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实验性药物可治疗乳糜泻患者因麸质引起的肠道损伤Experimental drug treats gluten-caused intestinal damage in celiac patients

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资料照片:2017年12月13日,法国图卢兹附近的科洛米耶,三明治旁展示着一张无麸质标签。路透社/雷吉斯·迪维尼奥/资料照片File Photo: A gluten-free sticker is displayed near sandwiches in Colomiers near Toulouse, France, December 13, 2017. REUTERS/Regis Duvignau/File Photo

今天我们为患有乳糜泻相关麸质问题的人们带来了一些令人鼓舞的消息。我们还报道了似乎推翻大麻作为睡眠增强剂作用的研究,以及人工智能可能帮助人们监测和更好地理解各种健康状况的另一种方式。

Today we feature some promising news for people dealing with gluten issues from celiac disease. We also report on studies that appear to debunk cannabis as a sleep enhancer, and another way in which artificial intelligence could help people monitor and better understand various health conditions.

根据一项小规模早期临床试验的结果,First Tracks Biotherapeutics(TRAX.O)正在研发的一种药物不仅减轻了乳糜泻患者因麸质暴露引起的症状,还展示了对麸质导致的肠道损伤的保护作用。

A drug being developed by First Tracks Biotherapeutics (TRAX.O) not only reduced symptoms from gluten exposure in patients with celiac disease but also demonstrated protection against gluten-induced intestinal damage, according to results of a small early-stage trial.

这项涉及32名患者的研究中,参与者至少在一年前被诊断出患有乳糜泻,并一直遵循无麸质饮食,但活检仍显示其肠道存在中度损伤。

Participants in the 32-patient study had been diagnosed with celiac disease at least a year prior and were following a gluten-free diet, but still had moderate damage to their intestines as seen on biopsies.

研究人员指出,即使是微量的麸质暴露,也可能在总体上遵循无麸质饮食的情况下引起肠道损伤和衰弱症状。

Even trace exposure to gluten can cause intestinal damage and debilitating symptoms despite general adherence to a gluten-free diet, the researchers noted.

试验开始时、第2周和第4周,参与者接受了First Tracks的ANB033药物或安慰剂的三次皮下注射,随后连续两周每天摄入6克麸质,大约相当于两片面包的量。

Patients were given three subcutaneous injections of First Tracks' ANB033 or a placebo at the start of the trial, at week 2 and week 4, followed by two weeks of daily consumption of 6 grams of gluten, or about the equivalent of two slices of bread.

根据公司的说法,研究结束时进行的第二组活检显示,接受药物治疗的患者相比接受安慰剂的患者,在防止肠道损伤方面具有统计学显著的保护作用。

A second set of biopsies done at the end of the study showed patients who received the drug had statistically significant protection against intestinal damage compared with those who got a placebo, according to the company.

此外,研究人员发现,ANB033在整个麸质挑战过程中防止了症状的增加,而安慰剂组患者则出现了症状的明显增加。

Furthermore, ANB033 prevented an increase in symptoms throughout the entire gluten challenge, whereas patients in the placebo group experienced a marked increase in symptoms, the researchers found.

只有6%接受ANB033治疗的患者需要使用许可药物来缓解其胃肠道症状,而安慰剂组这一比例高达47%。

Only 6% of ANB033 patients needed permitted medications to mitigate their gastrointestinal symptoms, compared with 47% of placebo patients.

研究负责人、纽约长老会/哥伦比亚大学欧文医学中心的本杰明·莱布沃尔博士在一份声明中说:“首次在同一项研究中看到对症状的统计学显著预防作用以及对(肠道)的益处,这非常了不起。”

“It is remarkable to see, for the first time, both statistically significant prevention of symptoms and (intestinal) benefit in the same study,” study leader Dr. Benjamin Lebwohl of New York-Presbyterian/Columbia University Irving Medical Center said in a statement.

