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International CF Conference Draws Thousands

2007 NACFC
Participants check out the various exhibit booths
between sessions at the NACFC in Anaheim, Calif.
October 8, 2007

The 2007 North American CF Conference opened in Anaheim, Calif., on Oct. 4 with more than 3,000 researchers, physicians and other CF professionals gathering from all over the globe to discuss the latest developments in CF research.
Following are summaries of what was discussed over the course of three days.

Thurs., Oct. 4

The opening plenary address, “From Basic Science to the Clinic,” by Dr. Margarida Amaral, a world-renowned researcher from Lisbon, Portugal, required five ballrooms to accommodate all those interested in hearing about the development of new CF therapies. Dr. Amaral, an expert in protein movement through cells, described many of the new technologies being harnessed in her lab.

The audience was particularly excited by her work on a new class of potential CF therapies known as correctors, which seek to address the basic defect in CF. Dr. Amaral showed correctors produced by Vertex Pharmaceuticals in collaboration with the CF Foundation, improve the production of mature CFTR channel proteins in in vitro cellular systems.  Vertex recently selected a corrector compound for development, the first to emerge from the Foundation’s collaboration with Vertex for corrector research. 

Drug development was also highlighted in other exciting news from the first day of the conference.

  • Gilead Sciences -- Other exciting news from the first day of the conference concerned a drug that is approaching the end of the long clinical trials process. Several hundred attendees crowded into the conference room, lined the walls and sat on the floors to hear the announcement of the results of the Phase 3 AIR-CF1 trial of aztreonam lysine for inhalation (AZLI), an antibiotic being developed by Gilead Sciences to treat Pseudomonas aeruginosa infection in CF airways. The Phase 3 trial demonstrated clear improvements in lung functions and other clinical symptoms in volunteers with CF who received the drug over those who received placebo. While additional Phase 3 trials are continuing, Gilead Sciences is making AZLI available for use by patients with severe disease and limited treatment options.

  • Inspire Pharmaceuticals -- Among other updates, Inspire Pharmaceuticals announced the completion of enrollment in the TIGER-1 trial of denufosol, an inhaled drug designed to activate an alternative ion channel and improve respiratory symptoms. Because this is a yearlong trial, results will not be available for some time. However, to speed the drug development process, the company also announced plans for a second trial, TIGER-2, to begin in early 2008 in the United States, followed by additional enrollment worldwide. Both trials are targeted at individuals with milder lung disease.

  • CF Diagnosis -- In another session on CF diagnosis, researchers addressed important issues related to newborn screening. Because newborn screening for CF is now being implemented throughout most of the United States, infants are increasingly being diagnosed before the appearance of obvious symptoms. While the great majority of cases are easy to diagnose using the sweat test, diagnosis is not as clear in others.

    Dr. Carlo Castellani from Verona, Italy, reported on international efforts to analyze the effect of different mutations in the CF gene on the diagnosis. Dr. Phil Farrell, at the forefront of the CF Foundation’s efforts to develop a clear diagnostic process for infants, as well as for individuals who first show signs of CF in late childhood or adulthood, also spoke.

    The fervid interest in this topic was evident in the many questions posed by physicians who must ultimately explain the diagnosis to families. The new diagnostic recommendations, due to be submitted this month for publication, are sure to generate tremendous interest in the CF community.

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Fri., Oct. 5

  • CF Drug Development Progress -- The second day of the 2007 NACFC opened with a major address, entitled “CF Drug Development: What’s New?” by Dr. Felix Ratjen, a highly respected physician and scientist from the European CF research community, now practicing in Canada.

    In addition to promising results from the clinical trials of denufosol and Moli1901 for treatment of CF lung disease, Dr. Ratjen announced trial plans for the newest entries into the drug pipeline, compounds from Vertex designed to repair broken (mutant) CF proteins.

  • Clinical Trials Participation -- Excitement about this new class of drugs was tempered by the recognition that many trials are slowed by the need for volunteers. Dr. Ratjen explained that though many people with CF have participated in numerous trials throughout the years, the need for volunteers is even greater now: from 500 in 2003 to 6,000 expected in 2009.

    “I Am the Key” buttons and badges worn by conference attendees were a reminder of the CF Foundation’s efforts to bolster clinical trials participation through information pamphlets, a toll-free clinical trials hotline and other outreach methods. Speakers throughout the day also urged people to speak with their care center staff and talk to patients and families about the crucial need to participate in trials and ensure that much-needed treatments continue to be developed.

  • Quality Improvement Initiative -- Participation by people with CF and their families on quality improvement initiatives was also the center of discussions throughout the sessions today. At one symposium, a team from Children’s Memorial Hospital in Chicago described their success in improving outcomes by engaging families actively during clinic visits.

    The CF Foundation strongly supports the concept and convened a first-of-its-kind session for Family Advisory Groups. Physicians, nurses, social workers and families came together to brainstorm ways to help care centers overcome potential barriers to the formation of these groups. Centers with successful groups shared ideas with those who are struggling to get family advisory groups started. Parents in advisory groups spoke about how empowered they felt by making decisions that improve the delivery of care to their children. People left energized with new ideas and ways to form productive teams between families and clinic staff that can result in improving the outcomes for their centers and, of course, improving the health and lives of those affected by CF.

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Sat., Oct. 6

  • Roundtable Discussions -- The final day of the conference began with Rise ’n Shine Roundtable discussions at 7:00 a.m. Conference participants had the chance to break into one of 100 interest groups and delve into a range of topics, including the latest news from the bench, CFTR structure and inflammatory components in the airway. Some roundtable groups focused on care, inviting staff from different care centers to compare notes on a wide variety of issues, such as methods to treat intestinal obstructions or ways to encourage adherence to airway clearance.

  • Newborn Screening -- Later, some of these discussions continued in workshops set up to present data more formally. One workshop was dedicated to discussion of newborn screening programs for CF, now being implemented in 40 states—up from two states just a few years ago. Results from ongoing CF newborn screening programs were presented at the workshop to help new programs improve their ability to detect infants with CF before serious damage is done to their lungs and to help them have much healthier lives.

  • Quality Improvement Initiative -- At the last plenary address of the conference, Dr. Michael Boyle from the Johns Hopkins Hospital spoke about the opportunity provided by the CF care center network and CF Foundation Patient Registry to improve the health of people with CF across the nation. Dr. Boyle described how the information collected by care centers and placed into the Registry was studied to find the centers with the best nutritional or pulmonary ratings. Those centers were then visited and their treatment policies and philosophies were carefully analyzed.

    From this analysis, 10 suggestions were designed that other CF centers might take to improve outcomes in their own patients. Dr. Boyle then showed the remarkable improvements that two centers had made by instituting these changes. As the conference came to a close, CF caregivers left excited by the realization that adopting the suggestions on treatment policies and using tools already available could lead to a real difference in the lives of people with CF everywhere.
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