CF Foundation Hosts Most Successful North American CF Conference

October 30, 2008

This year’s North American Cystic Fibrosis Conference was the largestand most excitingin its 22 year history. More than 3,800 basic scientists and health care professionals from the United States, Canada, United Kingdom, Argentina, New Zealand, Australia, France and many other countries attended plenary sessions, workshops, symposia, short courses, roundtables and luncheons, where they discussed the latest and greatest in CF research and care.

Click on image to watch a slideshow of photos from the NACFC.
Click on image above to watch a slideshow
of photos from the NACFC.
“You can feel the enthusiasm in the hallways and meeting rooms,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “It’s one of the most exciting events I’ve ever participated in in my history at the Foundation, and I think, one of the most exciting events in medicine today.”

The NACFC program featured talks by researchers who presented data on the basic science of CF, and seminars where physicians showed that treating the symptoms of CF with ever-improving drugs is increasing the life expectancy of people with this disease.

A highlight of the conference was data that was presented from a number of studies aimed at treating the basic defect in cystic fibrosis, including those by Vertex Pharmaceuticals, Inc., PTC Therapeutics, Inc., and Inspire Pharmaceuticals, Inc.

Emily Schaller (L) participated in the VX-770 clinical trial and was invited by Dr. Bob Beall to perform with her band, Hellen, at the first plenary session of the NACFC.
Emily Schaller (L) participated in the VX-770 clinical
trial and was invited by Dr. Bob Beall to perform with
her band, Hellen, at the first plenary session
of the NACFC.

Emily Schaller, a 26-year old woman with CF who participated in a clinical trial for Vertex’s compound VX-770, wowed the attendees of the first plenary session when her band, Hellen, performed for the group.

Invited by Beall to join him on stage following his welcoming remarks, Emily also shared her experiences with CF and her gratitude to those assembled in the audience for their work to treat her disease and extend her life.

Scroll down to read more about the three plenary sessions at NACFC, and check back later next week for in-depth conference highlights and video interviews of leading researchers and physicians.

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Plenary Session 1: Preventing CF Lung Disease
Speaker: Jeffrey J. Wine, Ph.D.

In the opening plenary of the 2008 North American CF Conference, Jeffrey J. Wine, Ph.D., director of the CF Research Laboratory at Stanford University, spoke about the progression of lung disease in people with cystic fibrosis.

Wine provided an overview of innate defenses within the lung and described how these are diminished in CF due to absent CFTR function. In his presentation, Wine built a case for aggressive treatment based on earlier pioneering studies in Europe, and suggested that this approach may be a way to preserve lung function.

To view Plenary Session 1:

  1. Watch Professor Wine’s introductory statement (2 mins.)
  2. Then click here to watch to plenary (1 hour, 46 mins.)

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Plenary Session 2: The CFF Pipeline — The Amazing Story of Progress, Hope and Challenge
Speaker: Preston W. Campbell, III, M.D.

In the second plenary session, Preston W. Campbell, III, M.D., executive vice president of medical affairs at the CF Foundation, presented a report of the tremendous progress that has been achieved in the past decade in CF therapeutics development.

Campbell began by stating, “CF is the most amazing story in medicine today.” The steady increase in the median predicted age of survival of people with CF, explained Campbell, is one of the most significant signs of the success of the Foundation’s efforts. Another is the number of rare disease organizations that use the Foundation as a model for therapeutics development.

Campbell described how the varied potential therapies in the Foundation’s pipeline are developed through collaborations with both industry and academia. He also discussed the exciting results from trials of CFTR modulatorssmall compounds designed to improve the function of the defective CFTR protein in the cells of individuals with CF.

While the potential CF drugs show great promise, Campbell reminded the audience that it is important to manage expectations. Only one in five drugs that enters into clinical trials is eventually approved. There will be failures, he concluded, but soon CF will be a disease that people live with, not die of.

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Plenary Session 3: Taking the CF Battle to the Extremes — Healthy Starts with Newborn Screening; Healthy Aging with Improved Adult Care
Speakers: Philip M. Farrell, M.D., Ph.D. and Richard H. Simon, M.D.

The final plenary of the 2008 NACFC was a joint presentation by physicians who have made outstanding advances in the care of people with cystic fibrosis. Philip M. Farrell, M.D., Ph.D., professor of pediatrics and former dean of the school of medicine at the University of Wisconsin, is a tireless advocate for CF newborn screening, and Richard H. Simon, M.D., director of the adult CF care center at the University of Michigan, has extensive experience working with adults with CF.

Farrell opened the plenary with a history of newborn screening for CF, noting the impressive changes that have been achieved in recent years: In 2005, only five states had newborn screening programs; today, 47 states and the District of Columbia are actively screening newborns for CF.

As CF newborn screening becomes a nationwide practice, Farrell suggested focusing on enhancing the quality of the screening process to make it as efficient and error-free as possible.

Infants who are diagnosed with CF through newborn screening often lead to healthier adolescents and adults. In part two of the plenary, Simon described the striking improvements in lung function of adults with CF over the past 20 years.

In addition, Simon suggested that finding ways to help adults adhere to time-consuming treatments and strengthening adult CF care center programs is important for continued improvement. He concluded by suggesting that the CF community should begin to anticipate the needs of an elderly patient population with CF.

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