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Cystic Fibrosis cure getting closer at San Diego lab

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No one will call it a cure yet, but a new drug developed in the San Diego lab of Vertex Pharmaceuticals was designed as an actual fix for the gene defect called cystic fibrosis. Ivacaftor, trademarked as Kalydeco,  is a drug that currently is used to treat 4-5% of sufferers of cystic fibrosis, those who have the G551D gene defect. However, if used in combination with another potential CF therapy, VX-661, recent clinical testing results have shown a significant improvement in lung function in people with two copies of the CF mutation Delta F508. The actual government study is available here: Study of VX661 Alone and in Combination With VX-770 in Subjects

30,000 people in the United States have cystic fibrosis, most of them will not live to be 40. Cystic fibrosis is caused by any one of several defects in a protein, the cystic fibrosis transmembrane conductance regulator, which regulates fluid flow within cells and affects the components of sweat, digestive fluids, and mucus. The defect is caused by a mutation in the individual’s DNA. The CF gene causes clogging in several vital organs, with the most damage usually occurring in the lungs.

There has been a continual increase in the success of CF treatment in the last 50 years. In 1960, most sufferers of the disease did not live to be 10 years old.

The two treatments are currently going through the last phase of trial on humans. The next step would be to gain FDA approval to use Kalydeco with VX-661.CF1

Part of the cost of developing the new drug was paid for by the Cystic Fibrosis Foundation. The organization has raised funds for a CF cure since 1955. San Diego & Imperial Counties CFF spokesperson Laurent Quenaud said,  “There’s little to no government funding for cystic fibrosis research so we are involved in every single drug that comes out.

(Editor’s Note: This article has been changed to make it more accurate and clear since its initial publication. Thanks goes to Jenn Whinnem for the comments that provided some of the additional information about this topic.)

4 Comments

  1. Glen McMahon

    May 27, 2013 at 7:32 am

    This is fantastic news!!!!

  2. Jenn Whinnem

    May 27, 2013 at 7:57 am

    While I’m as excited about Kalydeco – that’s the drug you wrote about here but inexplicably did not name – as anyone else, this is really slopping reporting. No mention of the name of the drug, no mention of the fact that Kalydeco is only effective for 4% of the CF population at this time, no explanation of the little picture which even *I* don’t understand, a sloppy description of CF, and the claims of this spokesperson are the first I’ve heard of some of these things (bigger lungs? being able to have kids – it’s going to allow the vas deferns to grow in the 98% of CF males who don’t grow one?). Please try harder next time.

  3. Marla

    May 27, 2013 at 2:13 pm

    This article is extremely vague. Who is “the majority of sufferers”? DDF508?

    And this sentence “The next step would be it available to all cystic fibrosis patients”… this is not completely accurate is it? That is not “the next step” right? There are many, many steps that need to happen before any drug or combination will be effective with ALL mutations.

    Sorry, I am not trying to rain on the parade. I just want to make sure that no one forgets that there is a LONG way to go. There is joy for the next round of CFers that will be helped. My understanding is, that it is people with DDF508 (2 copies of DF508), the rest will still be left waiting…

  4. Jenn Whinnem

    May 28, 2013 at 2:03 pm

    Kudos and many thanks to the author for updating the piece. Applause. This is now an article I’m happy to share with friends.

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