softie
Posts:
1,070
From:
Croydon, Surrey
Registered:
07-Feb-2004
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Re: gene therapy?
Posted:
03-Dec-2006 00:05
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How effective gene therapy will be on people with severe lung disease has yet to be seen. It is assumed that people with very bad lung disease won't get much benefit from it. It is assumed that gene therapy has to be targeted at epithelial cells - these are the ones that line the lungs and have the job of maintaining the wateriness of the mucous. Scarring, inflammation and damage all remove the epithelial cells, and it is assumed that scarred, damaged, or inflamed areas of lung won't respond to therapy.
There are other treatments in the pipeline which will be more likely to help people with more serious lung damage. A lot of work is being done into suppressing the inflammation that is one cause of our problems. There is also work being done into fooling pseudo into not sticking to the lung walls, making it easier to kill it with antibiotics. There is ongoing research into new antibiotics, and, finally, work being done into new, cheaper, possibly better ways of thinning the mucous than using DNAse.
My understanding is that drug trials are done using volunteers with moderate lung problems. People with very mild problems will be unlikely to use new drugs, and so results of trials performed on them are unrepresentative. People with extremely severe problems are too prone to "accidents" such as fatal viral infections or hospital-acquired bacterial infections, side-effects of other drugs and so on; which means that it is very hard to see if the new drug is having any effect.
Once a treatment has been approved then it is up to the doctors who they give it to, and this is done on a risks/benefits basis. So, you don't give a very powerful but dangerous drug to a person with very mild problems as giving them the drug will put them in more danger than NOT giving it. Similarly you don't waste time with a very safe but not very effective drug on people with severe problems, but you give them the drug which is powerful but dangerous, since they will be in more danger from the disease than from the side-effects of the drug.
Safe, powerful drugs are handed out eagerly. Cipro used to be one such - extremely effective against pseudomonas, and very safe. Until strains of pseudo started to develop resistance to it, that is!
Richard.
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