10桩出现可怕差错的临床试验(下)

5 Stem Cell Vision Treatment

5 视神经干细胞治疗

In January 2017, three women entered a study with their vision and left without it. Their ages ranging from 72 to 88, all three of these women suffered from macular degeneration, an eye disease closely related to old age. The patients each paid $5,000 to have both eyes treated with stem cell therapy, a process that was "both atypical and unsafe" according to several ophthalmology experts.

2017年1月，3名女性在她们参与的研究中丧失了视力。她们的年龄在72-88之间，患有 黄斑部变性，是一种老年性疾病。她们每人都交了5000美元的治疗费，接受了干细胞疗法，但据多个眼科专家所说，该疗法“既不典型又不安全”。

Just days after the procedure, all three women reported severe side effects, including bleeding and retinal detachment. One patient entirely lost her eyesight, while the other two lost most. None of the patients are expected to recover their sight. But scientists knew that this trial had flaws from the beginning. First and foremost, the patients were required to pay for their own procedures, which is a flagrant sign of illegitimate research. Additionally, medical professionals have tried to erase the history of the trial; when you visit government records of the trial online, it only says that the study was "withdrawn prior to enrollment," which clearly was not the case.

就在治疗几天后，3名女性都出现严重的副作用，包括出血及视网膜脱落。其中一个病人完全丧失了视力，其余两人大部分视力受损。她们恢复视力的可能性几乎没有。但是科学家从一开始就知道该试验存在缺陷，而且最重要的是，要求病人自费参与治疗的试验项目，通常都意味着是非法的。另外，医学专家们竭力抹去了这场试验的存在；如果你在网上查找该试验的官方记录，只会显示“注册前即取消”，但事实根本不是这样！

4 Leukemia CAR-T Trial

4 白血病的，嵌合抗原受体T细胞免疫疗法（CAR-T）

In July 2016, three adult leukemia patients died in a trial of a new cellular-level medicine. Nicknamed CAR-T, this new treatment option was supposed to attack the malignant cells until they appeared to have vanished. The technology was an up-and-coming phenomenon that many researchers called the "fifth pillar" of cancer treatment, but hopes were soon dashed by the results of the 2016 study.

2016年7月，3名成年白血病患者死于一场临床试验，该实验使用了一种新的细胞层面的药物，叫做CAR-T（嵌合抗原受体T细胞免疫疗法），新疗法的治疗理念是攻击恶性细胞直到完全消灭它们。这项技术非常具有前景，许多研究员称它为癌症治疗的“第五支柱”，但2016年的这场研究结果，彻底毁灭了治疗希望。

The cause of death for the three patients was swelling in the brain, medically known as cerebral edema. Representatives from the sponsoring medical company Juno admit that cerebral edema is rather common in patients who have been given CAR-T treatments, as are immune system reactions and increased neurological toxicity.

3名病人的死因为脑水肿。来自医药公司的赞助商——朱诺的代表们承认，在接受CAR-T治疗的病人中，脑水肿的发生比较普遍，因为是免疫系统的反应增加了神经毒性。

After the news of the deaths had been released, Juno's stock fell 27 percent. Their practices are currently under FDA review, and it is unclear whether they will be allowed to continue their studies.

在死亡事件曝光后，朱诺的股价跌了27%。他们的试验正在被FDA重新审查，目前尚不清楚他们的研究是否还能进行。

3 New York Lidocaine Disaster

3 纽约利多卡因灾难

In 1996, University of Rochester sophomore Nicole Wan needed some pocket money. So she decided to sign up, without her parents' permission, for a clinical trial that paid $150. The researchers inserted a tube down her throat and into her lungs to see the effects of pollution on her respiratory system, a common procedure called a bronchoscopy.

1996年，罗彻斯特大学的大二学生妮可·王需要一点零花钱，于是她瞒着父母参加了一场临床试验，以赚取150美元。研究员从她的喉咙插入一根管子，一直深入到肺部，以查看环境污染对她呼吸系统的影响，这是个很常见的步骤，叫做支气管镜检。

But what Nicole did not know was that they took far more cell samples than originally outlined in the proposal. And as they took more samples from her lungs, they increased the dose of her anesthetic, Lidocaine, far above the levels approved by the FDA. She was released feeling incredibly weak and in enormous amounts of pain, and two days later was found dead. An autopsy revealed that lethal levels of Lidocaine, due to malpractice in the study, had caused her heart to stop beating and the rest of her body to fail along with it.