莱布沃尔指出,ANB033抑制免疫细胞蛋白IL-15和IL-2,这两种蛋白是导致肠道损伤的炎症的关键驱动因素,其中IL-2与症状的发生密切相关。

ANB033 inhibits immune cell proteins IL-15 and IL-2, two key drivers of inflammation that damage the intestine, with IL-2 closely linked to symptom development, Lebwohl noted.

作为同一试验的一部分,研究人员正在评估第二组患者12周时的肠道愈合情况和症状严重程度,初步结果预计将于明年初公布。

As part of the same trial, the researchers are assessing intestinal healing and symptom severity at 12 weeks in a second group of patients, with initial results expected early next year.

他们预计将于下个月在澳大利亚墨尔本举行的国际乳糜泻研讨会上公布这项研究的完整结果。

They expect to present the full results of the study at the International Celiac Disease Symposium in Melbourne, Australia, next month.

最近有两项研究对 cannabis 有助于改善睡眠这一观点提出了质疑。

Two recent studies challenge the idea that cannabis helps to improve sleep.

在其中一项研究中,研究人员回顾了九项试验的数据,涉及537名失眠成人患者,他们被随机分配接受医用 cannabis、安慰剂或抗抑郁药治疗。

For one study, researchers reviewed data from nine trials involving 537 adults with insomnia who were randomly assigned to treatment with medical cannabis, a placebo or an antidepressant.

研究人员周二在《Cochrane系统评价数据库》上报告称,与安慰剂或抗抑郁药相比,医用 cannabis 在睡眠质量和满意度、总睡眠时间或日间功能方面几乎没有显示出具有临床意义的改善。

Medical cannabis showed little to no clinically meaningful improvement in sleep quality and satisfaction, total sleep time, or daytime functioning compared with placebo or the antidepressant, the researchers reported on Tuesday in The Cochrane Database of Systematic Reviews.

然而,研究人员指出,各项试验使用了不同的 cannabis 产品,没有一项试验对参与者的随访超过12周,而且由于可能存在偏倚和其他局限性,证据的总体质量和确定性被评为极低至低。

However, the various trials used different cannabis products, none of them followed participants for more than 12 weeks, and the overall quality and certainty of the evidence were rated very low to low because of possible bias and other limitations, the researchers noted.

他们说,因此,这些发现无法可靠地回答医用 cannabis 是否对失眠患者有帮助。

Therefore, the findings cannot reliably answer whether medical cannabis helps people with insomnia, they said.

该研究的负责人、阿根廷布宜诺斯艾利斯意大利大学医院的路易斯·加雷尼亚尼博士在一份声明中表示:“临床医生和患者不应将这些发现视为对医用大麻的认可或否定,而应认识到其中的不确定性,并在生成更好的证据的同时考虑个体情况。”

“Rather than viewing the findings as either an endorsement or a rejection of medical cannabis, clinicians and patients should recognize the uncertainty and consider individual circumstances while better evidence is generated,” study leader Dr. Luis Garegnani from Universidad Hospital Italiano de Buenos Aires, Argentina, said in a statement.

另一项对1266名参与者从青年期追踪至中年期的研究发现,长期使用大麻与腕戴式监测器所测得的睡眠质量较差有关。

A separate study that followed 1,266 participants from young adulthood into middle age found long-term cannabis use was linked to lower sleep quality as measured by wrist-worn monitors.

这些发表在《美国医学会杂志网络开放版》上的发现还表明,与大麻相关的睡眠紊乱可能不会被使用者本人察觉。

The findings, published in JAMA Network Open, also suggested that cannabis-related sleep disruptions may go unnoticed by users themselves.

该研究的负责人、西北大学范伯格医学院的阮清玄博士在一份声明中表示:“可穿戴设备检测到长期接触大麻的参与者在夜间有更多的睡眠中断,但参与者自己通常并未报告睡眠变差。”

“The wearable device detected more nighttime disruption among participants with long-term cannabis exposure, but the participants themselves generally did not report worse sleep,” study leader Dr. Thanh-Huyen T. Vu of Northwestern University Feinberg School of Medicine said in a statement.

