Featured Research

from universities, journals, and other organizations

Major cystic fibrosis breakthrough made: Drug combination improves lung function

Date:
June 25, 2014
Source:
Queen's University, Belfast
Summary:
A major breakthrough to change the lives of cystic fibrosis sufferers. The trial looked at the treatment of patients with two copies of the F508DEL mutation which is carried by roughly half of all cystic-fibrosis patients. It found that a combination of ivacaftor with lumicaftor was effective in improving lung function between 2.6 and 4 per cent.

A Queen's University doctor has played a key role in a major breakthrough to change the lives of cystic fibrosis sufferers.

Related Articles


Queen's University's Professor Stuart Elborn, an international authority in respiratory medicine, with colleagues from the United States and Australia have led pivotal studies of a new treatment for people with Cystic Fibrosis. The combination therapy, developed by Vertex (a Boston, USA company), improves lung function and reduces hospitalizations for patients with the most common type of cystic fibrosis.

Two Phase 3 studies of the drugs ivacaftor and lumacaftor, which included over 1,100 patients worldwide, built on previous studies of ivacaftor in patients with G551D and other related mutations. Ivacaftor is the first drug to treat the underlying causes of cystic fibrosis rather than just its symptoms and is currently approved for patients with the 'celtic gene' mutation carried by about four per cent of all patients and 10-15 per cent of patients in Ireland. This therapy is a leading example of precision medicine, where treatment is based on a test for genetic mutations.

This recent trial looked at the treatment of patients with two copies of the F508DEL mutation which is carried by roughly half of all cystic-fibrosis patients. It found that a combination of ivacaftor with lumicaftor was effective in improving lung function between 2.6 and 4 per cent.

Cystic fibrosis is a fatal lung disease affecting 75,000 children and adults world-wide, and is caused by inherited genetic mutations that vary among different patient groups.

Professor Stuart Elborn, Dean of the School of Medicine, Dentistry and Biomedical Sciences at Queen's University, and the European lead on the study, said: "This is a very significant breakthrough for people with cystic fibrosis. While we had previously found an effective treatment for those with the 'celtic gene' this new combination treatment has the potential to help roughly half of those with cystic fibrosis, those who have two copies the F508DEL mutation.

"This is another example of how Queen's scientists are working internationally to change lives around the world."

The study was led by a team from Europe -- Dr Stuart Elborn, Queen's University Belfast, the USA -- Dr Bonnie Ramsey, and Dr Michael Boyle, and Australia -- Dr Claire Wainwright. Twelve patients from the Northern Ireland Adult and Paediatric Cystic Fibrosis Centres participated in the study. The local investigators were Dr Damian Downey and Dr Alistair Reid from the Belfast Health and Social Care Trust.


Story Source:

The above story is based on materials provided by Queen's University, Belfast. Note: Materials may be edited for content and length.


Cite This Page:

Queen's University, Belfast. "Major cystic fibrosis breakthrough made: Drug combination improves lung function." ScienceDaily. ScienceDaily, 25 June 2014. <www.sciencedaily.com/releases/2014/06/140625132106.htm>.
Queen's University, Belfast. (2014, June 25). Major cystic fibrosis breakthrough made: Drug combination improves lung function. ScienceDaily. Retrieved November 23, 2014 from www.sciencedaily.com/releases/2014/06/140625132106.htm
Queen's University, Belfast. "Major cystic fibrosis breakthrough made: Drug combination improves lung function." ScienceDaily. www.sciencedaily.com/releases/2014/06/140625132106.htm (accessed November 23, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, November 23, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Ebola-Hit Sierra Leone's Late Cocoa Leaves Bitter Taste

Ebola-Hit Sierra Leone's Late Cocoa Leaves Bitter Taste

AFP (Nov. 23, 2014) The arable district of Kenema in Sierra Leone -- at the centre of the Ebola outbreak in May -- has been under quarantine for three months as the cocoa harvest comes in. Duration: 01:32 Video provided by AFP
Powered by NewsLook.com
Don't Fall For Flu Shot Myths

Don't Fall For Flu Shot Myths

Newsy (Nov. 23, 2014) Misconceptions abound when it comes to your annual flu shot. Medical experts say most people older than 6 months should get the shot. Video provided by Newsy
Powered by NewsLook.com
WFP: Ebola Risks Heightened Among Women Throughout Africa

WFP: Ebola Risks Heightened Among Women Throughout Africa

AFP (Nov. 21, 2014) Having children has always been a frightening prospect in Sierra Leone, the world's most dangerous place to give birth, but Ebola has presented an alarming new threat for expectant mothers. Duration: 00:37 Video provided by AFP
Powered by NewsLook.com
Could Your Genes Be The Reason You're Single?

Could Your Genes Be The Reason You're Single?

Newsy (Nov. 21, 2014) Researchers in Beijing discovered a gene called 5-HTA1, and carriers are reportedly 20 percent more likely to be single. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins