Did you ever raise money for cystic fibrosis research with Great Strides National Walk Event? Well, if you did or if you ever gave money to the Foundation, you probably helped create a promising new drug!
Even as a young kid I was not overly optimistic that the money I raised with the Great Strides Event would ever help the 3 older girls I knew with cystic fibrosis (CF). Cures and treatments seemed very far in the future, an improbable hope. However, the improbable has become more than probable!!
A new drug, Kalydeco, was recently discovered for CF treatment (FDA approved Jan. 2012). The Cystic Fibrosis Foundation website reveals, "It is the first drug available that targets the underlying cause of CF — a faulty gene and its protein product, CFTR." As we know from class, the CFTR receptor is able to localize to the cell membrane, but the mutant receptor causes a thick mucus to form as a result of nonfunctional chloride ion transport. This drug helps the mutant receptor function properly and improve lung function. They are unsure if this is a cure for CF individuals or a specific treatment. Unfortunately, this drug is only approved for individuals with a specific gene mutation: the G551D mutation.
Personally, I am getting very hopeful for CF treatments and possible cures. When I was younger I was devastated to hear that most cystic fibrosis patients did not live to see their 30th birthday. And now, I know five people with CF ages 26, 28, 30, and 31. And the reason I even found out about this new drug to blog about is that the 31 year old just delivered healthy baby TWINS recently! This would have been unheard of 30 years ago. The drug company Vertex is working on developing a similar drug to Kalydeco (VX-770) for the mutation that most CF patients have (and that the 31 year old new mother and her 28 year old sister both have.) This is very exciting for them and I hope that researchers are able to develop a similar drug quickly.
References:
I got the idea from a personal newsletter from our family friends. The title of the blog comes from the fact that many young kids refer to cystic fibrosis as "sixty-five roses" because they sound so similar and it is easier to say.
More drug information obtained from The Cystic Fibrosis Foundation website, http://www.cff.org/treatments/Therapies/Kalydeco/
Image from: ASBMB Today at http://www.asbmb.org/asbmbtoday/asbmbtoday_article.aspx?id=18382
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