美国国家公共电台 NPR At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever

 

MARY LOUISE KELLY, HOST:

The Food and Drug Administration approved a new form of therapy today for a devastating¹ genetic³ disease. It is providing hope for babies born with this rare but often fatal disorder⁴. But as NPR health correspondent Rob Stein reports, at more than $2 million, the treatment is the most expensive drug ever approved.

ROB STEIN, BYLINE⁵: When Donovan Weisgarber was born, he seemed perfectly⁶ healthy. But within weeks, his mom, Laura, says it became clear something was wrong - terribly wrong.

LAURA WEISGARBER: It was when he was about 1 month old - was when we started to notice some symptoms.

STEIN: He started getting really fussy⁷, stopped squirming and got weaker and weaker. Turns out Donovan had a genetic disorder, spinal⁸ muscular atrophy⁹. It was destroying the nerves that control his muscles. Many babies don't live beyond their second birthday. It's the most common genetic cause of death among infants.

WEISGARBER: We were devastated¹⁰ obviously. Yeah, you know, obviously you're devastated. It was definitely the worst time of our lives.

STEIN: But then doctors told Laura and her husband, Matthew, about something new. They might be able to replace the defective¹¹ gene² killing¹² Donovan with a new type of gene therapy. So they agreed to let doctors infuse Donovan with genetically¹³ modified viruses carrying healthy genes¹⁴ into his body. Donovan slowly started to improve, and three years later, Donovan still needs a wheelchair and a feeding tube but otherwise is doing great, his mom says.

WEISGARBER: You know, he loves going outside. He loves playing with his family. He goes to preschool. So yeah, he gets to do a lot of normal things. So it's just - I mean, it's amazing.

STEIN: And Donovan isn't alone. This gene therapy has been saving other babies with spinal muscular atrophy. David Lennon is the president of AveXis, the company that makes what's now called Zolgensma.

DAVID LENNON: First of all, a hundred percent of the kids survived. And this is a population where 92% of the kids would expect to have died or be on permanent ventilation by the time they're 20 months old. But then we also saw additional things. A lot of these kids could swallow. Seventy-five percent of these kids were able to sit. And we actually had a few kids who were able to stand and walk independently.

STEIN: Based on these results, Lennon says the company is justified¹⁵ in setting the price at $2.125 million for each child, which would make it the most expensive drug ever approved. And that price tag is making a lot of jaws¹⁶ drop.

PETER BACH: You know, it's absolutely stunning¹⁷.

STEIN: Peter Bach studies health policy at Memorial Sloan Cancer Center in New York.

BACH: It's just alarming that we have gotten to a point where, you know, any treatment that is a product of this collective scientific enterprise that has grown out of the Human Genome Project that was publicly funded has now been captured by a single drug company and is now going to turn around and charge potentially millions.

STEIN: Bach says it's just the most extreme example of how drug prices are draining resources from society.

BACH: We have been slowly subjected to price increases the same way the frog in the boiling water, you know, is slowly boiled to death.

STEIN: Now, Lennon acknowledges the price might seem shocking. But he argues it's worth it. The only existing treatment for spinal muscular atrophy costs hundreds of thousands of dollars a year, and this will hopefully be a one-time, lifesaving treatment that will last a lifetime.

LENNON: What we're talking about and we have to remember is we're talking about a lifetime of benefit being condensed down into a one-time treatment. We're not used to thinking about this that way. We're used to a system of chronic¹⁸ medication where we spread costs out over years, if not decades.

STEIN: Drug companies need to be able to recoup the costs of developing lifesaving, cutting-edge treatments, Lennon says, if they're going to be encouraged to find new breakthroughs. Donovan's parents didn't have to pay because their son was part of a research study, but they think the treatment is worth the price.

WEISGARBER: Giving someone a life, someone that would have died in infancy¹⁹ or early childhood the opportunity to live into adulthood²⁰, I mean, I don't know. I think that's valuable. I think it's a valuable investment.

STEIN: Rob Stein, NPR News.

