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Potential new treatment for cystic fibrosis?

I just found an interesting article at the PHG Foundation about a new potential treatment for cystic fibrosis, a genetic condition affecting the exocrine (mucus) glands of the lungs, liver, pancreas, and intestines. It is caused by a mutation in the CFTR gene. The product of that gene is a a chloride ion channel that plays role in creating digestive juices and mucus. If there is no normal copy of the gene, the person will be affected by CF.

The new drug VX-770 was developed by the Cystic Fibrosis Foundation in collaboration with Vertex Pharmaceuticals; it targets the defective CFTR protein to improve chloride transport. The Cystic Fibrosis Trust supports a group at the University of Bristol in investigating how new drugs restore function to defective CFTR proteins; group leader Dr David Sheppard reported results at the BA Festival of Science indicating that the new drug could cause a near 50% reduction in salt levels in sweat and a 10% improvement in lung function in cystic fibrosis patients. He said: “The early results with VX-770 suggest that drug therapies which target defects at the root of the disease have the potential to improve greatly the quality of life of CF patients”

Here is a video describing the symptoms:

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5 Comments Post a comment
  1. jeanne B #

    how about vx 809 for the population of c f’rs with the most common cystic fibrosis mutation Delta F508..Where is it ? We need it a.s.a.p.

    September 24, 2008
  2. MARIA PLONKA #

    MY SON HAS CYSTIC FIBROSIS. HE IS 26 OLD. I NEED INFORMATIONS LIKE THIS TO TAKE ONE DAY AT THE TIME.

    October 21, 2008
  3. Lily #

    Hi Jeanne B, This is Lily from the Cystic Fibrosis Foundation. I think I can help answer your question. VX-770 and VX-809 are two individual compounds being evaluated in separate clinical studies. VX-770 is more advanced in clinical trials (Phase 2), and VX-809 is still in Phase 1 studies (meaning it’s being tested in healthy volunteers). I know the Foundation is working to make promising new treatments available to patients as quickly as possible. All compounds need to be proven safe and effective before they are available to patients. Check out our Web site periodically to see updates on VX-809 and other CF treatments in development (http://www.cff.org/treatments/Pipeline/), or you can sign up to receive e-mail updates at: http://www.cff.org/register. Hope this helps!

    October 23, 2008
  4. william wagner #

    my name is william i am a 22 yr old male with cystic, i am in relatively good health with decent PFT’s is there anyway that i can volunteer for this drug(VX-770)? and if so how would i go about doing this?

    December 3, 2008
  5. Lily #

    Hi, William, Thanks for the question. You can find information about volunteering for VX-770 clinical trials on our Web site at: http://www.cff.org/research/ClinicalResearch/FAQs/VX-770/#How_can_I_participate?. For more information about CF clinical trials, you can also talk to your CF doctor or call (877) 8CF-JOIN.

    December 10, 2008

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