但是妮可不知道的是他们取了很多肺部细胞样本，超出了一开始合同里规定的量。并且在他们取样的过程中，不停地增加对她使用利多卡因（一种局部麻醉剂）的剂量，远远大于FDA的安全剂量。她离开时非常虚弱，还承受着巨大的疼痛，在2天后，妮可就去世了。尸检显示体内残留致死剂量的利多卡因。由于在此研究中的玩忽职守，才导致妮可心脏停跳，其他身体器官随之衰竭。

2 John Hopkins Asthma Trial

2 约翰·霍普金斯医院哮喘试验

Ellen Roche, a technician at Johns Hopkins Hospital, volunteered to take part in an asthma trial for healthy individuals. The trial's goal was to discover what mechanism kept healthy people from developing the symptoms of asthma, so the doctors induced a mild asthmatic reaction and then treated it with hexamethonium.

艾伦·罗切是约翰·霍普金斯医院的一名技术员，自愿参加了一项针对健康人的哮喘试验。该试验的目的是找出健康人没有发生哮喘的机制。于是医生引发艾伦出现一点点哮喘的症状，并用六甲铵来治疗。

At first, inhaling this medicine simply caused Ms. Roche to develop a cough. But as time progressed, she was put on a ventilator as her lung tissue broke down and her kidneys began to fail. She died one month later, on June 2, 2001. Medical officials from the trial admit that the hexamethonium "was either solely responsible for the subject's illness or played an important contributory role." To make matters worse, participants learned after the trial that hexamethonium is not even an FDA-approved drug. This fact was not included in the consent form, so Johns Hopkins has been forced to take full responsibility for Roche's death.

一开始，吸入这种药只让罗切出现咳嗽症状。但随着时间的推移，她的肺组织遭到了破换，肾脏也出现了衰竭，不得不用呼吸机维持生命。她一个月后去世，就在2001年6月2号这一天。该试验的医学人员承认六甲铵“可能是导致研究对象生病的唯一因素，或至少在其中起了很大作用。”更糟糕的是，参与者事后才发现六甲铵根本不是FDA批准使用的药物。这件事在知情同意书中根本没有提到，所以，约翰·霍普金斯医院对罗切的死应负全责。

1 The Elephant Man Trial

1 象人试验

The most famous clinical trial of all time, the elephant man trial took place in London in 2006. The trial, which was testing a new cancer treatment called TGN1412, seemed harmless to the eight men who took part in it; medical professionals had assured them that the worst symptoms would only include a headache and nausea.

有史以来最出名的临床试验，应该算是2006年发生在伦敦的象人试验了。试验是为了检验一种叫TGN1412的新型抗癌药，该药看似对8个参与者没什么伤害；医学专家保证最坏的副作用只会有头疼和恶心。

But the results were much more gruesome than that. Shortly after they were given the doses, all of the patients began writhing in pain and vomiting. One of the participants lost his fingers and toes, while another had to have his foot partially amputated. The trial earned its nickname, "The Elephant Man Trial," because one participant's head swelled up so large that his girlfriend teased him about looking like an elephant.

但结果比这个可怕多了。给药不久，所有的病人都因剧痛出现扭动，并开始呕吐。其中一名参与者失去了他的手指和脚趾，另一个不得不把部分脚截肢。还有一个参与者的头出现严重肿胀，被他的女朋友嘲笑像一只大象，于是这个试验就有了外号——象人试验。

No one is completely sure what went wrong, but the patients have a few ideas. One suggests that the timing of the dosage made it dangerous; researchers spent 90 minutes slowly injecting animals with the drug, but took a mere six minutes to inject it into the human subjects. Another claims that the preliminary animal testing was inaccurate because instead of testing on a bonobo, whose DNA is a 98 percent match to humans, the agency cut costs and used a macaque, whose DNA is only a 94 percent match. These men may never know exactly what went wrong that fateful day, or how it will continue to affect their lives.

没有人能确定出错的原因，但参加的病人有几点想法。一个认为给药的时间造就了危险；研究员给动物用药时花了90分钟缓慢注射，但在人这，只用了6分钟就注射完了。另一位认为之前的动物预试验就是不准确的，因为他们没有用倭黑猩猩（DNA与人类有98%相似度）做试验，为了节省开支，研究机构使用了猕猴（DNA与人类仅有94%相似度）。这些人也许永远都不会知道那悲剧的一天到底哪出了问题，也不会知道有没有其他问题会继续影响他们未来的人生。