VOA健康报道2023 FDA或批准首个可能治愈镰状细胞病的疗法

FDA或批准首个可能治愈镰状细胞病的疗法

A group of health experts recently announced that a new gene¹ editing treatment for sickle² cell disease was safe for patients.

一个健康专家小组最近宣布，一种新的针对镰状细胞病的基因编辑疗法对患者来说是安全的。

The treatment could provide a new cure that attacks the disease at its genetic³ source.

这种疗法可能会提供一种新的从基因源头攻克这种疾病的治愈方法。

Sickle cell disease (SCD) is a genetic blood disorder⁴ that affects millions of people worldwide.

镰状细胞病是一种遗传性血液疾病，影响全球数百万人。

The disease is common in places with high cases of malaria⁵, like Africa and India.

这种疾病在疟疾高发区很常见，比如非洲和印度。

It strikes among Black Americans, Africans, and Middle Easterners more than other groups.

相较于其他群体，美国黑人、非洲人和中东人更容易患镰状细胞病。

Scientists believe being a carrier of SCD helps protect against severe malaria.

科学家认为，成为镰状细胞病的携带者有助于预防严重的疟疾。

People with SCD carry unhealthy blood cells that result in blockages⁶, called blood clots⁷.

患有镰状细胞病的人携带着不健康的血细胞，它们会导致阻塞，即血栓。

This can cause pain and damage bones and organs in the human body.

这会引起疼痛，损害人体的骨骼和器官。

The World Health Organization said many children with the most severe form of the disease die before the age of five, usually from an infection or severe blood loss.

世界卫生组织表示，许多患有最严重镰状细胞病的儿童在五岁之前死亡，通常死于感染或严重失血。

Vertex Pharmaceuticals⁸ and CRISPR Therapeutics make the new treatment, called "exa-cel."

福泰制药和CRISPR Therapeutics公司创造了这种名为“exa-cel”的新疗法。

The treatment involves permanently⁹ changing the genetic material, DNA¹⁰, in a patient's blood cells.

这种疗法包括永久改变患者血细胞中的遗传物质DNA。

The goal is to help the body return to healthy blood cell production.

其目标是帮助身体恢复健康的血细胞生产功能。

Healthy blood cells are present at birth but abnormal cells begin to develop in people with sickle cell disease.

健康的血细胞在出生时就存在，但患有镰状细胞病的人开始出现异常细胞。

When patients receive the treatment, stem cells are removed from their blood and the gene-editing tool CRISPR is used to remove the switching gene.

当患者接受治疗时，医生会从他们的血液中移除干细胞，并使用基因编辑工具CRISPR移除开关基因。

Patients get medicines to kill off other damaged blood-producing cells and then are given back their changed stem cells.

患者服用药物来杀死其他受损的造血细胞，然后医生再把编辑过的干细胞放回原位。

Vertex reported 46 people got the treatment in its study.

福泰制药报告称，有46人在其研究中接受了这种治疗。

Among 30 of them who had at least 18 months of observation, 29 were free of pain crises for at least a year.

在30名至少被观察了18个月的患者中，有29名患者至少一年没有出现疼痛危机。

All 30, Vertex reported, avoided being hospitalized for pain crises for that long.

据福泰制药报告，所有30名患者在很长一段时间内都没有因疼痛危机而住院。

Victoria Gray, of Mississippi, was the first patient to test the "exa-cel" treatment.

密西西比州的维多利亚·格雷是第一个测试这种“exa-cel”疗法的患者。

Gray told researchers at a scientific gathering¹¹ earlier this year that she had suffered from pain since childhood.

格雷在今年早些时候的一次科学会议上告诉研究人员，她从小就饱受疼痛之苦。

She described feeling "reborn" the day she got the treatment.

她称，在接受治疗的那一天，她感觉自己“重获新生”。

"My children no longer have a fear of losing their mom to sickle cell disease," she said.

她说：“我的孩子们再也不用害怕因为镰状细胞病而失去他们的母亲”。

The U.S. Food and Drug Administration (FDA) had already found the treatment effective in a study of SCD patients.

美国食品药品监督管理局已经在一项针对镰状细胞病患者的研究中发现了这种治疗方法的有效性。

The health agency, however, was not sure if the small genetic sample size used in the study captured the entire U.S. population for sickle cell disease.

然而，该卫生机构不确定研究中使用的少量基因样本是否涵盖了整个美国镰状细胞病患者群。

With the experts' findings, the FDA is likely to approve the treatment in early December for patients age 12 and older.

根据专家的研究结果，美国食品药品监督管理局可能会在12月初批准对12岁及以上患者使用这种疗法。

If the treatment is approved, Vertex has also proposed to follow up with patients to study its safety for another 15 years.

