美国国家公共电台 NPR Advice From Patients On A Study's Design Makes For Better Science

 

AUDIE CORNISH, HOST:

Many studies of new drugs simply languish¹. They don't attract enough patients, and they aren't completed. That slows medical progress. Well, here's a counterexample. So many volunteers signed up for a federally funded trial of unproven cancer treatments that researchers had to put the brakes on it temporarily. NPR's Richard Harris takes a close look at what makes this study so attractive.

RICHARD HARRIS, BYLINE²: The study is called the NCI-MATCH trial, and as its leader, Robert Comis explains it discards the normal way of classifying cancers based on the organ where they originate.

ROBERT COMIS: So instead of thinking of a breast cancer treatment or a lung cancer treatment or colon³, it looks at the different mutations that occur in the tumors.

HARRIS: The study recruits people who have tried and failed traditional cancer treatments, people like 74-year-old Nancy Nahmias.

NANCY NAHMIAS: It all started actually when I was diagnosed with the cancer of the liver. I was put on chemo, which I reacted very poorly to, and I spent six weeks in the hospital.

HARRIS: Her daughter, a physician, learned about the NCI-MATCH trial and encouraged her to give it a try. Scientists screened the genetic⁶ pattern of her tumor⁵ and found a mutation⁴ that might be amenable⁷ to a treatment not usually given to liver cancer patients. Nahmias signed up about two months ago at Thomas Jefferson University, one of the many sites running the study. It's too early to say whether it's working, but it's not hurting.

NAHMIAS: No side effects whatsoever⁸. I get tired very easily, but that was before I started taking this drug.

HARRIS: The MATCH study recruited patients at a record pace. In its first three months, it had enrolled⁹ 800 patients, far more than the 150 the researchers had expected. They paused the study briefly¹⁰ to catch up with the lab work. That rapid clip is no doubt because the study is aimed at people like Nancy Nahmias who are running out of traditional options. But it's also because the people who designed the study stopped to ask what would appeal to potential participants. Nancy Roach, a longtime patient advocate, got involved with the medical researchers planning the study early on.

NANCY ROACH: This is going to sound goofy, but my dad was in advertising¹¹. So you know the Scrubbing Bubbles, the Dow Scrubbing Bubbles? That was my dad, right? So I grew up watching commercials and thinking about what consumers wanted.

HARRIS: She brought that sensibility to the conferences where the NCI-MATCH trial was being planned. Dr. Peter O'Dwyer, who was in on those conversations, recalls that the original design would have split patients who seemed to be doing well into two groups.

PETER O'DWYER: And in one, continue the treatment, and in the other group, take them off the treatment for what was called a drug holiday.

HARRIS: Roach remembers her immediate¹² reaction to that design.

ROACH: Taking a patient who's responding to treatment, taking them off treatment - that is not going to fly.

HARRIS: She correctly anticipated how patients like Nancy Nahmias would have reacted as they thought about whether to sign up for the trial.

NAHMIAS: I would not have liked that.

HARRIS: Why so?

NAHMIAS: I mean if it seems to be working, let's face it; I don't want to do anything to sabotage¹³ myself.

O'DWYER: The design had certain attractions, but it clearly had certain flaws.

HARRIS: O'Dwyer says on the one hand, the drug holiday would help doctors tell whether a tumor was just growing slowly or actually responding to treatment. But on the other hand, they heard the argument from Nancy Roach and others in the patient advisory¹⁴ group.

O'DWYER: We all agreed and changed the design of the study accordingly.

HARRIS: So how do you get that information without having the so-called drug holiday then?

O'DWYER: You don't.

HARRIS: That would come out in a follow-up study. Comis says time was when researchers designed studies without any patient input¹⁵ back in the days when patients tended not to question their physicians to begin with. But as patients have gotten more involved in their own care, so too have their advocates become more involved in the technical discussions of study design.

COMIS: That has increasingly become the norm in the development of clinical trials.

HARRIS: Comis and O'Dwyer work together in Philadelphia at the ECOG-ACRIN Cancer Research Group. Years ago, Comis was involved in a landmark¹⁶ study which put patient cooperation to the test. Back in the 1990s, doctors were increasingly encouraging breast cancer patients to undergo very aggressive treatment involving bone marrow¹⁷ transplants. That advice was based on poor evidence, so Comis wanted to run a rigorous trial to see if that really worked.

COMIS: And we struggled throughout the '90s to put enough patients on clinical trials, which ultimately showed that it didn't work.

HARRIS: Comis recalls that patients and their advocates as well as doctors really didn't want to question the prevailing¹⁸ wisdom about bone marrow transplants.

COMIS: And I think that one of the reasons that some of those early trials took so long was because the whole external environment was against participation¹⁹ in these particular trials.

HARRIS: That experience validated²⁰ his view that patient advocates are central to doing good research. From Nancy Roach's perspective, it takes a bit of nerve to speak up in a room of doctors and scientists and ask basic questions like, will the results of this study actually help anybody?

ROACH: And that's what I do. I'm not a scientist. I'm not a clinician. I'm there on behalf of patients.

HARRIS: Her own journey started when her mother-in-law developed colorectal cancer. Roach went from being an advocate for one patient to an advocate for many. And while she's gratified to see that more and more people are stepping into that role, she notes that most are white and economically advantaged.

ROACH: Honestly, most of them look like me, and that's a problem, you know? That's really a problem.

HARRIS: African-Americans are underrepresented in clinical trials. That may be because their doctors aren't so likely to suggest them, or it may be that the trials aren't attuned²¹ to their particular needs. Roach hopes this will be the next frontier for patient involvement. Richard Harris, NPR News.

