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首个白血病治疗基因疗法在美获批

文章来源:威尼斯欢乐娱人成app         发布时间:2021-10-25 01:24

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.美国管控组织于8月20日准许后了第一个应对癌病的

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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.美国管控组织于8月20日准许后了第一个应对癌病的基因疗法方式——用以患者的细胞免疫应对白血病。白血病是全世界死亡率最少的病症之一,该化疗方式打开了应对白血病的新世界。

The treatment is made by Novartis and is called Kymriah.该疗法由诺华公司产品研发,并被取名为Kymriah。This type of anti-cancer immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now.这类应对癌病的免疫力疗法也称之为CAR-T体细胞疗法,并因CTL019疗法(一种CAR-T体细胞疗法)被大家了解。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.诺华制药CEO江慕忠在一次会议电话上对新闻记者说道:“它是全世界初次允许用以CAR-T体细胞疗法。

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”It uses a new approach that is wholly personalized by using a patients own T-cells.“这类新疗法用以患者自身的T体细胞,是基本上人性化的。”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获得美国食品类药监局准许后,作为化疗二十五岁下列的亚急性淋巴结性白血病患者。To qualify for treatment, patients must have B-cell precursor ALL that is refractory, or the patient has relapsed at least twice.仅有得了不易治型B体细胞亚急性网织红细胞白血病的患者或发病2次之上的患者才可以获得化疗资质。

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.一份申明觉得,美国食品类药监局将此次准许后描述为“一次里程碑式的行为”和“化疗癌病以及他相当严重或恐怖病症的有效途径”。The treatment is not a pill or a form of chemotherapy, which can weaken the bodys natural defenses.这类化疗方式并不用以药物或化疗,会推进身体本身的抵抗能力。Instead, it harnesses a patients own immune cells, called T-cells and white blood cells, and trains them to recognise and fight cancer.该疗法用以患者本身的细胞免疫——T体细胞和白细胞计数,并训炼体细胞识别并应对癌病。

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.历经相近血夜过滤装置程序流程检测后,患者的细胞免疫被送至试验室,以后医疗人员将体细胞新的进行基因编码后,突显其捕获肿瘤细胞的工作能力。These re-engineered T-cells are then transfused back into the patient, where they can begin attacking leukemia.这种改造过的T体细胞不容易被输回患者身体,应对白血病。Studies have shown that 83 percent of patients responded to the treatment, achieving remission within three months, Novartis said.诺华制药称作,科学研究寻找,化疗对83%的患者合理地,患者的病况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.诺华制药预估将于今年底向欧州药物管理处提交申请。The price of Kymriah - which is delivered to a patient just once - is $475,000, said Bruno Strigini, CEO of Oncology at Novartis.诺华肿瘤业务部CEO比埃尔?斯蒂格尼说道,Kymriah的价钱是每一次化疗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.他对新闻记者说道,假如化疗一个月内对患者违宪,那麼患者必须缴纳。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,白血病更为罕见的化疗方式是骨髓移植法,第一年骨髓移植的价钱在美国一般来说接近54万美金到80万美金中间。Meanwhile, outside analyses have set a cost-effective price for Kymriah between $600,000 and $750,000, he added.他还说道,与此相比,外部剖析强调,Kymriah的价钱接近60万美金到75万美金更加有效。Recognising our responsibility we set the price below that level, said Strigini.“大家知道义务在身,因此 沒有将价钱以定得那麼低,” 斯蒂格尼说道。

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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.诺华制药的新闻发言人答复,大部分满足条件的患者很可能会用保险缴纳花费,由于她们都法定年龄二十五岁,因此 不容易用以爸爸妈妈的商业保险或是政府部门支助的诊疗补助费。The treatment was pioneered by Carl June at the University of Pennsylvania.该疗法由宾夕法尼亚大学的梅帝?琼创新。Its most high-profile patient is Emily Whitehead, now 12.2020年十二岁的艾米丽?怀特海是该疗法尤其人了解的一位患者。

Six years ago, she was the first child to receive what was widely considered a risky treatment.六年前,艾米丽沦落第一个拒不接受那时候社会发展广泛认为具有风险性的Kymriah化疗的少年儿童。She has been cancer-free ever since.但是,Kymriah治疗了她的癌病。In 2014, US regulators designated CTL019 as a breakthrough therapy and put the experimental immunotherapy agent on the fast track to market approval.二零一四年,美国管控组织确定CTL019为“开创性的疗法”,并迅速将这类试验性的免疫力疗法推上去市场准入制度环节。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.它是第一个称之为“开创性”的癌病免疫力疗法。

伴随着免疫力疗法行业的发展趋势,大家有期待在未来看到更为多开创性的疗法面世。


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