CF Conference Draws More than 3,000 from Around the World
November 8, 2006
The 20th annual North American Cystic Fibrosis Conference (NACFC) convened November 2 in Denver, Colo. with more than 3,000 scientists and physicians from around the world.
The overarching theme of the conference was described by CF physician/scientist Dr. Pam Davis from Case Western Reserve University as “Turning Promises into Reality” and attendees spent the next four days immersed in the basic science and clinical research needed to accomplish this goal. From describing new techniques for finding novel drugs to correct the CFTR protein that is defective in people with CF, to announcing results of on-going clinical trials designed to correct the defect at the cellular level, each session focused on delivering on the promise of health and long life to everyone with CF.
Early results from several clinical trials were described. The compound known as MOLI1901, which may help fix the faulty salt balance in CF cells, seemed to improve lung functions slightly in volunteers in a small Phase 2A trial. The compound referred to as PTC124, which is designed to repair CFTR “stop” or nonsense mutations found in about 5 percent of people with CF, also improved lung function in trials conducted in Israel. However, confirmation of these results as well as testing other drugs in the development pipeline requires more volunteers.
In a major address to the NACFC attendees, Dr. J.P. Clancy from the University of Alabama described ways the CF Foundation is working to try to reach the 3,000-plus volunteers with CF needed in the next two years alone to complete scheduled trials for new potential drugs. Without these volunteers, new therapies cannot be tested and approved.
In the continuing search for new therapies, scientists also presented results centered on new technologies with exotic names like "in silico drug discovery," “proteomics,” and "metabolomics." The in silico method, for example, uses high-powered computer modeling to design drugs that bind and interact very specifically to correct defective CFTR and improve health from the cellular level on up. Many cutting-edge technologies were enthusiastically discussed as scientists compared notes on ways to find new targets for drugs to repair the CF defect. “That’s a good question” was frequently heard, as scientists offered suggestions to thorny problems in the research.
In addition to basic and clinical researchers, the NACFC provides an opportunity for nurses, respiratory therapists, and other caregivers to discuss the best ways to deliver on the promise of optimal health to their patients. A rousing discussion of differences in health status of girls and boys with CF occurred; physicians left that session determined to achieve good nutrition and aggressively care for lungs in all people with CF in an attempt to close the “gender gap.” Another set of sessions discussed ways for clinics to emphasize the importance of physical activity in keeping people with CF happy and healthy.
A final talk, presented by renowned CF-adult clinician Dr. James Yankaskas, focused on quality improvement efforts by CF care centers to improve delivery of care, to ensure the best possible chance for long and healthy lives. Dr. Yankaskas predicted that aggressively applying currently available treatments will add years to the lives of people with CF. The uplifting talk was a perfect way to end this year’s NACFC, and researchers and clinicians dispersed to their homes across the globe, re-energized to turn promises of a better life into reality.