Conquering Cystic Fibrosis

November 3, 2008 12:00:00 AM PST
More than 70,000 people worldwide have cystic fibrosis (CF), the most common genetic disease affecting Caucasians. Though treatments have improved, many patients with CF don't live past their 37th birthday. Now, a new drug is showing promise in treating a basic defect of the disease and giving hope to patients who fight a battle with CF every day.Facing a pile of pills is part of 19-year-old Elyse Campbell's daily routine. Breathing isn't easy either ? twice a day Campbell needs a lung treatment.

"The vibration breaks up the mucus and helps me cough it out so that I can breathe better," Campbell explained to Ivanhoe.

It's all part of living with cystic fibrosis, a disease linked to a faulty protein called CFTR

People with CF suffer from an imbalance of water and salt. It causes thick mucus and inflammation in the lungs and other organs, sometimes leading to infection. There is no cure but there is new hope.

Pulmonologist Steven Rowe, M.D., M.S.P.H., a pulmonologist at the University of Alabama at Birmingham, is leading a research team that's testing a new drug called VX-770.

"It's one of the more exciting things that has happened in CF research," Dr. Rowe told Ivanhoe.

CF patients who took the drug for two weeks showed significant improvement in lung function and sweat chloride levels, indicating the drug may fix the faulty protein.

"It really allows us to be very optimistic that our strategies to try and attack the underlying defect of cystic fibrosis might bear fruit in the improved quality of life for people living with CF," Dr. Rowe explained.

Now, as the VX-770 trial begins the next phase, a very excited Campbell is among the participants.

"If I didn't have to deal with this for at least a day, that would be amazing," she said.

The cystic fibrosis foundation has invested $79 million in the VX-770 research project. Patients like Campbell hope even if it doesn't prove to be a cure, it will help make life easier for her thousands of others with cystic fibrosis.

FOR MORE INFORMATION, PLEASE CONTACT:
University of Alabama at Birmingham
Bob Shepard, Public Relations
bshep@uab.edu
research.dom.uab.edu


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