Nov/Dec 2009

3,500+ Scientists & Caregivers Tackle CF at International Conference - Connections - Nov/Dec 2009 - Cystic Fibrosis Foundation
NIH Director Dr. Francis Collins gave
the keynote address at the 2009
North American CF Conference.

NIH Director Francis Collins joins Top CF Scientists at International Conference

More than 3,500 CF scientists and medical professionals from around the world gathered this October to discuss the future of CF research and care at the CF Foundation’s annual North American Cystic Fibrosis Conference (NACFC) in Minneapolis, Minn.

This year’s conference came at a pivotal point in the history of cystic fibrosis. For the first time, promising new treatments are in development to treat the underlying cause of CF. Several of these potential drugs are in advanced stages of clinical testing.

During the three-day conference, experts focused on recent scientific progress and other pressing topics in CF research and care, including:

  • Improving the health of people with CF using cutting-edge healthcare quality improvement tools.
  • CF care and treatment strategies for adults.
  • Coping with barriers to health care coverage for people with CF.

In addition to discussing the best approach for finding new treatments and a cure, scientists reflected on the remarkable progress that has been made since the discovery of the CF gene. Francis Collins, M.D., Ph.D., director of the National Institutes of Health, and co-discover the CF gene, revealed how big a challenge the hunt was.

“It was like trying to find a needle in a haystack. There were no neon signs that said ‘look here!’ A lot of people said, this is just too hard. It may seem impossible now to imagine, but I think there was a substantial sense amongst many people that this was not a solvable problem, at least not in our lifetime.”

Fortunately, Collins and his co-discoverers Lap-Chee Tsui, Ph.D. and Jack Riordan, Ph.D. were not deterred. They discovered the gene in 1989, bringing much excitement to the CF community and hope for a cure.

Finding the CF gene allowed scientists to better understand cystic fibrosis. They learned about a protein called CFTR. In CF, this protein malfunctions or is missing entirely, leading to thick, sticky mucus in the lungs and other organs.

Without the gene discovery, researchers would not be as knowledgeable about CFTR as they are today. And without that knowledge, they would not have been able to develop the therapies that target CFTR in hopes of correcting the underlying cause of the disease.

Although there is no cure, researchers have made leaps and bounds in the treatment of CF. Currently, the median predicated age of survival for CF is more than 37 years — more than double of what it was 25 years ago.

“Once we gained the extraordinary amount of scientific information from the discovery of the gene, we didn’t sit on our laurels,” said Bonnie Ramsey, M.D., professor of pediatrics at the University of Washington and director of the CF Therapeutics Development Network Coordinating Center.

“We’ve used that information to really understand what the CFTR protein does, what’s wrong with the faulty CFTR protein in CF, and then we translated it into actual treatments. The fact that we have made that progress is amazing.”

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