November 2nd, 2011
05:09 PM ET

New drug 'big deal' in cystic fibrosis fight

A new pill may help in the fight with cystic fibrosis.

A study, funded in part by Vertex Pharmaceuticals, found the drug ivacaftor (pronounced eye va kaf tor) caused patients with a specific type of cystic fibrosis to improve lung function, gain weight, and fight other aspects of the disease.

“This is the first time that we have a therapy that is directed at the cause of cystic fibrosis,” says Dr. Michael Konstan, the chair of the Department of Pediatrics at Rainbow Babies and Children’s Hospital in Cleveland and one of the authors of the study. There is no cure for this disease and existing treatments just help alleviate some of the symptoms.

“This is a big deal,” says Robert Beall, the president and CEO of the Cystic Fibrosis Foundation.  “The fact is it is obviously going to make a very big difference in the lives of patients.”

Cystic fibrosis is a genetic disease that causes problems with the balance of salt going in and out of cells. A sticky mucus clogs organs, particularly the lungs and liver. It can also cause digestive problems. About 1,000 new cases are diagnosed each year, according to the Cystic Fibrosis Foundation, and the average person with the disease dies before age 40.

Ivacaftor works for people with the G551D mutation of cystic fibrosis. Konstan says that only about 4% of the people with the disease have this specific mutation. “It really is a major milestone in the treatment of cystic fibrosis,” he says. “Even thought it only affects a small percentage (of patients) the hope is there that we can finally do something about this disease.”

The study, published in the New England Journal of Medicine Wednesday, looked at 84 people who got the drug. Those taking ivacaftor saw improvements within two weeks, which continued for the nearly yearlong trial. Drug recipients could blow out more than 10% more air from their lungs compared with patients who were not taking the drug, and were about half as likely to have a “pulmonary exacerbation” or a worsening of their lung disease that often requires hospitalization. On average, patients taking the drug grew more and put on more weight than the placebo group, according to the study, and had lower sweat chloride – an indicator of the disease.

Beall says the improved lung function helped patients double their ability to walk and one patient even started going the gym for the first time.

“We have an incredible proof of concept,” Beall says. He points to other research under way with ivacaftor combined with another drug that could help people with the most common mutation of cystic fibrosis. That trial is ongoing and the treatment is at least four or five  years away from approval, but if it works, Beall says, it could help 90% of the people with cystic fibrosis.

In an editorial accompanying the study, Dr. Pamela Davis, of Case Western University paraphrased Winston Churchill, “This study is also a great victory in the war against genetic diseases and marks the end of the beginning for the treatment of the cystic fibrosis defect.”

Ivacaftor has been submitted to the FDA for approval, and could be available next year, according to Konstan. When it goes on sale it will be sold under the brand name Kalydeco (pronounced kuh-LYE-deh-koh).

soundoff (307 Responses)
  1. BbdfMile

    how to take viagra with water or milk https://gensitecil.com/ buy viagra cheaper

    April 27, 2021 at 20:23 | Report abuse | Reply
  2. LhdvZisse

    best prices for cialis http://asciled.com/ cialis 20 mg cheap

    April 27, 2021 at 23:00 | Report abuse | Reply
  3. priligy

    priligy tablets for sale buy avanafil online

    April 28, 2021 at 01:40 | Report abuse | Reply
  4. LrbsEncox

    levitra generique 40 mg levitra walmart walgreens levitra price

    April 28, 2021 at 15:40 | Report abuse | Reply
  5. tendances du coronavirus - france

    drug chloroquine https://chloroquineorigin.com/ malaria drugs list

    April 28, 2021 at 22:55 | Report abuse | Reply
  6. RfvbZisse

    generic cialis in usa http://cileve.com/ best price on cialis

    April 29, 2021 at 05:11 | Report abuse | Reply
  7. RfvbZisse

    google south africa cialis https://cileve.com/ – canadian pharmacy no prescription generic cialis cialis black review

