财富杂志 AI如何让老药新用?(2)

In October, Pharnext reported positive results for a Phase III trial in humans of one of its drug combinations.

在10月，Pharnext报告了其一种药物组合的第三期人体试验的积极成果。

The compound is PXT3003, a treatment for a neurodegenerative condition called Charcot-Marie-Tooth disease (CMT),

这种化合物名为PXT3003，用于治疗神经退行性疾病——腓骨肌萎缩症（CMT），

a rare disorder² for which no cure has been found. The primary cause of CMT is duplication of a single gene¹,

一种尚未找到治疗方法的罕见疾病。CMT的原发性原因是单个基因的复制，

but a whole cascade³ of bad things ensues "downstream" from that mutation⁴.

但从这一突变的“下游”开始会发生一连串不好的事情。

Schwann cells, which protect nerves, regress into stem cells that don't do their job.

保护神经的施万细胞退化成不工作的干细胞。

Axons in the nerves begin to die off. Muscles can't be controlled, and they shrink as a consequence.

神经中的轴突开始死亡。肌肉无法被控制，因此它们会萎缩。

According to Pharnext, its Phase III results (which have not yet been peer-reviewed)

根据Pharnext，其第三阶段结果（还未经同行评审）表明，

showed CMT not merely stabilizing⁵ under PXT3003 but also being reversed, as cells began regenerating⁶.

在PXT3003的影响下，CMT不仅趋于稳定，而且随着细胞开始再生而逆转。

Previous treatments, Cohen says, had managed only to slow patients' decline.

科恩表示，之前的治疗只成功地减缓了患者病情的恶化。

Under PXT3003, patients showed statistically⁷ significant improvement on two measures of disability.

在PXT3003的影响下，病人在两项残疾指标上均有显著改善。

Based on those results, in February the U.S. Food and Drug Administration granted Pharnext "fast track" status for that therapy—

基于这些结果，美国食品和药物管理局于二月授予Pharnext的这个疗法“快速通道”资格——

an accelerated review process, awarded only when the agency thinks a drug demonstrates “superior effectiveness” in treating a serious disease.

并加速了审核过程，只有在管理局认为这种药物在治疗某种重疾方面展现出“出众的有效性”时，才会被授予这种嘉奖。

It is, to be sure, only one hopeful step against one rare ailment⁸.

当然，这只是对抗一种罕见疾病的有希望的一步。

Still, technology has shortened Pharnext's path in ways with promising⁹ long-term implications, shaving years off the drug-design timeline.

但科技缩短了Pharnext的研发道路，带来了有前途的长远影响，缩短了药物设计的时间。

Preclinical testing and clinical trials generally take eight to 10 years,

临床前测试和临床试验通常需要8到10年，

and developing a novel drug completely from scratch can add seven years to the process, sometimes much more.

完全从零开始研发一种新药需在此基础上再加7年时间，有时甚至更长。

In the case of PXT3003, in contrast, A.I. helped Pharnext select three existing drugs to repurpose:

相比之下，在PXT3003的例子中，AI帮助Pharnext选择了三种现有的药物进行再利用：

baclofen, a muscle relaxant; naltrexone, used to treat opioid dependence¹⁰; and sorbitol, a glucose¹¹ reduction used as a laxative.

巴氯芬，一种肌肉松弛剂，曾用于治疗阿片类依赖；以及山梨糖醇，一种被用作泻药的降血糖药。

Because the drugs were already in use, Pharnext could skip the Phase I trials normally required to ensure their safety—and eliminate the "build from scratch" stage.

因为这些药物使用稳定，所以Pharnext可以跳过确保药物安全性的第一阶段试验——并取消“从头开始构建”阶段。

FDA fast-tracking increases the odds¹² that PXT3003 could be on the market as soon as 2020—and it's only one of Pharnext's many projects.

FDA快速通道增加了PXT3003尽快于2020年上市的几率——并且它只是Pharnext众多项目之一。

The company will soon begin a second Phase II trial of a drug with indications for Alzheimer's and a first Phase II trial for an ALS therapy,

该公司很快将开始对一种有阿尔茨海默症适应症的药物进行第二阶段试验，并对肌萎缩侧索硬化症（ALS）疗法进行第一次第二阶段试验，

in both cases using a similar repurposed combination.

两种都使用了类似的再利用药物组合。

Just as important: If these experiments succeed, copycats with deeper pockets could follow suit.

同样重要的是：如果这些实验成功，资金雄厚的模仿者也可以效仿。

Kathleen Sebelius, a secretary of Health and Human Services in the Obama administration

Kathleen Sebelius，是奥巴马政府卫生与公众服务部的部长，

who is now a consultant¹³ and board member for several health care companies, sees the efforts as part of a growing trend to repurpose.