1 devastating   

adj.毁灭性的，令人震惊的，强有力的

参考例句：

• It is the most devastating storm in 20 years.这是20年来破坏性最大的风暴。
• Affairs do have a devastating effect on marriages.婚外情确实会对婚姻造成毁灭性的影响。

2 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。

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 byline   

n.署名；v.署名

参考例句：

• His byline was absent as well.他的署名也不见了。
• We wish to thank the author of this article which carries no byline.我们要感谢这篇文章的那位没有署名的作者。

6 perfectly   

adv.完美地，无可非议地，彻底地

参考例句：

• The witnesses were each perfectly certain of what they said.证人们个个对自己所说的话十分肯定。
• Everything that we're doing is all perfectly above board.我们做的每件事情都是光明正大的。

7 fussy   

adj.为琐事担忧的，过分装饰的，爱挑剔的

参考例句：

• He is fussy about the way his food's cooked.他过分计较食物的烹调。
• The little girl dislikes her fussy parents.小女孩讨厌她那过分操心的父母。

8 spinal   

adj.针的，尖刺的，尖刺状突起的；adj.脊骨的，脊髓的

参考例句：

• After three days in Japan,the spinal column becomes extraordinarily flexible.在日本三天，就已经使脊椎骨变得富有弹性了。
• Your spinal column is made up of 24 movable vertebrae.你的脊柱由24个活动的脊椎骨构成。

9 atrophy   

n./v.萎缩，虚脱，衰退

参考例句：

• Patients exercised their atrophied limbs in the swimming pool.病人们在泳池里锻炼萎缩的四肢。
• Many hoped he would renew the country's atrophied political system.很多人都期望他能使该国萎靡的政治体系振作起来。

10 devastated   

v.彻底破坏( devastate的过去式和过去分词)；摧毁；毁灭；在感情上（精神上、财务上等）压垮adj.毁坏的；极为震惊的

参考例句：

• The bomb devastated much of the old part of the city. 这颗炸弹炸毁了旧城的一大片地方。
• His family is absolutely devastated. 他的一家感到极为震惊。

11 defective   

adj.有毛病的，有问题的，有瑕疵的

参考例句：

• The firm had received bad publicity over a defective product. 该公司因为一件次品而受到媒体攻击。
• If the goods prove defective, the customer has the right to compensation. 如果货品证明有缺陷， 顾客有权索赔。

12 killing   

n.巨额利润；突然赚大钱，发大财

参考例句：

• Investors are set to make a killing from the sell-off.投资者准备清仓以便大赚一笔。
• Last week my brother made a killing on Wall Street.上个周我兄弟在华尔街赚了一大笔。

13 genetically   

adv.遗传上

参考例句：

• All the bees in the colony are genetically related. 同一群体的蜜蜂都有亲缘关系。
• Genetically modified foods have already arrived on American dinner tables. 经基因改造加工过的食物已端上了美国人的餐桌。 来自英汉非文学 - 生命科学 - 基因与食物

14 genes   

n.基因( gene的名词复数 )

参考例句：

• You have good genes from your parents, so you should live a long time. 你从父母那儿获得优良的基因，所以能够活得很长。 来自《简明英汉词典》
• Differences will help to reveal the functions of the genes. 它们间的差异将会帮助我们揭开基因多种功能。 来自英汉非文学 - 生命科学 - 生物技术的世纪

15 justified   

a.正当的，有理的

参考例句：

• She felt fully justified in asking for her money back. 她认为有充分的理由要求退款。
• The prisoner has certainly justified his claims by his actions. 那个囚犯确实已用自己的行动表明他的要求是正当的。

16 jaws   

n.口部；嘴

参考例句：

• The antelope could not escape the crocodile's gaping jaws. 那只羚羊无法从鱷鱼张开的大口中逃脱。
• The scored jaws of a vise help it bite the work. 台钳上有刻痕的虎钳牙帮助它紧咬住工件。

17 stunning   

adj.极好的；使人晕倒的

参考例句：

• His plays are distinguished only by their stunning mediocrity.他的戏剧与众不同之处就是平凡得出奇。
• The finished effect was absolutely stunning.完工后的效果非常美。

18 chronic   

adj.(疾病)长期未愈的，慢性的；极坏的

参考例句：

• Famine differs from chronic malnutrition.饥荒不同于慢性营养不良。
• Chronic poisoning may lead to death from inanition.慢性中毒也可能由虚弱导致死亡。

19 infancy   

n.婴儿期；幼年期；初期

参考例句：

• He came to England in his infancy.他幼年时期来到英国。
• Their research is only in its infancy.他们的研究处于初级阶段。

20 adulthood   

n.成年，成人期

参考例句：

• Some infantile actions survive into adulthood.某些婴儿期的行为一直保持到成年期。
• Few people nowadays are able to maintain friendships into adulthood.如今很少有人能将友谊维持到成年。