UAB Investigators Part of Exciting Cystic Fibrosis Trial

A new drug for cystic fibrosis (CF) has shown promise in treating a basic manifestation of the disease, according to results released today by the Cystic Fibrosis Foundation and Vertex Pharmaceuticals Incorporated.

March 27, 2008

• New drug corrects CF defect

• UAB treats first patient worldwide

BIRMINGHAM, Ala. - A new drug for cystic fibrosis (CF) has shown promise in treating a basic manifestation of the disease, according to results released today by the Cystic Fibrosis Foundation and Vertex Pharmaceuticals Incorporated. UAB's (University of Alabama at Birmingham) Gregory Fleming James Cystic Fibrosis Research Center was one of nine sites testing the drug in CF patients. In May 2007, scientists at UAB were the first to treat a CF patient with the new drug, VX-770.

The CF Foundation reports that patients who took VX-770 for 14 days showed significant improvements in several key indicators of cystic fibrosis, including lung function, nasal potential difference measurements and sweat chloride levels. The findings suggest that the drug improves function of what is known as the faulty CFTR protein.

"Patients with CF have a defective chloride channel protein that in effect causes a door to shut too tightly in that channel," said Steven M. Rowe, M.D., assistant professor of medicine and lead investigator of the trial at UAB. "The drug seems to repair the protein, so that the door opens and closes more properly."

This is the first time that any potential therapy has improved the abnormal sweat chloride levels in a person with CF.  Excessive sweat chloride is a key clinical indicator of CF.  The sweat test is the traditional diagnostic test for CF.

VX-770 was discovered in a collaboration between the Cystic Fibrosis Foundation (CFF) and Vertex using a cutting edge technology known as high throughput screening, a very advanced technique for drug discovery. The Foundation has invested $79 million in the project.

"These early results are an extraordinary endorsement of our hypothesis...that small molecules can correct the basic defect and affect the clinical indicators of cystic fibrosis," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation.

Part One of the Phase 2a trial of VX-770 studied 20 CF patients over a 14-day period. Part Two of the study, in which UAB will participate, is expected to begin in the second quarter of 2008.

CF is a life-threatening genetic disease that affects about 30,000 people in the United States and 70,000 worldwide.  It is caused by a genetic mutation that results in a malfunctioning or missing protein that causes an imbalance of salt and water transport. This imbalance causes a cascade of mucus plugging, infection and inflammation in the lungs and other organs.