Corporate Banner
Satellite Banner
Pharma Outsourcing
Scientific Community
 
Become a Member | Sign in
Home>News>This Article
  News
Return

Major Cystic Fibrosis Breakthrough

Published: Thursday, June 26, 2014
Last Updated: Wednesday, June 25, 2014
Bookmark and Share
Combination of ivacaftor with lumicaftor improves lung function and reduces patients hospitalizations.

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

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.


Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 3,900+ scientific posters on ePosters
  • More than 5,300+ scientific videos on LabTube
  • 35 community eNewsletters


Sign In



Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into TechnologyNetworks.com you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Drug Testing without the Pain at Queen’s
Microneedles on a sticking-plaster-like patch may be the painless and safe way doctors will test for drugs and some infections in the future.
Wednesday, December 04, 2013
£2.25 Million Trial for Vascular Dementia Treatment
Researchers aim to find out if a drug for blood pressure could be effective in treating vascular dementia in a new £2.25million clinical trial.
Wednesday, December 04, 2013
Scientific News
BioCision Forms MedCision
The new company will focus on technologies for the management and automation of vital clinical processes.
First New HIV Vaccine Study for Seven Years Begins
South Africa hosts historic clinical trial of experimental HIV vaccine aiming to safely prevent HIV infection.
Study to Assess Shorter-Duration Antibiotics in Children
Physicians plan a clinical trial to evaluate whether short course anti-biotics are effective at treating CAP in children.
Cancer Gene Predicts Treatment Response in Leukaemia
Study indicates the patients suffering from a lethal for of acute myeloid leukemia may live longer when receiving milder chemotherapy drugs.
Injectable Biologic Therapy Reduces Triglycerides
Study finds first-of-its-kind therapy promising for patients with high triglycerides, cholesterol.
Testing Zika Vaccine in Humans Begins
The first of five planned clinical trials to test ZPIV vaccine in humans has begun.
Combination Therapy Improved Chemoresistance in Ovarian Cancer
The study demonstrates how an existing class of targeted therapies could be used to potentiate the tumor suppression induced by cisplatin.
Gene Therapy for Blistering Skin Disease Shows Promise
Grafting genetically altered skin onto patients’ chronic wounds marks the first time skin-based gene therapy has been demonstrated to be safe and effective in humans.
Alzheimer’s Treatment Moves a Step Closer
Merck scientists have reported the discovery of verubecestat, a structurally unique, orally bioavailable small molecule that has been shown to target the most visible sign of the disease in the brain.
Lynparza Phase III Progression-Free Survival Benefit
AstraZeneca announces results from Phase III SOLO-2 trial designed to determine the efficacy of Lynparza as a monotherapy for ovarian cancer.
Scroll Up
Scroll Down
SELECTBIO

SELECTBIO Market Reports
Go to LabTube
Go to eposters
 
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
3,900+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
5,300+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!