    April 29, 2021 at 09:07 | Report abuse | Reply
  8. LhdvZisse

    cialis canadian http://asciled.com/ cialis in canada

    April 29, 2021 at 09:45 | Report abuse | Reply
  9. GvdbCrulk

    tadalafil online reviews https://boxtadafil.com/ cialis tadalafil 20mg preis

    April 29, 2021 at 10:58 | Report abuse | Reply
  10. buy cialis online canadian

    do you need prescription for canadian perscribition of cialis in usa https://canadacialisstore.com/ buying canadian cialis

    April 29, 2021 at 14:01 | Report abuse | Reply
  11. buy cialis online canadian

    canadian pharmacy generic cialis https://canadacialisstore.com/ cialis canadian

    April 29, 2021 at 21:00 | Report abuse | Reply
  12. avana

    buy avanafil uk buy avanafil online

    April 30, 2021 at 14:15 | Report abuse | Reply
  13. LrbsEncox

    levitra clinical trials http://uslevitraanna.com/ levitra dosages

    April 30, 2021 at 14:36 | Report abuse | Reply
  14. FbsfEncox

    Vasotec http://uspharmus.com/ Yasmin

    May 2, 2021 at 13:55 | Report abuse | Reply
  15. FbsfEncox

    Prozac http://uspharmus.com/ Duphaston

    May 2, 2021 at 15:23 | Report abuse | Reply
  16. LebnEncox

    best online international pharmacies canadian pharmacies-24h canada pharmacy not requiring prescription

    May 3, 2021 at 21:29 | Report abuse | Reply
  17. RebfZisse

    cialis lilly 20mg https://rcialisgl.com/ – order generic cialis cialis australia

    May 4, 2021 at 04:54 | Report abuse | Reply
  18. RebfZisse

    cocaine cialis http://rcialisgl.com/ research cialis

    May 4, 2021 at 09:23 | Report abuse | Reply
  19. LbsoZisse

    generic cialis from canada https://ucialisdas.com/ cialis overnight

    May 4, 2021 at 10:07 | Report abuse | Reply
  20. GtnbCrulk

    amazon viagra https://jokviagra.com/ best place to buy viagra online forum

    May 4, 2021 at 20:49 | Report abuse | Reply
  21. FmrfEncox

    buy cialis overnight delivery http://cialisee.com/ generic cialis 20 mg online

    May 5, 2021 at 15:16 | Report abuse | Reply
  22. FmrfEncox

    cialis viagra http://cialisee.com/ – name brand cialis generic cialis tadalafil 20mg

    May 5, 2021 at 16:53 | Report abuse | Reply
  23. JebgEncox

    cheap online pharmacy rate canadian pharmacies best rx pharmacy online

    May 6, 2021 at 22:43 | Report abuse | Reply
  24. JebgEncox

    ed meds online http://pharmacylo.com/ Asacol

    May 7, 2021 at 04:07 | Report abuse | Reply
  25. BbshMile

    generic viagra names https://loxviagra.com/ viagra buy online

    May 7, 2021 at 13:29 | Report abuse | Reply
  26. AnooEncox

    viagra other uses http://llviagra.com/ medical viagra side effects

    May 8, 2021 at 15:27 | Report abuse | Reply
  27. AnooEncox

    viagra before and after pictures https://llviagra.com/ viagra e tellimine

    May 8, 2021 at 22:14 | Report abuse | Reply
  28. GtnbCrulk

    como funciona el viagra https://jokviagra.com/ what happens if a woman takes viagra