她补充说:“这项研究挑战了人们普遍认为大麻有助于改善睡眠的观念。我们的发现表明,大麻实际上可能与较差的睡眠质量有关,即使人们并未察觉到这些变化。”

“This study challenges the common belief that cannabis helps people sleep better,” she added. “Our findings suggest cannabis may actually be associated with poorer sleep quality, even if people don't perceive those changes.”An AI platform allowed patients to “converse” with their medical devices in a pilot study of 13 patients, all with frequent seizures due to epilepsy who were undergoing continuous brain monitoring using electroencephalography, researchers reported on Tuesday.

研究人员周二报告称,在一项针对13名患者的初步研究中,一个人工智能平台让患者能够与其医疗设备“对话”。这些患者均因癫痫而频繁发作,正在接受使用脑电图的持续脑部监测。

Through a smartphone app, the patients could ask their monitoring equipment about their brain activity, request charts and other graphics showing trends in their data, and receive alerts or surveys throughout the day.

通过一款智能手机应用,患者可以向其监测设备询问自己的脑部活动情况,索取显示其数据趋势的图表和其他图形,并全天接收警报或调查问卷。

The device could contact the patient when it saw changes that might be of interest, and it could learn “on the fly” based upon patient responses.

当设备发现可能值得关注的变化时,它可以联系患者,并且可以根据患者的回应“即时”学习。

Although the study focused on epilepsy, the researchers say the platform could have broad applications for conditions that use cardiac devices, insulin pumps, and emerging treatments for neuropsychiatric disorders such as Parkinson’s disease, depression, obsessive-compulsive disorder, addiction, and cognitive disorders.

尽管这项研究聚焦于癫痫,但研究人员表示,该平台可能具有广泛的应用前景,可用于使用心脏设备、胰岛素泵的病症,以及帕金森病、抑郁症、强迫症、成瘾和认知障碍等神经精神疾病的新兴疗法。

“We envision medical devices becoming partners in care that learn alongside patients and help them make better decisions every day,” study leader Dr. Brian Litt of the University of Pennsylvania said in a statement.

“我们设想医疗设备成为护理伙伴,与患者共同学习,帮助他们每天做出更好的决定,”该研究的负责人、宾夕法尼亚大学的布莱恩·利特博士在一份声明中表示。

As reported in Nature Medicine, researchers analyzed 1,307 messages exchanged between patients and the system over an average of about five days, most involving discussions about seizure events, sleep, mood, energy levels, and other factors that can influence seizure activity.

据《自然·医学》报道,研究人员分析了患者与该系统在平均约五天时间内交换的1307条信息,其中大多数涉及癫痫发作事件、睡眠、情绪、精力水平以及其他可能影响癫痫活动的因素的讨论。

Several participants said the platform helped explain patterns they had previously observed but struggled to understand, such as associations of seizure episodes with emotions.

几位参与者表示,该平台帮助他们解释了此前已观察到但难以理解的模式,例如癫痫发作与情绪之间的关联。

Patients could also teach the system about their condition, noting which seizures the program correctly identified and which were false alarms, helping the system to improve its accuracy. The patients’ doctors could also converse with the system.

患者还可以教系统了解自己的病情,指出程序正确识别了哪些癫痫发作、哪些是误报,从而帮助系统提高准确性。患者的医生也可以与该系统对话。“患者往往了解传感器无法直接测量的疾病方面,”利特说。

“Patients often understand aspects of their disease that sensors cannot measure directly,” Litt said.

“想象一下,一台胰岛素泵能够回答某顿饭将如何影响血糖的问题,或者一台帕金森病设备能够根据患者在散步或写信时的感受来调整治疗,”他说。

“Imagine an insulin pump that can answer questions about how a meal will affect blood sugar, or a Parkinson’s device that adjusts treatment based on how a patient feels during a walk or writing a letter,” he said.