Corporate Banner
Satellite Banner
Genomics
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 3,300+ scientific posters on ePosters
  • More than 4,900+ 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

Big Data Can Save Lives
The sharing of genetic information from millions of cancer patients around the world could be key to revolutionising cancer prevention and care, according to a leading cancer expert from Queen's University Belfast.
Tuesday, May 17, 2016
Bowel Cancer Breakthrough May Benefit Thousands of Patients
Researchers at Queen’s University have discovered how two genes cause bowel cancer cells to become resistant to treatments used against the disease.
Monday, July 21, 2014
Scientific News
Gene Therapy for Metabolic Liver Diseases
Researchers have tested gene therapy in pigs from hereditary tyrosinemia type 1, with corrected liver cells being transplanted into the diseased liver.
Gene Terapy for Muscle Wasting Developed
New gene therapy could save millions of people suffering from muscle wasting disease.
Gene-Editing 'Toolbox' Targets Multiple Genes Simultaneously
Researchers have designed a system that modifies, or edits, multiple genes in a genome at once while minimising unintentional effects.
Discovering the First Farmers
Genetic analyses reveal a collection of highly distinct groups in the Near East and Europe at the dawn of agriculture.
Fighting Cancer Through Protein Pathways
Researchers have found a new drug target within a protein production pathway critical to regulating growth and proliferation of cells.
Mutations in DNA-Repair Genes Found in Advanced Prostate Cancers
New findings indicate that nearly 12% of male advanced prostate cancer sufferers have inherited mutation in DNA-repair genes.
Ice Bucket Challenge Instrumental in Gene Discovery
Donations from the ALS Ice Bucket Chellenge allowed for the largest-ever study of inherited ALS, which identified a new ALS gene.
Triple-Action Therapy Patch Shows Promise
Patch that delivers drug, gene, and light-based therapy to tumor sites shows promising results in mice.
Cancer Gene-Drug Combinations Ripe for Precision Medicine
The study aims to expand the number of cancer gene mutations that can be paired with a precision therapy.
Targeting BRAF Mutations in Thyroid Cancer
Treating metastatic thyroid cancer patients harboring a BRAF mutation with vemurafenib showed anti-tumor activity in a third of patients.
Skyscraper Banner

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
3,300+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
4,900+ scientific videos
Close
Premium CrownJOIN TECHNOLOGY NETWORKS PREMIUM FOR FREE!