2019-05-29 At $2.1 Million, New
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内容简介：基因疗法。
At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever
May 24, 2019
MARY LOUISE KELLY, HOST:The Food and Drug Administration approved a new form of therapy today for a devastating genetic disease. It is providing hope for babies born with this rare but often fatal disorder. But as NPR health correspondent Rob Stein reports, at more than $2 million, the treatment is the most expensive drug ever approved.
ROB STEIN, BYLINE: When Donovan Weisgarber was born, he seemed perfectly 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 fussy, stopped squirming and got weaker and weaker. Turns out Donovan had a genetic disorder, spinal muscular atrophy. 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 devastated 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 defective gene killing Donovan with a new type of gene therapy. So they agreed to let doctors infuse Donovan with genetically modified viruses carrying healthy genes 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 justified 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 jaws drop.
PETER BACH: You know, it's absolutely stunning.
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 chronic 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 infancy or early childhood the opportunity to live into adulthood, I mean, I don't know. I think that's valuable. I think it's a valuable investment.
STEIN: Rob Stein, NPR News.
知识点笔记：
1.gene thrapy:基因疗法。文中在病例介绍中说明了这一过程：So they agreed to let doctors infuse Donovan with genetically modified viruses carrying healthy genes into his body. 原来它是通过病毒为载体，将健康基因带入体内，用于治疗疾病。
2.devastate:1.毁坏；2.使某人震惊、伤心。文中用了"devastating genetic disease"和"...we were devastated"描述了疾病给家庭的打击。
e.g.1.Long-term unemployment can be devastating.
2.Another devastating week for Ebola in west Africa.
3.Bishop Daly said he was devastated by news of the Cardinal's death...
4.Teresa was devastated, her dreams shattered.
3.spinal muscular atrophy: 一种疾病，如果不知道医学名词是什么意思，后面的一句就对此进行了说明。It was destroying the nerves that control his muscles.损害了控制肌肉的神经，因此严重影响运动功能、甚至吞咽等动作都变得困难。
4....that price tag is making a lot of jaws drop.这句很形象生动，价格让许多人惊掉了下巴。
后面的一句"You know, it's absolutely stunning."也是强调震惊。stunning既可表示绝妙的、很棒的；也可表示令人吃惊的。
e.g.1.She was 55 and still a stunning woman...
2.A stunning display of fireworks lit up the sky.
3.The minister resigned last night after a stunning defeat in Sunday's vote...
4.The secret that the priest had confided to him was a stunning piece of news.
5.drain：流干，引申义：(金钱或资源的)大量消耗，耗竭
e.g.1.Huge imports were draining the country's currency reserves.
2....an ultra-modern printing plant, which has been a big drain on resources...
3.brain drain人才流失
6.cutting-edge：最前沿的、最新的
e.g.1.What we are planning is cutting-edge technology never seen in Australia before...
2.Some of what is happening at Packard is cutting-edge medicine.
