More than 3,500 leading cystic fibrosis scientists and health care professionals gathered in Minneapolis, Minn. from Oct. 15 - Oct. 17 to exchange ideas and discuss developments in cystic fibrosis research and care.
The 23rd annual North American Cystic Fibrosis Conference (NACFC) included presentations by researchers who discussed the basic science of CF, and seminars in which physicians talked about advancements in adult care and quality improvement.
The most widely attended sessions of the conference were the three plenary sessions, which included information on basic science research and emerging therapies to treat inflammation and infection in CF lungs.
Scroll down to read summaries of each of the three plenary sessions and watch full-length videos of each presentation.
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Plenary Session 1: Two Decades of CFTR Research: From Gene Discovery to Therapeutic TargetSpeakers: Steven M. Rowe, M.D., M.S.P.H. and Francis S. Collins, M.D., Ph.D.
Steven Rowe, M.D., assistant professor of medicine at the University of Alabama at Birmingham, opened the first plenary of the 23rd NACFC with his presentation on how CF researchers are moving discoveries about the faulty CF gene from the laboratory into clinical trials in hopes of treating CF patients.
Rowe described how understanding the basic defect led to rapid identification of small molecules (potential drugs) that in the laboratory can correct the defect. Even more exciting is the fact that these therapies, which are now being tested in adults and children with CF, are showing positive results in late-stage clinical trials.
Rowe presented the results of several biotech companies which have therapies aimed at treating the basic defect in clinical trials, including Vertex Pharmaceuticals and PTC Therapeutics.
Francis Collins, M.D., Ph.D., director of the National Institutes of Health and co-discoverer of the CF gene, took the stage after Rowe for the keynote address. Collins spoke to the audience on the history of CF research.
He described the tremendous progress that has been made in the last 20 years, from the seemingly impossible task of finding the CF gene without knowing its function, to today’s efforts to identify a way to repair the defective protein with oral therapies. In each of these endeavors, Collins described how the CF community has led the way, demonstrating one success after another.
One of the leading scientists of our time, Collins then led the audience in a song as he strummed a guitar. The song, “Dare to Dream,” was written by Collins about the hopes for a promising future for all those with CF.
Plenary Session 2: Inflammation and Infection: Update on the Pipeline
Speakers: James F. Chmiel, M.D. and Lisa Saiman, M.D., M.P.H.
The second plenary focused on inflammation and infection in CF lung disease. James F. Chmiel, M.D., assistant professor of pediatrics at Case Western Reserve University School of Medicine at Rainbow Babies and Children’s Hospital in Cleveland spoke first.
Chmiel described the early onset of inflammation in CF, which plays a key role in lung damage. Because reducing inflammation in CF lessens lung damage, the pipeline of potential CF therapeutics contains several anti-inflammatory treatments, including a number that are already FDA-approved for other conditions. In addition, Chmiel described other approaches CF researchers are exploring to address inflammation in CF.
The second part of the plenary session was presented by Lisa Saiman, M.D., professor of clinical pediatrics at Columbia University in New York and a leading expert on CF lung infections.
Saiman presented exciting results showing how early antibiotic therapy is delaying the onset of chronic infection by P. aeruginosa, a common bacteria that leads to lung infections in people with CF. Saiman noted that rapid treatment of lung infections improves overall lung function in people with CF.
Saiman also noted that antibiotics continue to be a mainstay of good CF care, and expanding antibiotic choices is a crucial effort in therapeutic development. She described promising results from a number of antibiotic trials, including studies of inhaled levofloxacin, tobramycin powder, slow-release amikacin and more.
Plenary Session 3: Early Airway Infection in Young Children with CF – What is the Optimal Therapy?
Speakers: Bonnie W. Ramsey, M.D.; Claire E. Wainwright, M.D., M.B.B.S.; and George Z. Retsch-Bogart, M.D.
The final plenary of the 2009 NACFC focused on improving the health of children with CF by preventing chronic lung infections with aggressive antibiotic treatment.
Bonnie W. Ramsey, M.D., professor of pediatrics at the Children's Hospital and Regional Medical Center in Seattle began the presentation by describing CF lung disease and showing evidence that early antibiotic therapy helps minimize lung disease. Because the most effective and safe antibiotic treatment is unknown, clinical trials were designed to determine how best to use antibiotics to treat lung infections.
The next two speakers, Claire Wainwright, M.D., of Royal Children's Hospital, Brisbane, Queensland, Australia and George Z. Retsch-Bogart, M.D. of the University of North Carolina at Chapel Hill presented early results from two such trials.
The complex trials were designed to identify whether regular courses of antibiotics were more effective in preventing lung infections than case-by-case courses of antibiotics when CF patients had lung infections.
After the data was shown, Ramsey returned to the stage to summarize the findings, stating that most young children with CF can clear P. aeruginosa lung infections when treated with antibiotics. Ramsey suggested that while antibiotic therapy is crucial, using regular cycles of antibiotics in hopes of preventing lung infections may be neither necessary nor beneficial.
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Updated 10/23/09
