新药可能让斑秃患者毛发再生

The first thing Brian H. noticed was that he could grow a real beard. It had been years since that had been possible, years he spent bedeviled by hair loss on his head, face, arms and legs.

布莱恩·H(Brian H.)注意到的第一件事情是，他可以长出真正的胡须来。在这件事成为可能之前，他已经被头上、脸上以及四肢上毛发的脱落困扰了很多年。

Brian, 34, who asked that his last name be withheld to protect his privacy, suffers from alopecia areata, an autoimmune disease afflicting about 1 percent of men and women, causing hair to fall out, often all over the body. He believes that the “mangy patches” of baldness that have plagued him since his 20s have cost him jobs and relationships.

34岁的布莱恩患有斑秃，为保护隐私起见，他要求隐去姓氏。斑秃是一种自身免疫性疾病，在人群中的发病率约为1%，通常会造成全身各处毛发的脱落。从20多岁开始，布莱恩一直被那些“癞癞巴巴的秃斑”折磨着。他认为它们严重影响了他的工作和人际关系。

After trying various treatments, Brian enrolled this year in a study at Columbia University Medical Center testing whether a drug approved for a bone marrow disorder could help people with alopecia. One of the study’s leaders, Angela Christiano, is a dermatology professor and geneticist who herself has alopecia areata.

今年，曾尝试过各种疗法的布莱恩，在哥伦比亚大学医疗中心(Columbia University Medical Center)报名参加了一项旨在测试一种获批治疗骨髓疾病的药物能否帮人摆脱斑秃的研究。该研究的负责人之一安吉拉·克里斯蒂雅诺(Angela Christiano)是皮肤科教授和遗传学专家，同时也是一名斑秃患者。

After successfully testing on mice two drugs from a new class of medicines called JAK inhibitors, which suppress immune system activity by blocking certain enzymes, the researchers began testing one of the drugs, ruxolitinib, on seven women and five men. Some of their findings were published Sunday in the journal Nature Medicine.

研究人员先拿两种新型JAK抑制剂类药物在老鼠身上做了测试——JAK抑制剂可以通过阻断特定种类的酶来抑制免疫系统的活动——随后让参与人体测试的7位女性和5位男性使用了其中一种名叫鲁索利替尼(Ruxolitinib)的药物。周日，他们的部分研究成果被发表在了《自然医学》(Nature Medicine)杂志上。

The results for Brian and several other participants have been significant.

布莱恩和其他几个参与者的病情有了很大的起色。

“Pretty quickly, there were sort of fringes,” Brian said. Then “three or four large areas started to show hair growth,” and by five months, he had plenty of hair on his head, arms, and even his back. “I was blown away,” he said.

“很快就看到了丝须状的东西，”布莱恩说。接着“有三四个地方开始长出毛发”，到五个月的时候，他的头上、胳膊上甚至后背上都有了大量毛发。“我当时觉得难以置信，”他说。

The disease differs from other types of hair loss, including male pattern baldness, and there is no evidence the drug will work for those conditions. Experts caution that even for alopecia areata, it is too early to know if the treatment will work for most patients and if there are significant side effects or safety concerns.

斑秃不同于包括男性型秃发在内的其他类型的脱发，没有证据证明这种药物对后者也有疗效。专家们提醒说，即便就斑秃而言，目前也远远不能确定该疗法是否适用于大多数患者，以及是否存在较大的副作用或安全隐患。

The study is continuing, but so far a few participants did not regrow hair, said Dr. Julian Mackay-Wiggan, director of Columbia’s dermatology clinical research unit and an author of the study.

这项研究的发起者之一、哥伦比亚大学皮肤病学专业临床研究部主任朱利安·麦凯-维根(Julian Mackay-Wiggan)博士表示，研究仍在继续，但到目前为止，还有几个参与者并没重新长出毛发。

“It appears to work — not in everyone, but in the majority,” she said. “We need a lot more data on the long-term risks in healthy individuals. But it’s certainly very exciting in terms of hair growth. It was surprising how quickly and impressively the growth occurred.”

“它似乎对大部分人有效，但并非在每个人身上都能起作用，”她说。“我们还需要获得更多与健康人群长期使用该药物的风险有关的数据。但在促进毛发生长方面，其效果确实令人振奋。这种生长来得如此快速和明显，简直出人意料。”

Dr. Luis Garza, a dermatologist at Johns Hopkins Hospital who was not involved in the research, said the results were encouraging enough that he would consider prescribing ruxolitinib to patients who could not be treated with other methods and who understood potential side effects.

约翰·霍普金斯医院(Johns Hopkins Hospital)的皮肤科医生路易斯·加尔萨(Luis Garza)说，鲁索利替尼疗效可喜，他会考虑给那些不能用其他方法治疗，且对其潜在副作用有所了解的病人开鲁索利替尼。加尔萨未参与该项研究。

Cortisone injections often work for patients with isolated patches of baldness, but they must be done regularly and are painful. For patients with severe baldness, “it’s impossible to inject their whole scalp,” he said.

对单纯性斑秃而言，注射可的松(Cortisone )通常是有效的，但注射必须定期进行，而且会给患者带来痛苦。他说，有的患者秃得厉害，“总不可能给整张头皮都注射药物”。

“There’s a major need for improving the treatment,” he added. “It’s not ludicrous to try on a patient.”

“这项疗法的改进是有巨大需求的，”他补充道，“在患者身上进行试验并不过分。”

But Dr. George Cotsarelis, a dermatologist at the University of Pennsylvania, urged caution until further research is conducted. He said it makes sense that drugs suppressing immune system activity would work for a disorder caused by an overly active immune reaction.

不过，宾夕法尼亚大学(Pennsylvania)的皮肤病专家乔治·柯萨莱利斯(George Cotsarelis)博士，极力主张在进行更深入的研究之前保持谨慎。他表示，抑制免疫系统活动的药物对因免疫反应过度灵敏而引发的疾病有效，这不出奇。

But because patients in the study received twice-daily pills that circulated ruxolitinib throughout their bodies, rather than topical cream, he said they were “treated systemically with a very toxic drug” that can cause liver and blood problems, infections and other ailments.

但由于参与这项研究的患者每天两次服用鲁索利替尼片剂——而不是涂抹膏剂——药物成分会在整个身体内循环，他认为他们在接受以毒性很强的药物为手段的全身性治疗，该药物有可能让肝脏和血液出问题，还有可能引发感染以及其他疾病。

Although the patients have experienced few side effects, the study is small and not a randomized trial comparing ruxolitinib to other treatments.

尽管患者几乎还没感受到什么副作用，但这项研究规模很小，也并非一次拿鲁索利替尼和其他治疗手段作对比的随机试验。

If ruxolitinib could be applied topically, Dr. Cotsarelis said, “this would be an amazing breakthrough.” Until then, “patients are going to rush in demanding this treatment, and I would not give it.”

柯萨莱利斯博士说，假如可以局部应用鲁索利替尼，“那将是惊人的突破”。在此之前，“要求用该疗法治疗的患者未免显得草率，我是不会给他们开处方的”。

Dr. Raphael Clynes, a co-leader of the research while he was a Columbia professor (he now works for Bristol-Myers Squibb), said the team tested cream and pills on mice, and planned to test a cream on people.

必治妥施贵宝公司(Bristol-Myers Squibb)的拉斐尔·克莱因 (Raphael Clyne)博士在哥伦比亚大学当教授期间是这项研究的负责人之一，他表示，研究团队拿老鼠做测试时用过膏剂和片剂，并且打算进行膏剂的人体测试。

So far, he considered ruxolitinib “an expensive therapy that’s probably effective based on the small number of patients that we’ve treated, and it’s likely to have a reasonable safety profile. But there’s no way that I can endorse it fully unless we do larger trial.”

他认为鲁索利替尼迄今为止还是“一种价格高昂的治疗手段。就我们治疗的为数不多的患者而言，它或许是有效的，在安全性方面的表现似乎也说得过去。但除非进行更大规模的测试，否则我绝不会完全认可这种疗法”。

The team also plans to test on people another JAK inhibitor, tofacitinib, which is approved for rheumatoid arthritis and grew hair on mice. In June, Dr. Brett King, a dermatologist at Yale, reported that tofacitinib caused full hair growth and no negative effects for a man with alopecia universalis, a variant involving almost total hair loss.

研究团队还打算对另外一种JAK抑制剂——托法替尼(Tofacitinib)——进行人体测试。这是一种获批治疗类风湿关节炎的药物，能让老鼠长出毛发。今年6月，耶鲁大学(Yale)皮肤病专家布莱特·金(Brett King)博士宣称，托法替尼(Tofacitinib)能促进男性普秃患者的毛发生长，且没有任何副作用。普秃是一种几乎可以造成全身毛发全部脱落的疾病。

For Brian, five months on the drug yielded a full head of hair. For unknown reasons, the new hair is white instead of black, its original color.

对布莱恩而言，服用鲁索利替尼五个月换来了满头的发丝。他原先的头发是黑色的，不知道为什么，新长出来的头发却是白色的。

Still, “It’s a lot easier to shrug that off than to pass the silent judgment of people” who he said he felt were staring at his bald splotches. He said side effects, including slight anemia, were minor.

但他说，“相比面对人们无声的成见，这样要好受多了”——他以前总感觉人们在打量他的秃斑。他还表示，包括轻微贫血在内的副作用是非常小的。