A drug that can permanently cure cystic fibrosis, the most common hereditary disease known to man. That is the dream of the many sufferers around the globe who have an inability to transfer chloride ions so mucus builds up in their lungs causing breathlessness and possible death due to lack of oxygen transfer to cells. Two drugs have surged to combat this disease however. One of them is called VX-770, or as they like to call it, a placebo. This is quite confusing to me since a placebo is not a drug, it is simply the belief of a patient that he is being treated when in actual fact he is not. But I digress. After receiving VX-770, 20% of patients actually improved their lung function somehow.
Most other treatments for cystic fibrosis actually targeted the symptoms and tried to alleviate those. This new drug goes directly to the source: a faulty protein in the transmembrane regulator. As the drug fixes the problem the channel proteins open up again allowing the transfer of chloride ions through its plasma membrane. This slows down the onset of symptoms and the effect on the digestive system is eased.
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| The cystic fibrosis allele is recessive |
The problem of this new treatment is that it only 5% of the total population of cystic fibrosis sufferers can be cured. That is where the second, as of yet unknown, drug comes in. Scientists hope that a combination of both these drugs could be final cure that is required. An ultimatum for the disease if you will. Hopefully science will be successful once again.
3 comments:
Good job Lucas!
great post, you might like to read that,
http://www.newscientist.com/article/dn20169-new-drug-is-champagne-moment-for-cystic-fibrosis.html
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