The hurdles for using RNAi are 1) find one that works and 2) deliver it in a way that keeps it in working condition. No. 2 is very important as RNA is a very fragile molecule. The news talks about one particular RNAi molecule delivered in a clever protective way and they see a positive effect. That's awesome news in and of itself.
Is this a general enough technique to make "every protein... druggable"? Maybe, but they don't know that yet. Do some people have an immune reaction to the particles? They didn't see any in 15 patients, so we can't be sure yet if this is a big problem, a minor problem, or not a problem at all.
Very correct. This is just the second time that RNAi has been tried in humans [1] and delivery was always the big problem. When Merck acquired SIRNA, much of the discussion was around what were the options beyond ocular delivery and while nanoparticle-based delivery was always the leader in the clubhouse, no one really knew if it would work. So, it's good to be cautious. As Derek points out in the article that's been linked to, we don't know if the therapy itself was useful at this point.
Having said that, given how difficult it has been to get here, this is super cool.
If the technique is innocuous, this is the beginning of a revolution. It could be used in a wide range of other diseases, spanning from viral infections to genetic problems where a gene is overexpressed, and possibly others as well.
Her ideas about cancer really make sense to me, and match a lot of my own ideas. If she is correct, that cancer is the body's natural healing mechanism --- but for all tissues except muscle tissue, evolution never bothered working the bugs out of the code ---, then cancer approaches such as the one in this study seem a bit doomed to me.
The hurdles for using RNAi are 1) find one that works and 2) deliver it in a way that keeps it in working condition. No. 2 is very important as RNA is a very fragile molecule. The news talks about one particular RNAi molecule delivered in a clever protective way and they see a positive effect. That's awesome news in and of itself.
Is this a general enough technique to make "every protein... druggable"? Maybe, but they don't know that yet. Do some people have an immune reaction to the particles? They didn't see any in 15 patients, so we can't be sure yet if this is a big problem, a minor problem, or not a problem at all.
So let's get excited, and let's keep some perspective. The Phase II trials are the ones to watch. They don't seem to have started the process yet: http://www.clinicaltrials.gov/ct2/results?term=rrm2 .