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Wednesday, September 15, 2004
Will Wet AMD Genes Be Turned Off By RNAi? Human Trials Begin
If all goes according to plan, about half a dozen elderly people at risk of blindness will visit Dr. Lawrence J. Singerman's retina clinic in the coming weeks to receive injections in the whites of their eyes.
The experimental injections will contain a new type of drug based on a recently discovered genetic phenomenon, called RNA interference, that has excited scientists with its versatile and powerful ability to turn off genes. Having quickly become a standard tool for genetic studies in the laboratory, the technique is now set to be tested in people for the first time.
'Method to Turn Off Bad Genes Is Set for Tests on Human Eyes' (The New York Times, 14 September 2004).
The US Food and Drug Administration has granted Philadelphia-based company Acuity Pharmaceuticals permission to begin trials in humans. The New York Times article includes comments from researchers who believe the treatment may have a future as well as from scientists who believe there is little evidence to suggest the injections of RNAi will work.
Acuity Pharmaceuticals is not the only company working on finding ways of using RNAi to treat disease and there is an Australian connection:
"There's no doubt in my mind that this is the clear winner," said Mark A. Kay, a Stanford professor. He hopes to test an RNAi treatment for hepatitis C in cooperation with Benitec, an Australian company that now owns a company he founded.
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