现在是一名顾问兼几家卫生保健公司的董事会成员，她将这些努力视为一种不断增长的再利用趋势的一部分。

"That all leads to the possibility of a lot shorter investment cycle, and potentially a very different pricing point,

“所有这些都可能导致投资周期大大缩短，定价点可能会非常不同，

and lots of possibilities for rare diseases where there just hasn't been enough of a financial incentive," she says. "And that has lots of appeal."

也为那些没有足够财务激励的罕见疾病提供了多种可能性，”她说到。“这很有吸引力。”

Eric Kandel of the Kavli Institute for Brain Science at Columbia University,

哥伦比亚大学Kavli 脑科学研究所的Eric Kandel

a winner of the Nobel Prize in physiology¹⁴ or medicine who is an adviser¹⁵ to Pharnext,

是诺贝尔生理学、医学奖得主，同时也是Pharnext的一名顾问，

says that the startup is at the leading edge of the trend, calling its methodology "both original and powerful."

他表示，这家初创公司处于这一趋势的前沿，并称其研究方法“既新颖又强大”。

As for whether that approach will catch on widely, Kandel adds, "We should know soon."

至于这种方法是否会广泛流行，Kandel补充道，“我们应该很快就能知道了。”

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 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。

3 cascade   

n.小瀑布，喷流；层叠；vi.成瀑布落下

参考例句：

• She watched the magnificent waterfall cascade down the mountainside.她看着壮观的瀑布从山坡上倾泻而下。
• Her hair fell over her shoulders in a cascade of curls.她的卷发像瀑布一样垂在肩上。

4 mutation   

n.变化，变异，转变

参考例句：

• People who have this mutation need less sleep than others.有这种突变的人需要的睡眠比其他人少。
• So far the discussion has centered entirely around mutation in the strict sense.到目前为止，严格来讲，讨论完全集中于围绕突变问题上。

5 stabilizing   

n.稳定化处理[退火]v.（使）稳定， （使）稳固( stabilize的现在分词 )

参考例句：

• The disulfide bridges might then be viewed primarily as stabilizing components. 二硫桥可以被看作是初级的稳定因素。 来自辞典例句
• These stabilizing design changes are usually not desirable for steady-state operation. 这些增加稳定性的设计改变通常不太符合稳态工作的要求。 来自辞典例句

6 regenerating   

v.新生，再生( regenerate的现在分词 )；正反馈

参考例句：

• It is not proposed to deal with the detailed histology of regenerating tissues here. 这里未提出详细的再生组织的组织学。 来自辞典例句
• This is accomplished by using a thermocompressor to recycle regenerating steam through the absorber. 它用热压机使再生蒸汽经吸附器循环完成解吸过程。 来自辞典例句

7 statistically   

ad.根据统计数据来看，从统计学的观点来看

参考例句：

• The sample of building permits is larger and therefore, statistically satisfying. 建筑许可数的样本比较大，所以统计数据更令人满意。
• The results of each test would have to be statistically independent. 每次试验的结果在统计上必须是独立的。

8 ailment   

n.疾病，小病

参考例句：

• I don't have even the slightest ailment.我什么毛病也没有。
• He got timely treatment for his ailment.他的病得到了及时治疗。

9 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。

10 dependence   

n.依靠，依赖；信任，信赖；隶属

参考例句：

• Doctors keep trying to break her dependence of the drug.医生们尽力使她戒除毒瘾。
• He was freed from financial dependence on his parents.他在经济上摆脱了对父母的依赖。

11 glucose   

n.葡萄糖

参考例句：

• I gave him an extra dose of glucose to pep him up.我给他多注射了一剂葡萄糖以增强他的活力。
• The doctor injected glucose into his patient's veins.医生将葡萄糖注入病人的静脉。

12 odds   

n.让步，机率，可能性，比率；胜败优劣之别

参考例句：

• The odds are 5 to 1 that she will win.她获胜的机会是五比一。
• Do you know the odds of winning the lottery once?你知道赢得一次彩票的几率多大吗？

13 consultant   

n.顾问；会诊医师，专科医生

参考例句：

• He is a consultant on law affairs to the mayor.他是市长的一个法律顾问。
• Originally,Gar had agreed to come up as a consultant.原来，加尔只答应来充当我们的顾问。

14 physiology   

n.生理学，生理机能

参考例句：

• He bought a book about physiology.他买了一本生理学方面的书。
• He was awarded the Nobel Prize for achievements in physiology.他因生理学方面的建树而被授予诺贝尔奖。

15 adviser   

n.劝告者，顾问

参考例句：

• They employed me as an adviser.他们聘请我当顾问。
• Our department has engaged a foreign teacher as phonetic adviser.我们系已经聘请了一位外籍老师作为语音顾问。