RNA interference passes a big test - RNAi

RNA interference passes a big test
June 7, 2006
Eric Berger
SciGuy
You may never have heard of it before, but a technique called RNA interference, or RNAi, has been all the rage in biology labs for some time now. The premise is quite simple, really. It exploits a biological mechanism within the body that can be activated to "silence" genes.
Got a gene (or a mutated gene) that's making a harmful protein? Simply inject some tiny bits of RNA and it will make the gene stop. I wrote more about RNAi last year.
There are some reasons to believe RNAi might not work -- there are ways the body can neutralize the tiny bits of RNA before they reach the genes one wants to silence -- but there was great hope when a company called Acuity Pharmaceuticals launched clinical trials last year. Acuity hoped its RNAi drug would be successful at treating the most common cause of blindness in the elderly, macular degeneration.
You know what? The drug worked, and the company says a Phase 3 trial could begin as soon as the end of next year:
The trial on 129 patients found that Bevasiranib reduced blood-vessel growth in the eyes and improved vision slightly. At the lowest doses, these effects lasted for several months; at higher doses the positive responses are still present, says Dale Pfost, Acuity's president.
No adverse side effects were seen other than the anticipated swelling and inflammation at the site where the drug was injected into the eye.
If I were a betting man -- and I only am during football season -- I'd say RNA interference may just be the biotechnology product that lives up to the hype.