美国国家公共电台 NPR FDA Panel Endorses Gene Therapy For A Form Of Childhood Blindness

 

KELLY MCEVERS, HOST:

The dream of gene¹ therapy took a big step forward today. A Food and Drug Administration advisory² committee unanimously endorsed³ the first gene therapy for an inherited disorder⁴. NPR health correspondent Rob Stein is with us now for the details. Hey, Rob.

ROB STEIN, BYLINE⁵: Oh, hey there.

MCEVERS: So let's start by talking about the disease that this gene therapy would be used to treat. Tell us about it.

STEIN: Yeah, it's called retinal dystrophy. And it's a genetic⁶ disorder that causes progressive damage to the retina in the eye often starting in childhood and slowly leading to blindness. It's a pretty rare disease. There are maybe about 6,000 cases worldwide. A couple of thousand of those are in the United States.

MCEVERS: And so these 6,000 people could benefit from gene therapy. But what does that mean, gene therapy? How does it work?

STEIN: Yeah, it's pretty interesting stuff, Kelly. So what they did is scientists took a harmless virus, and they genetically⁷ engineered it to make a - to make it into kind of like a delivery drone to ferry a healthy version of the gene that's defective⁸ in this disease into patients' eyes. And then doctors inject billions of these genetically modified viruses into the patients' eyes. That gets the healthy gene into the retina, where it pumps out a healthy version of an enzyme⁹ that's needed for healthy vision.

MCEVERS: How well does it work?

STEIN: Well, so far it looks like it's working really well, you know? Now, it's only been tested on a couple of dozen patients so far, but it seemed like almost all of them benefited to some degree, or at least most of them did. For some of them, it was just kind of modest improvement. But for others, their vision started to improve within days of getting these injections.

And it improved pretty dramatically within a year. You know, they went from being legally blind - you know, they were unable to walk around without a cane¹⁰ or someone guiding them. And they were - a lot of them were totally trapped in their houses at night because they had terrible night vision. And then they suddenly could do things they could never do before.

Like, during today's hearings, some kids got up, and some teenagers described that they could for the first time do things like read, ride a bike, play sports, play baseball, see their parents' faces. You know, and one young man got up and said for the first time, he could go out at night, look up at the sky and see the stars and the moon. It was pretty dramatic, and it really opened up the world for a lot of these people.

MCEVERS: Wow. So an FDA committee endorses¹¹ it. So does that mean that it is safe? I mean, are there side effects? And how long might those last?

STEIN: Yeah, yeah. So far it seems pretty safe. You know, there sometimes were some complications that occurred from the procedure of injecting something into the eye, the physical procedure. But so far there are no signs at all that the gene therapy itself was dangerous, that injecting a gene into the eye caused any significant complications.

Now, so far it does seem to be lasting¹², but that's a big question. How long will it continue to last? These patients have been followed for maybe three years, and it's continuing to work. But they're going to have to follow these patients for longer to be sure.

MCEVERS: Any idea of how much it would cost?

STEIN: So that's a big question. There's a lot of speculations¹³ by analysts¹⁴ that it could be pretty expensive. You know, any - some of the newer treatments that have been approved recently have cost hundreds of thousands of dollars. And so you can imagine if this costs, you know, $400,000 or $500,000 for each eye, we're talking about potentially a million-dollar treatment. But the company hasn't said yet what it's going to charge for this product.

MCEVERS: What does today's news say about where gene therapy stands overall?

STEIN: You know, scientists and doctors have been dreaming about doing gene therapy for decades, but early on, there were some real setbacks. Some patients died, and people thought it was never going to happen. But recently there was another treatment approved, and it looks pretty promising¹⁵ for a number of conditions. So it looks like we might be at a turning point here.

MCEVERS: NPR health correspondent Rob Stein, thanks a lot.

STEIN: Oh, sure. Nice to be here.

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 advisory   

adj.劝告的，忠告的，顾问的，提供咨询

参考例句：

• I have worked in an advisory capacity with many hospitals.我曾在多家医院做过顾问工作。
• He was appointed to the advisory committee last month.他上个月获任命为顾问委员会委员。

3 endorsed   

vt.& vi.endorse的过去式或过去分词形式v.赞同( endorse的过去式和过去分词 )；在（尤指支票的）背面签字；在（文件的）背面写评论；在广告上说本人使用并赞同某产品

参考例句：

• The committee endorsed an initiative by the chairman to enter discussion about a possible merger. 委员会通过了主席提出的新方案，开始就可能进行的并购进行讨论。 来自《简明英汉词典》
• The government has broadly endorsed a research paper proposing new educational targets for 14-year-olds. 政府基本上支持建议对14 岁少年实行新教育目标的研究报告。 来自《简明英汉词典》

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 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。

7 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

8 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。

9 enzyme   

n.酵素，酶

参考例句：

• Above a certain temperature,the enzyme molecule will become unfolded.超过一定温度，酶分子将会展开。
• An enzyme that dissolves the fibrin of blood clots.能溶解血凝块中的纤维的酶。

10 cane   

n.手杖，细长的茎，藤条；v.以杖击，以藤编制的

参考例句：

• This sugar cane is quite a sweet and juicy.这甘蔗既甜又多汁。
• English schoolmasters used to cane the boys as a punishment.英国小学老师过去常用教鞭打男学生作为惩罚。

11 endorses   

v.赞同( endorse的第三人称单数 )；在（尤指支票的）背面签字；在（文件的）背面写评论；在广告上说本人使用并赞同某产品

参考例句：

• There isn't one country in the Middle East that now endorses the Eisenhower Doctrine. 但至今没有一个中东国家认可它。 来自辞典例句
• Whether any of this truly endorses Dr Patel's hypothesis is moot. 这些视频能否真正证明帕特的假设成立还是个未知数。 来自互联网

12 lasting   

adj.永久的，永恒的；vbl.持续，维持

参考例句：

• The lasting war debased the value of the dollar.持久的战争使美元贬值。
• We hope for a lasting settlement of all these troubles.我们希望这些纠纷能获得永久的解决。

13 speculations   

n.投机买卖( speculation的名词复数 )；思考；投机活动；推断

参考例句：

• Your speculations were all quite close to the truth. 你的揣测都很接近于事实。 来自《现代英汉综合大词典》
• This possibility gives rise to interesting speculations. 这种可能性引起了有趣的推测。 来自《用法词典》

14 analysts   

分析家，化验员( analyst的名词复数 )

参考例句：

• City analysts forecast huge profits this year. 伦敦金融分析家预测今年的利润非常丰厚。
• I was impressed by the high calibre of the researchers and analysts. 研究人员和分析人员的高素质给我留下了深刻印象。

15 promising   

adj.有希望的，有前途的

参考例句：

• The results of the experiments are very promising.实验的结果充满了希望。
• We're trying to bring along one or two promising young swimmers.我们正设法培养出一两名有前途的年轻游泳选手。