藥物不能減緩遺傳性阿爾茨海默病的衰退

藥物不能減緩遺傳性阿爾茨海默病的衰退

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.

在一項研究中，兩種實驗性藥物未能阻止或減緩智力衰退。這項研究的對象是那些實際上注定要在相對年輕的年齡患上阿爾茨海默病的人，因為他們繼承了罕見的基因缺陷。

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.

周一公布的結(jié)果是科學(xué)家們多年來一直關(guān)注的另一個令人失望的方法——試圖去除一種在老年癡呆癥患者大腦中積聚的有害蛋白質(zhì)，老年癡呆癥是導(dǎo)致老年癡呆的主要原因。

“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.

密蘇里州圣路易斯市華盛頓大學(xué)的研究負責(zé)人蘭德爾·貝特曼博士說：“我們實際上還不知道這些藥物在去除蛋白質(zhì)方面起了什么作用，因為這些結(jié)果還在分析中。”。

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，研究的主要目標(biāo)沒有達到——服用這兩種藥物的人在思維和記憶測試中的得分與服用安慰劑的人差不多。

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.

在美國有超過500萬人和全世界有數(shù)百萬人患有老年癡呆癥。目前的藥物只是暫緩癥狀，不會改變病程。

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.

這項研究測試了禮來公司生產(chǎn)的索拉尼珠單抗和瑞士羅氏制藥公司及其美國子公司基因泰克生產(chǎn)的甘特努馬。這兩種藥物在早期的一些研究中都給出了令人失望的結(jié)果，但這一種藥物的劑量高達4到5倍，研究人員希望可以證明更有效。

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

這項研究是由美國國立衰老研究所、阿爾茨海默病協(xié)會和一些基金會資助的。

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

它涉及到美國、歐洲和其他地方約200人，其中一個有基因缺陷。

“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.

華盛頓大學(xué)(Washington University)另一項研究的負責(zé)人埃里克·麥克達德(Eric McDade)博士說，“如果你發(fā)生了其中一種基因突變，你幾乎肯定會患上阿爾茨海默病，”一般是在30多歲、40多歲或50多歲的時候。

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.

像這樣的人只占阿爾茨海默癥患者的1%，但他們的大腦變化和癥狀與那些在老年時患病的人相似。這為測試潛在的治療方法提供了一個獨特的機會。

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

貝特曼說：“我們知道每個人都會生病，我們也知道他們生命中什么時候會生病。”。

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

大多數(shù)研究參與者的大腦中已經(jīng)有有害蛋白質(zhì)的跡象，即使他們在研究開始時沒有表現(xiàn)出任何癥狀。