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药物不能减缓遗传性阿尔茨海默病的衰退

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2020年02月11日

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Drugs fail to slow decline in inherited Alzheimer’s disease

药物不能减缓遗传性阿尔茨海默病的衰退

Two experimental drugs failed to prevent or slow mental decline in a study of people who are virtually destined to develop Alzheimer’s disease at a relatively young age because they inherited rare gene flaws.

在一项研究中,两种实验性药物未能阻止或减缓智力衰退。这项研究的对象是那些实际上注定要在相对年轻的年龄患上阿尔茨海默病的人,因为他们继承了罕见的基因缺陷。

The results announced Monday are another disappointment for the approach that scientists have focused on for years — trying to remove a harmful protein that builds up in the brains of people with Alzheimer’s, the leading cause of dementia.

周一公布的结果是科学家们多年来一直关注的另一个令人失望的方法——试图去除一种在老年痴呆症患者大脑中积聚的有害蛋白质,老年痴呆症是导致老年痴呆的主要原因。

“We actually don’t even know yet what the drugs did” in term of removing that protein because those results are still being analyzed, said study leader Dr. Randall Bateman at Washington University in St. Louis, Missouri.

密苏里州圣路易斯市华盛顿大学的研究负责人兰德尔·贝特曼博士说:“我们实际上还不知道这些药物在去除蛋白质方面起了什么作用,因为这些结果还在分析中。”。

But after five years on average, the main goal of the study was not met — people on either of the drugs scored about the same on thinking and memory tests as others given placebo treatments.

但平均5年后,研究的主要目标没有达到——服用这两种药物的人在思维和记忆测试中的得分与服用安慰剂的人差不多。

药物不能减缓遗传性阿尔茨海默病的衰退

More than 5 million people in the United States and millions more worldwide have Alzheimer’s. Current drugs only temporarily ease symptoms and do not alter the course of the disease.

在美国有超过500万人和全世界有数百万人患有老年痴呆症。目前的药物只是暂缓症状,不会改变病程。

The study tested solanezumab by Eli Lilly & Co. and gantenerumab by Swiss drugmaker Roche and its US subsidiary, Genentech. Both drugs gave disappointing results in some earlier studies, but the doses in this one ranged up to four to five times higher and researchers had hoped that would prove more effective.

这项研究测试了礼来公司生产的索拉尼珠单抗和瑞士罗氏制药公司及其美国子公司基因泰克生产的甘特努马。这两种药物在早期的一些研究中都给出了令人失望的结果,但这一种药物的剂量高达4到5倍,研究人员希望可以证明更有效。

The study was funded by the US National Institute on Aging, the Alzheimer’s Association and some foundations.

这项研究是由美国国立衰老研究所、阿尔茨海默病协会和一些基金会资助的。

It involved about 200 people in the United States, Europe and elsewhere with flaws in one of three genes.

它涉及到美国、欧洲和其他地方约200人,其中一个有基因缺陷。

“If you get one of these genetic mutations you’re almost guaranteed to get Alzheimer’s,” typically in your 30s, 40s or 50s, said Dr. Eric McDade, another study leader at Washington University.

华盛顿大学(Washington University)另一项研究的负责人埃里克·麦克达德(Eric McDade)博士说,“如果你发生了其中一种基因突变,你几乎肯定会患上阿尔茨海默病,”一般是在30多岁、40多岁或50多岁的时候。

People like this account for only about 1 percent of Alzheimer’s cases, but their brain changes and symptoms are similar to those who develop the disease at a later age. That gives a unique chance to test potential treatments.

像这样的人只占阿尔茨海默症患者的1%,但他们的大脑变化和症状与那些在老年时患病的人相似。这为测试潜在的治疗方法提供了一个独特的机会。

“We know everyone will get sick and we know about what time that is” in their lives, Bateman said.

贝特曼说:“我们知道每个人都会生病,我们也知道他们生命中什么时候会生病。”。

Most study participants already had signs of the harmful protein in their brain even if they were showing no symptoms when the study started.

大多数研究参与者的大脑中已经有有害蛋白质的迹象,即使他们在研究开始时没有表现出任何症状。


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