RNAi干扰药物首次在人体内生效

readok2009

编译自华尔街健康博客：作者雅各布戈尔茨坦

Alnylam公司，一家热门的生物技术小公司一直在猴子身上试验遗传物质核糖核酸RNA治疗疾病的效果，这次它已经可以对外宣布它们正在试验的一个药品在人身上已显示疗效效。

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。

在本次成功的试验中， Alnylam公司研究人员让88男子接触感染呼吸道合胞病毒，这是婴儿和老人经常经历的感染性病毒。其中有一半受试者还接受RNAi的鼻喷剂，用来干扰病毒的蛋白质产生，另一半受试则用安慰剂喷鼻作对照。

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。

虽然这些小规模临床数据并不意味治愈疾病的\"灵丹妙药\"已横空出世 ，它只是显示，该方法能够在人体临床应用和治疗疾病中能有正面影响，这对一个尚未有任何上市应用于人体临床的药品但已吸引了这么多的关注和投资的领域，是很大的进步。

据华尔街日报报道，另一家开发RNAi药物的公司OPKO健康，在Alnylam公司的“概念性证明“公布之前，已有成功的先例，它早在2006年就成功地用RNAi药物治疗黄斑degenration。但这些结果在统计学意义上还不显著，因此其科学有效性还要打很多折扣。
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