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Wednesday, February 23, 2011

Drug Offers New Hope For People With Cystic Fibrosis

Emily Schaller of Detroit has Cystic Fibrosis, and has noticed a dramatic improvement in her breathing after trying a new drug developed by Vertex Pharmaceuticals of Cambridge and the Cystic Fibrosis Foundation. (Cystic Fibrosis Foundation)

Emily Schaller of Detroit has cystic fibrosis, and has noticed a dramatic improvement in her breathing after trying a new drug developed by Vertex Pharmaceuticals of Cambridge and the Cystic Fibrosis Foundation. (Cystic Fibrosis Foundation)

The Cystic Fibrosis Foundation and Vertex Pharmaceuticals of Cambridge, Mass. have announced a successful trial for a new drug that treats the underlying cause of cystic fibrosis.

About 30,000 people in the United States have CF, which is caused by a genetic mutation that creates a buildup of thick mucus in the lungs and digestive system. People with CF rarely live beyond their mid-30′s. Drugs currently on the market only treat symptoms of the disease, while the new medication targets the defective protein that causes it.

Patients in the study experienced dramatic improvements in lung function and weight gain. We speak with one of them, Emily Schaller of Detroit, who is breathing better than she can ever remember.


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  • Jgonski

    your headline is misleading, the story is not “about the new drug”, it is about what it is like to live with C F. I learned nothing about the drug, only that this women was in a trial, may or may not have had the drug, and that she can take a deeper breath and not cough. Interviewing the research team, finding out why this maybe a break through, will it help infants or will it help the entire C F community, how long before it might be approved, all would have been more educational than the personal profile of one person with C F. My daughter has C F, this interview was mainly old news…

    • Suburbiatexas

      under the headline it reads, so it does mention that she has taken the drug and showed an improvement in her breathing.

      “Emily Schaller of Detroit has cystic fibrosis, and has noticed a dramatic improvement in her breathing after trying a new drug developed by Vertex Pharmaceuticals of Cambridge and the Cystic Fibrosis Foundation. (Cystic Fibrosis Foundation)”

  • papa bear

    It would have been nice to have more information on the Drug itself. When will the drug be available? Who can get in the study? Here is the press release site, note it is in the investor section of the website.
    http://investors.vrtx.com/releasedetail.cfm?ReleaseID=551869

  • REBEXD

    you have to listen to the story. click on the link above that says “LISTEN TO THE STORY”

  • Rolanda Rettkowski

    My daughter has cf and when i seen this headline i was given hope there was a break through in medicine but i am left wondering WHAT!Great she can breath better.What is the medicine????Those of us who deal with this everyday see something like this with thrill.What a major let down.We want to know more about the drug because we are well aware what cf is and how it affects the body.How do find out about the drug?

    • Guest

      Check out the Vertex page of Yahoo finance…there are lots of links to articles about VX-770

      http://finance.yahoo.com/q?s=VRTX

    • Lynn Jolicoeur

      Hi Rolanda,

      Thank you for your message, and I do hope this medication will give your daughter new hope.

      There is a link to the Cystic Fibrosis Foundation website above, but here is the link to their website’s page with the announcement about the drug.
      http://www.cff.org/aboutCFFoundation/NewsEvents/2-23-Phase-3-Study-VX-770-Shows-Positive-Results.cfm

      There is no name for the medication yet as it is not approved by the FDA. Vertex plans to file papers to seek approval later this year, but that process can take some time. Also, Vertex has said this medication cannot help everyone with cystic fibrosis. But for a certain segment of the population it is showing great promise.

      I wish your daughter the best in battling this disease.

      Lynn Jolicoeur
      Here & Now producer

    • http://pulse.yahoo.com/_V46SKDJ52XFPWNASLTXHP3XBPY Holly

      Rolanda,

      If you’re not already, get involved with your local CFF for more information about VX-770. Until it goes before the FDA for approval it won’t have an official name.
      It’s probably too late to be part of the study that Emily was in but if they do a future study (without placebos) you may be able to get her in that trial.

      Stay positive, this may be the breakthrough we’re all hoping for. :)

  • http://pulse.yahoo.com/_V46SKDJ52XFPWNASLTXHP3XBPY Holly

    I’m so happy for Emily!! I hope that this drug gets FDA approval soon if it’s actually targeting that protein!

    Yes, all of us with CF knew that Vertex has been in the trial stage of this drug but to finally hear someone that’s benefited from this trial is amazing!

    Emily.. take a clear, deep breath for me!!

  • http://pulse.yahoo.com/_V46SKDJ52XFPWNASLTXHP3XBPY Holly

    Also, for people that are interested in CF trials for new medicines – http://www.clinicaltrials.gov/ct2/search

    Just make certain you talk to your doctor in detail before you join.

  • Pingback: Special Report: New Cystic Fibrosis Drug Brings Gift Of A Future | CommonHealth

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