1 languish   

vi.变得衰弱无力，失去活力，（植物等）凋萎

参考例句：

• Without the founder's drive and direction,the company gradually languished.没有了创始人的斗志与指引，公司逐渐走向没落。
• New products languish on the drawing board.新产品在计划阶段即告失败。

2 byline   

n.署名；v.署名

参考例句：

• His byline was absent as well.他的署名也不见了。
• We wish to thank the author of this article which carries no byline.我们要感谢这篇文章的那位没有署名的作者。

3 colon   

n.冒号，结肠，直肠

参考例句：

• Here,too,the colon must be followed by a dash.这里也是一样，应当在冒号后加破折号。
• The colon is the locus of a large concentration of bacteria.结肠是大浓度的细菌所在地。

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 tumor   

n.（肿）瘤，肿块(英)tumour

参考例句：

• He was died of a malignant tumor.他死于恶性肿瘤。
• The surgeons irradiated the tumor.外科医生用X射线照射那个肿瘤。

6 genetic   

adj.遗传的，遗传学的

参考例句：

• It's very difficult to treat genetic diseases.遗传性疾病治疗起来很困难。
• Each daughter cell can receive a full complement of the genetic information.每个子细胞可以收到遗传信息的一个完全补偿物。

7 amenable   

adj.经得起检验的；顺从的；对负有义务的

参考例句：

• His scientific discoveries are amenable to the laws of physics.他在科学上的发现经得起物理定律的检验。
• He is amenable to counsel.他这人听劝。

8 whatsoever   

adv.（用于否定句中以加强语气）任何；pron.无论什么

参考例句：

• There's no reason whatsoever to turn down this suggestion.没有任何理由拒绝这个建议。
• All things whatsoever ye would that men should do to you,do ye even so to them.你想别人对你怎样，你就怎样对人。

9 enrolled   

adj.入学登记了的v.[亦作enrol]( enroll的过去式和过去分词 )；登记，招收，使入伍（或入会、入学等），参加，成为成员；记入名册；卷起，包起

参考例句：

• They have been studying hard from the moment they enrolled. 从入学时起，他们就一直努力学习。 来自《简明英汉词典》
• He enrolled with an employment agency for a teaching position. 他在职业介绍所登了记以谋求一个教师的职位。 来自《简明英汉词典》

10 briefly   

adv.简单地，简短地

参考例句：

• I want to touch briefly on another aspect of the problem.我想简单地谈一下这个问题的另一方面。
• He was kidnapped and briefly detained by a terrorist group.他被一个恐怖组织绑架并短暂拘禁。

11 advertising   

n.广告业；广告活动 a.广告的；广告业务的

参考例句：

• Can you give me any advice on getting into advertising? 你能指点我如何涉足广告业吗?
• The advertising campaign is aimed primarily at young people. 这个广告宣传运动主要是针对年轻人的。

12 immediate   

adj.立即的；直接的，最接近的；紧靠的

参考例句：

• His immediate neighbours felt it their duty to call.他的近邻认为他们有责任去拜访。
• We declared ourselves for the immediate convocation of the meeting.我们主张立即召开这个会议。

13 sabotage   

n.怠工，破坏活动，破坏；v.从事破坏活动，妨害，破坏

参考例句：

• They tried to sabotage my birthday party.他们企图破坏我的生日晚会。
• The fire at the factory was caused by sabotage.那家工厂的火灾是有人蓄意破坏引起的。

14 advisory   

adj.劝告的，忠告的，顾问的，提供咨询

参考例句：

• I have worked in an advisory capacity with many hospitals.我曾在多家医院做过顾问工作。
• He was appointed to the advisory committee last month.他上个月获任命为顾问委员会委员。

15 input   

n.输入(物)；投入；vt.把(数据等)输入计算机

参考例句：

• I will forever be grateful for his considerable input.我将永远感激他的大量投入。
• All this information had to be input onto the computer.所有这些信息都必须输入计算机。

16 landmark   

n.陆标，划时代的事，地界标

参考例句：

• The Russian Revolution represents a landmark in world history.俄国革命是世界历史上的一个里程碑。
• The tower was once a landmark for ships.这座塔曾是船只的陆标。

17 marrow   

n.骨髓；精华；活力

参考例句：

• It was so cold that he felt frozen to the marrow. 天气太冷了，他感到寒冷刺骨。
• He was tired to the marrow of his bones.他真是累得筋疲力尽了。

18 prevailing   

adj.盛行的；占优势的；主要的

参考例句：

• She wears a fashionable hair style prevailing in the city.她的发型是这个城市流行的款式。
• This reflects attitudes and values prevailing in society.这反映了社会上盛行的态度和价值观。

19 participation   

n.参与，参加，分享

参考例句：

• Some of the magic tricks called for audience participation.有些魔术要求有观众的参与。
• The scheme aims to encourage increased participation in sporting activities.这个方案旨在鼓励大众更多地参与体育活动。

20 validated   

v.证实( validate的过去式和过去分词 )；确证；使生效；使有法律效力

参考例句：

• Time validated our suspicion. 时间证实了我们的怀疑。 来自《现代英汉综合大词典》
• The decade of history since 1927 had richly validated their thesis. 1927年以来的十年的历史，充分证明了他们的论点。 来自辞典例句

21 attuned   

v.使协调( attune的过去式和过去分词 )；调音

参考例句：

• She wasn't yet attuned to her baby's needs. 她还没有熟悉她宝宝的需要。
• Women attuned to sensitive men found Vincent Lord attractive. 偏爱敏感男子的女人，觉得文森特·洛德具有魅力。 来自辞典例句