Navigation Links
Study shows new medication effectively treats underlying cause of cystic fibrosis
Date:11/2/2011

SEATTLE -- A new study has confirmed that the drug, ivacaftor (VX-770), significantly improves lung function in some people with cystic fibrosis (CF). The results of the phase III clinical trial study, "A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation," led by Bonnie W. Ramsey, MD of Seattle Children's Research Institute and the University of Washington, were published today in the New England Journal of Medicine.

Ivacaftor, also known as VX-770, was developed by Vertex Pharmaceuticals with financial support from the Cystic Fibrosis Foundation. The oral medicine targets the defective protein produced by the gene mutation called G551D that causes CF. Researchers found that patients carrying G551D approximately four per cent of all CF patients who were treated with VX-770 showed a 17 per cent relative improvement in lung function that was sustained over the course of 48 weeks.

Additionally, patients with G551D treated with VX-770 showed improvements in other areas critically important to the health of people with CF. Study participants experienced significant reductions in sweat chloride levels indicating an improvement in the body's ability to carry salt in and out of cells a process which when defective leads to CF. They also experienced decreased respiratory distress symptoms and improved weight gain. Those who received VX-770 gained on average seven pounds compared to those in the placebo group who gained approximately one pound. This is significant because many people with CF have difficulty gaining and maintaining weight due to reduced lung function and chronic infection.

"Our study shows that we are now able to improve the quality of life for cystic fibrosis patients with the G551D mutation with the administration of VX-770," said Dr. Ramsey, director of the Center for Clinical and Translational Research at Seattle Children's Research Institute and endowed chair in Cystic Fibrosis (CF) in the Department of Pediatrics at the University of Washington School of Medicine.

Dr. Ramsey and co-investigators evaluated lung function in patients 12 years or older who carry at least one copy of the G551D mutation. The study included 161 patients at multiple healthcare centers who received at least one dose of VX-770 or placebo. The study is the third and final in a series designed to assess VX-770's effectiveness and safety before it is approved for public use.

Approximately 30,000 children and adults in the United States and 70,000 people worldwide have CF. The disease is caused by a defective gene that affects many parts of the body, but is especially harmful to the lungs and pancreas. The gene mutation causes very thick, sticky mucus to clog the lungs and often cause life-threatening infections. In the 1950s, few children with CF lived beyond age five. Today, advances in research and medical treatments have made it possible for people with CF to live well into their 30s, 40s and beyond.


'/>"/>
Contact: Louise Maxwell
press@seattlechildrens.org
206-987-4500
Seattle Children's
Source:Eurekalert

Related biology news :

1. Long-term study shows effect of climate change on animal diversity
2. £2 million study to reveal workings of dementia genes
3. New study looks to define evangelicals and how they affect polling
4. CU-Boulder study suggests air quality regulations miss key pollutants
5. Researchers study acoustic communication in deep-sea fish
6. Study reveals homeowner perceptions in fire-prone areas
7. Researchers study how pistachios may improve heart health
8. Study: urban black bears live fast, die young
9. New study indicates link between weight gains during pregnancy and dieting history
10. Study reveals specific gene in adolescent men with delinquent peers
11. Sweat it out: UH study examines ability of sweat patches to monitor bone loss
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/7/2016)... -- Syngrafii Inc. and San Antonio Credit Union (SACU) ... Syngrafii,s patented LongPen™ eSignature "Wet" solution into SACU,s ... in greater convenience for SACU members and operational ... document workflow and compliance requirements. Logo ... Highlights: ...
(Date:6/2/2016)... , June 2, 2016 The Department ... has awarded the 44 million US Dollar project, for the ... Vehicle Plates including Personalization, Enrolment, and IT Infrastructure , ... in the production and implementation of Identity Management Solutions. Numerous ... however Decatur was selected for the ...
(Date:6/2/2016)... Perimeter Surveillance & Detection Systems, Biometrics & ... & Other Service  The latest report from ... of the global Border Security market . Visiongain ... billion in 2016. Now: In November 2015 ... and hardware technologies for advanced video surveillance. ...
Breaking Biology News(10 mins):
(Date:6/27/2016)... (PRWEB) , ... June 27, 2016 , ... Parallel ... clinical trials, announced today the Clinical Reach Virtual Patient Encounter CONSULT module ... circle with the physician and clinical trial team. , Using the CONSULT module, patients ...
(Date:6/27/2016)... -- Liquid Biotech USA , Inc. ... Research Agreement with The University of Pennsylvania ("PENN") ... patients.  The funding will be used to assess ... outcomes in cancer patients undergoing a variety of ... to support the design of a therapeutic, decision-making ...
(Date:6/24/2016)... , June 24, 2016 Epic ... sensitively detects cancers susceptible to PARP inhibitors by ... tumor cells (CTCs). The new test has already ... therapeutics in multiple cancer types. Over ... DNA damage response pathways, including PARP, ATM, ATR, ...
(Date:6/23/2016)... ... June 23, 2016 , ... Mosio, a leader in ... Trials Patient Recruitment and Retention Tips.” Partnering with experienced clinical research professionals, Mosio ... practical tips, tools, and strategies for clinical researchers. , “The landscape of how ...
Breaking Biology Technology: