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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 disorder for which no cure has been found. The primary cause of CMT is duplication of a single gene,
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 cascade of bad things ensues "downstream" from that mutation.
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According to Pharnext, its Phase III results (which have not yet been peer-reviewed)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 stabilizing under PXT3003 but also being reversed, as cells began regenerating.
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 statistically 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 ailment.
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 promising 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 dependence; and sorbitol, a glucose 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 odds 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 consultant 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 physiology or medicine who is an adviser 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。”
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