RNAi Interference?
Kjell, (who points us to good stuff from time to time) wrote us recently about a Wired article on RNA interference technology.
Simply put, RNA interference (commonly abbreviated RNAi) could be the magic bullet to treat all bacterial and viral diseases.
RNAi could be a universal tool in treating disease. [Sirna Therapeutics is using mice to experiment with RNAi on Hepatitis - with great success.] Another small biotechnology company, Alnylam Pharmaceuticals, is working on an RNAi-based treatment for flu. And human trials are already underway in using RNAi to treat macular degeneration, a leading cause of blindness in the elderly.
The problem with RNAi has been largely one of delivery.
Getting the RNA across the membrane has been the big bugaboo so far of RNAi-based therapies. "That's what's so exciting about this paper [from Sirna Therapeutics]," Shaul said. "They show an excellent transport method [encapsulation inside molecules of fatty acids] that seems to really work well. If we can get it into the cell, we have good reason to believe that it will work well there."
One reason that RNAi has scientists excited is that it does not affect DNA the way traditional gene therapy does. Instead, it simply silences the expression of some RNA genes. The hope, according to Wikipedia, is that RNAi can be used to repress "essential genes in eukaryotic human pathogens or viruses that are dissimilar from any human genes."
This must be what the scientists on DARPA's "Unconventional Pathogen Countermeasures" program are working on - in part. Last May I learned from Joel Garreau's book, Radical Evolution, that the goal of this DARPA program is:
…to create super-soldiers that are resistant to all disease. The object of the game is to discover the essential part of life common to many of these pathogens...and interrupt them. An example would be finding an enzyme that appears only in bacteria, but not in us…
I'm not a believer in a conspiracy theory of history. I don't, for example, think that Proctor and Gamble (or any other company) has a sonic washing machine they're withholding in order to sell us detergent. I was not suggesting any conspiracy in my "Top Men" post. My point with "Top Men" is that there are more ideas and resources available to us than any entity - even one as large as the government - can pursue on its own.
The Pentagon could not easily withhold a universal disease fighting drug from the public. If DARPA develops this treatment first and tried to keep it classified, the public would find out about it the first time super-soldiers are deployed. It's too big a secret to keep.
Still, I'm glad to learn that the private sector is working on RNAi. Profit-motivated companies have a great incentive to speed these treatments to the public. Of course, a conspiracy theorist might worry about the private sector too: "Why would a drug company invent a therapy that would render many other drugs obsolete?"
Answer: Because there is more than one drug company. These drug companies are stuck in a "prisoner's dilemma." Each has an interest in bringing any miracle drug to market before another company does so. I'm optimistic that the public will have access to any "miracle" therapy - including RNAi - as soon as its available and safe.
UPDATE: Althought RNAi is not gene therapy in the conventional sense, it shares a common problem - delivery. Would a common solution be possible?
Perhaps ORMOSIL molecules could be adapted for RNAi.
Comments
I have a feeling that there would be plenty of money in this for the drug companies for a long time to come. It would only be a potential magic bullet at first, becoming more useful as time goes forward.
This story is a good counterpoint to all the (appropriately) nervous talk about an imminent avian flu pandemic. I wonder if RANi would lend itself to pro-active treatment like an innoculation, or can it only be used to cure an infection that one has already caught.
Posted by: Phil Bowermaster
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September 12, 2005 03:10 PM
Phil:
There is so much of this I don't know or understand yet, but I think the hope is that this could be a universal innoculation.
You find an essential gene that many viruses or harmful bacteria have in common (that is not found in the human genome of course) and you block that gene with RNAi. One injection might provide protection against all forms of flu and many other illnesses as well.
How long this protection would last is unknown. But I'd happily schedule a monthly injection if it meant never getting sick.
The other good thing - this is a deadlier blow to bacterial infections than antibiotics. If you are blocking an essential gene it might prove impossible for these bacteria to evolve a defense. That's what the DARPA scientist that Garreau interviewed thought. And hey, even if it did, we'd just block a different essential gene.
Posted by: Stephen Gordon
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September 12, 2005 03:39 PM