(zhǔn)入階段。

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.

這是首個被稱為“突破性”的癌癥免疫療法。隨著免疫療法領(lǐng)域的發(fā)展，我們有希望在未來看到更多突破性的療法誕生。