    May 8, 2021 at 22:51 | Report abuse | Reply
  29. dapoxetine

    buy priligy uk priligy pills

    May 8, 2021 at 23:52 | Report abuse | Reply
  30. cloroquina

    cloriquine https://chloroquineorigin.com/ chloroquine drug class

    May 9, 2021 at 00:13 | Report abuse | Reply
  31. LbsoZisse

    cialis omline https://ucialisdas.com/ generic cialis forums

    May 9, 2021 at 05:13 | Report abuse | Reply
  32. LebnEncox

    canada prescription drugs express pharmacy canadian pharmaceuticals online

    May 9, 2021 at 12:14 | Report abuse | Reply
  33. BbshMile

    viagra pill http://loxviagra.com/ buy viagra connect usa

    May 9, 2021 at 16:43 | Report abuse | Reply
  34. LebnEncox

    Avapro http://xlnpharmacy.com/ Zyvox

    May 10, 2021 at 19:03 | Report abuse | Reply
  35. RebfZisse

    cialis/dapoxetine with overnight ship http://rcialisgl.com/ buy cialis online reviews

    May 11, 2021 at 02:52 | Report abuse | Reply
  36. RebfZisse

    where to by cialis http://rcialisgl.com/ buy cialis information

    May 11, 2021 at 07:03 | Report abuse | Reply
  37. LbsoZisse

    viagra/cialis black and gold 800 http://ucialisdas.com/ – cialis online from canada cialisprofessional

    May 11, 2021 at 07:37 | Report abuse | Reply
  38. GtnbCrulk

    viagra amazon http://jokviagra.com/ – how much does viagra cost per pill viagra government funded

    May 12, 2021 at 07:24 | Report abuse | Reply
  39. FmrfEncox

    cialis 20 mg http://cialisee.com/ canada cialis

    May 12, 2021 at 17:39 | Report abuse | Reply
  40. FmrfEncox

    buy cialis cheap online http://cialisee.com/ – generic cialis cialis with dapoxetine 80mg

    May 12, 2021 at 19:13 | Report abuse | Reply
  41. JebgEncox

    Sildalist http://pharmacylo.com/ drugs without prescription

    May 14, 2021 at 23:08 | Report abuse | Reply
  42. BbshMile

    who makes viagra http://loxviagra.com/ what does viagra look like

    May 15, 2021 at 00:50 | Report abuse | Reply
  43. JebgEncox

    Forzest http://pharmacylo.com/ discount pharmacy

    May 15, 2021 at 04:19 | Report abuse | Reply
  44. AnooEncox

    online viagra in uk http://llviagra.com/ rimedio naturale al viagra

    May 15, 2021 at 10:10 | Report abuse | Reply
  45. AnooEncox

    amount of money spent on viagra http://llviagra.com/ – canadian drugs viagra im 19 and want viagra

    May 15, 2021 at 15:44 | Report abuse | Reply
  46. cyclosporine

    buy cyclomune uk cyclosporine eye drops

    May 15, 2021 at 15:48 | Report abuse | Reply
  47. LbsoZisse

    drug categories viagra cialis http://ucialisdas.com/ purchase cialis online cheap

    May 16, 2021 at 06:31 | Report abuse | Reply
  48. avana

    avana 50 mg buy avana canada

    May 16, 2021 at 10:01 | Report abuse | Reply
  49. GtnbCrulk

    can you buy viagra online http://jokviagra.com/ viagra 150 mg

    May 16, 2021 at 10:58 | Report abuse | Reply
  50. dapoxetine

    dapoxetine tablets price generic priligy

    May 16, 2021 at 21:40 | Report abuse | Reply
1 2 3 4 5 6

Post a comment


CNN welcomes a lively and courteous discussion as long as you follow the Rules of Conduct set forth in our Terms of Service. Comments are not pre-screened before they post. You agree that anything you post may be used, along with your name and profile picture, in accordance with our Privacy Policy and the license you have granted pursuant to our Terms of Service.

About this blog

Get a behind-the-scenes look at the latest stories from CNN Chief Medical Correspondent, Dr. Sanjay Gupta, Senior Medical Correspondent Elizabeth Cohen and the CNN Medical Unit producers. They'll share news and views on health and medical trends - info that will help you take better care of yourself and the people you love.