如果该疗法获得批准，福泰制药还提议对患者进行随访，再进行15年的安全性研究。

Vertex has not said how much the treatment will cost.

福泰制药尚未透露治疗费用。

But the Institute for Clinical and Economic Review estimates the cost could be around $2 million for the treatment.

但临床和经济审查研究所估计，这种疗法的费用可能在200万美元左右。

By comparison, research earlier this year showed medical costs for sickle cell treatments, from birth to age 65, could add up to $1.7 million.

相比之下，今年早些时候的研究显示，镰状细胞病患者从出生到65岁的医疗费用总计可能高达170万美元。

Dr. Allison King of Washington University School of Medicine in St. Louis, Missouri cares for children and young adults with sickle cell disease.

密苏里州圣路易斯市华盛顿大学医学院的艾利森·金博士负责治疗患有镰状细胞病的儿童和年轻人。

She told the Associated Press that the new treatments would be costly¹².

她告诉美联社，新的治疗方法费用高昂。

"But if you think about it," she said, "how much is it worth for someone to feel better and not be in pain and not be in the hospital all the time?"

她说：“但如果你仔细想想，让人感觉好一些，不再痛苦，不再一直待在医院里，这值多少钱？”

1 gene   

n.遗传因子，基因

参考例句：

• A single gene may have many effects.单一基因可能具有很多种效应。
• The targeting of gene therapy has been paid close attention.其中基因治疗的靶向性是值得密切关注的问题之一。

2 sickle   

n.镰刀

参考例句：

• The gardener was swishing off the tops of weeds with a sickle.园丁正在用镰刀嗖嗖地割掉杂草的顶端。
• There is a picture of the sickle on the flag. 旗帜上有镰刀的图案。

3 genetic   

adj.遗传的，遗传学的

参考例句：

• It's very difficult to treat genetic diseases.遗传性疾病治疗起来很困难。
• Each daughter cell can receive a full complement of the genetic information.每个子细胞可以收到遗传信息的一个完全补偿物。

4 disorder   

n.紊乱，混乱；骚动，骚乱；疾病，失调

参考例句：

• When returning back,he discovered the room to be in disorder.回家后，他发现屋子里乱七八糟。
• It contained a vast number of letters in great disorder.里面七零八落地装着许多信件。

5 malaria   

n.疟疾

参考例句：

• He had frequent attacks of malaria.他常患疟疾。
• Malaria is a kind of serious malady.疟疾是一种严重的疾病。

6 blockages   

n.堵塞物( blockage的名词复数 )；堵塞，阻塞

参考例句：

• The storms could increase the risks posed by river blockages. 暴风雨会增加因河道堵塞所造成的危险。 来自互联网
• An angiogram shows the location and severity of blockages in blood vessels. 冠状动脉造影能够显示血管内的阻塞位置以及阻塞的严重程度。 来自互联网

7 clots   

n.凝块( clot的名词复数 )；血块；蠢人；傻瓜v.凝固( clot的第三人称单数 )

参考例句：

• When you cut yourself, blood clots and forms a scab. 你割破了，血会凝固、结痂。 来自《简明英汉词典》
• Milk clots when it turns sour. 奶变酸就凝块。 来自《现代英汉综合大词典》

8 pharmaceuticals   

n.医药品；药物( pharmaceutical的名词复数 )

参考例句：

• the development of new pharmaceuticals 新药的开发
• The companies are pouring trillions of yen into biotechnology research,especially for pharmaceuticals and new seeds. 这些公司将大量资金投入生物工艺学研究，尤其是药品和新种子方面。 来自《简明英汉词典》

9 permanently   

adv.永恒地，永久地，固定不变地

参考例句：

• The accident left him permanently scarred.那次事故给他留下了永久的伤疤。
• The ship is now permanently moored on the Thames in London.该船现在永久地停泊在伦敦泰晤士河边。

10 DNA   

(缩)deoxyribonucleic acid 脱氧核糖核酸

参考例句：

• DNA is stored in the nucleus of a cell.脱氧核糖核酸储存于细胞的细胞核里。
• Gene mutations are alterations in the DNA code.基因突变是指DNA密码的改变。

11 gathering   

n.集会，聚会，聚集

参考例句：

• He called on Mr. White to speak at the gathering.他请怀特先生在集会上讲话。
• He is on the wing gathering material for his novels.他正忙于为他的小说收集资料。

12 costly   

adj.昂贵的，价值高的，豪华的

参考例句：

• It must be very costly to keep up a house like this.维修这么一幢房子一定很昂贵。
• This dictionary is very useful,only it is a bit costly.这本词典很有用，左不过贵了些。