評論
打印
In October, Pharnext reported positive results for a Phase III trial in humans of one of its drug combinations.
在10月，Pharnext報告了其一種藥物組合的第三期人體試驗的積極成果。
The compound is PXT3003, a treatment for a neurodegenerative condition called Charcot-Marie-Tooth disease (CMT),
這種化合物名為PXT3003，用于治療神經退行性疾病——腓骨肌萎縮癥（CMT），
a rare disorder for which no cure has been found. The primary cause of CMT is duplication of a single gene,
一種尚未找到治療方法的罕見疾病。CMT的原發性原因是單個基因的復制，
but a whole cascade of bad things ensues "downstream" from that mutation.
但從這一突變的“下游”開始會發生一連串不好的事情。
Schwann cells, which protect nerves, regress into stem cells that don't do their job.
保護神經的施萬細胞退化成不工作的干細胞。
Axons in the nerves begin to die off. Muscles can't be controlled, and they shrink as a consequence.
神經中的軸突開始死亡。肌肉無法被控制，因此它們會萎縮。
According to Pharnext, its Phase III results (which have not yet been peer-reviewed)
根據Pharnext，其第三階段結果（還未經同行評審）表明，
showed CMT not merely stabilizing under PXT3003 but also being reversed, as cells began regenerating.
在PXT3003的影響下，CMT不僅趨于穩定，而且隨著細胞開始再生而逆轉。
Previous treatments, Cohen says, had managed only to slow patients' decline.
科恩表示，之前的治療只成功地減緩了患者病情的惡化。
Under PXT3003, patients showed statistically significant improvement on two measures of disability.
在PXT3003的影響下，病人在兩項殘疾指標上均有顯著改善。
Based on those results, in February the U.S. Food and Drug Administration granted Pharnext "fast track" status for that therapy—
基于這些結果，美國食品和藥物管理局于二月授予Pharnext的這個療法“快速通道”資格——
an accelerated review process, awarded only when the agency thinks a drug demonstrates “superior effectiveness” in treating a serious disease.
并加速了審核過程，只有在管理局認為這種藥物在治療某種重疾方面展現出“出眾的有效性”時，才會被授予這種嘉獎。
It is, to be sure, only one hopeful step against one rare ailment.
當然，這只是對抗一種罕見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.
但科技縮短了Pharnext的研發道路，帶來了有前途的長遠影響，縮短了藥物設計的時間。
Preclinical testing and clinical trials generally take eight to 10 years,
臨床前測試和臨床試驗通常需要8到10年，
and developing a novel drug completely from scratch can add seven years to the process, sometimes much more.
完全從零開始研發一種新藥需在此基礎上再加7年時間，有時甚至更長。
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幫助Pharnext選擇了三種現有的藥物進行再利用：
baclofen, a muscle relaxant; naltrexone, used to treat opioid dependence; and sorbitol, a glucose reduction used as a laxative.
巴氯芬，一種肌肉松弛劑，曾用于治療阿片類依賴；以及山梨糖醇，一種被用作瀉藥的降血糖藥。
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.
因為這些藥物使用穩定，所以Pharnext可以跳過確保藥物安全性的第一階段試驗——并取消“從頭開始構建”階段。
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盡快于2020年上市的幾率——并且它只是Pharnext眾多項目之一。
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,
該公司很快將開始對一種有阿爾茨海默癥適應癥的藥物進行第二階段試驗，并對肌萎縮側索硬化癥（ALS）療法進行第一次第二階段試驗，
in both cases using a similar repurposed combination.
兩種都使用了類似的再利用藥物組合。
Just as important: If these experiments succeed, copycats with deeper pockets could follow suit.
同樣重要的是：如果這些實驗成功，資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，是奧巴馬政府衛生與公眾服務部的部長，
who is now a consultant and board member for several health care companies, sees the efforts as part of a growing trend to repurpose.
現在是一名顧問兼幾家衛生保健公司的董事會成員，她將這些努力視為一種不斷增長的再利用趨勢的一部分。
"That all leads to the possibility of a lot shorter investment cycle, and potentially a very different pricing point,
“所有這些都可能導致投資周期大大縮短，定價點可能會非常不同，
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."
也為那些沒有足夠財務激勵的罕見疾病提供了多種可能性，”她說到?！斑@很有吸引力?！?/div>
Eric Kandel of the Kavli Institute for Brain Science at Columbia University,
哥倫比亞大學Kavli 腦科學研究所的Eric Kandel
a winner of the Nobel Prize in physiology or medicine who is an adviser to Pharnext,
是諾貝爾生理學、醫學獎得主，同時也是Pharnext的一名顧問，
says that the startup is at the leading edge of the trend, calling its methodology "both original and powerful."
他表示，這家初創公司處于這一趨勢的前沿，并稱其研究方法“既新穎又強大”。
As for whether that approach will catch on widely, Kandel adds, "We should know soon."
至于這種方法是否會廣泛流行，Kandel補充道，“我們應該很快就能知道了?！?/p>
譯文由可可原創，僅供學習交流使用，未經許可請勿轉載。
