For the First Time*, an RNAi Drug Works In People
Posted by Jacob Goldstein
Alnylam, one of the hot little companies trying to figure out how to monkey with the genetic material RNA to treat disease, said it’s just showed that one of its drugs showed a significant effect in people.
The company called it the first time an RNAi drug has demonstrated proof of concept in people. RNA, a close cousin of DNA, is essential in the process of creating proteins. The “i” in RNAi stands for “interference”; the drugs are supposed to work by using RNA to interfere with the production of proteins associated with disease.
In this case, Alnylam researchers exposed 88 men to the respiratory syncytial virus, which is largely a problem among infants and the elderly. Half also received nasal sprays designed to interfere with one of the proteins produced by the virus; the other half received a placebo.
Among those receiving the placebo, 88% ended up infected with the virus, as measured by various laboratory diagnostic tests, according to the WSJ. Among those getting the RNA spray, only 67% were infected.
Those numbers don’t scream “miracle cure.” But simply showing that the approach can have a positive effect people is a significant step for a field that’s attracted so much attention and investment before developing any drugs that have worked in people.
*OPKO Health, another company working on RNAi drugs, contested Alnylam’s proof-of-concept claim for the study, according to the WSJ. OPKO says it presented successful results of an RNAi drug for macular degenration in 2006. But those results weren’t quite dramatic enough to be statistically significant, the standard measure of scientific validity, the WSJ notes.
Photo: Respiratory syncytial virus by CDC via Wikimedia Commons
该公司称这是第一次RNAi药物治疗的概念在人体试验中获得成功。 RNA是DNA基因表达的过程制造蛋白必不可少的组分。 "I"在RNAi中的含义就是"干扰" ;RNAi药品就是通过干扰阻碍与其对应的DNA制造与疾病相关的蛋白过程而起效。
其中那些接受安慰剂处理的试验者中，有88 ％ 感染了禽流感病毒。那这些获得的RNA喷雾的患者，只有67 ％受感染。是否被病毒感染是通过各类实验室诊断试验决定的。
作者:admin@医学,生命科学 2011-04-18 17:14