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Cystic Fibrosis Drug Combo May Be Less Effective Than Hoped
Date:7/23/2014

By
HealthDay Reporter

WEDNESDAY, July 23, 2014 (HealthDay News) -- A powerful drug combo may not be as effective against cystic fibrosis as previously thought. New lab-based research on human cells suggests that one of the medications might stop the other from working properly.

However, this study's findings aren't definitive, and there's still hope for the medications known as ivacaftor (brand name Kalydeco) and lumacaftor, according to the study's senior author.

"The development of drugs like ivacaftor and lumacaftor is undoubtedly a step forward, but our study suggests that more work will need to be done before we can realize the full potential of these drugs," said Martina Gentzsch, an assistant professor with the department of cell biology and physiology at the University of North Carolina, Chapel Hill.

"Fortunately, we now have a better understanding of some of the potential pitfalls to these drug combinations and a means to test alternative strategies to make these drugs more effective," said Gentzsch.

For its part, the Vertex pharmaceutical company, which produces both medications, recently released the results of a phase 3 clinical trial on the drug combo, involving patients with cystic fibrosis. The findings, based on use of the drug combination by more than 1,100 patients, "showed consistent and statistically significant improvements in lung function and other measures of the disease," Vertex said in a statement.

Alan Smyth, a professor of child health and division head at the University of Nottingham in England, agreed that initial research in people, not on cells in the laboratory, was more promising. The drug combo seems to work, he said, and newer drugs of a similar type may help scientists improve on their performance.

However, Smyth cau
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