New Drug Helping to Reduce Cystic Fibrosis Symptoms

Ivacaftor reduced cystic fibrosis symptoms with G551D mutation

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) Cystic Fibrosis is a disease that runs in a family. A new drug targeting a specific mutation has been successful so far in helping to treat cystic fibrosis.

A new study involving a drug, Ivacaftor, for cystic fibrosis has been successful in preliminary tests on humans. The drug targets the specific gene mutation that causes cystic fibrosis. More studies are needed but researchers are optimistic in its potential to treat cystic fibrosis.

"Ask you doctor about clinical trials for cystic fibrosis treatments."

Cystic Fibrosis is a genetic condition that is caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), which is a protein in the body. It affects the lungs causing excess mucus to build up in the lungs. This can lead to difficulty breathing, disability, respiratory failure and ultimately death.

The average age of survival for individuals diagnosed with cystic fibrosis is approximately 37 years.

Ivacaftor is a new drug that targets a specific gene mutation that causes cystic fibrosis, G551D-CFTR. Ivacaftor works by restoring the normal function of the G551D gene.

The Ivacaftor study was conducted by the Lung Institute of Western Australia. It involved 167 cystic fibrosis patients aged 12 and over, 84 patients received one dose of Ivacaftor every 12 hours and 83 patients receiving a placebo. Patients were monitored for 48 weeks.

Lung function improved in as little as two weeks. There was a 55 percent decrease in pulmonary flare-ups in cystic fibrosis patients who were being treated with Ivacaftor. Improvement in weight gain and a decrease in sweat was also noticed in Ivacaftor patients.

All improvements due to Ivacaftor were sustained over the course of 48 weeks.

While the G551D mutation is not the most common source for cystic fibrosis, the effectiveness of Ivacaftor has researchers optimistic about finding a drug to help treat the F508 mutation, which is the most common source of cystic fibrosis.

For now, cystic fibrosis patients who have the G551D mutation have a possible treatment that can improve their symptoms. Future tests are needed to help determine how effective Ivacaftor can be in a larger population. Other tests can evaluate Ivacaftor's role in increasing the age of survival for cystic fibrosis patients.  

This study was published in the November edition of The New England Journal of Medicine.

Reviewed by: 
Review Date: 
November 30, 2011
Last Updated:
November 30, 2011