The Brightest Minds in Science, Medicine and Industry Meet to Discuss Advances and Challenges in CF Research

The Cystic Fibrosis Foundation recently hosted the CFF Research Conference: Pushing the Frontiers, bringing together nearly 150 basic scientists, clinical researchers, graduate students and pharmaceutical company representatives to share information on recent advances and critical challenges in CF drug discovery and development.

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Held May 31 - June 3 in Chantilly, Va., the conference offered a series of interactive sessions across a range of disciplines, with the goal of harnessing the potential of the latest basic science discoveries to inform the development of new treatments that can improve the health and quality of life of people with CF worldwide.

Future conferences will focus on different themes and bring in participants from other areas of the research community. The 2015 conference focused on three main topics:

  • Insights from therapies targeting CFTR (the key protein in cystic fibrosis), such as the impact on the airway biome, promotion of mucociliary clearance and implications for nutritional status 
  • Development of novel strategies, including gene delivery and editing, to replace and repair the defective CFTR protein, regardless of an individual's CF gene mutations
  • Optimizing model systems to accelerate CF research, drug discovery and personalized medicine

In his opening remarks, conference co-chair William R. Skach, M.D., vice president for research affairs at the CF Foundation, told attendees, “Despite the great progress we have seen in recent years, there is still an enormous amount to learn before we will have effective therapy and, ultimately, a cure for every individual with CF. To reach that goal, it is critical to maintain a vibrant research community, accelerate basic research, enable drug discovery and speed therapeutic development in the clinic.”

Attendees also got a firsthand perspective on how their work can make a difference in the daily lives of individuals living with CF. Jonathan Quigley, 33, who was diagnosed with CF as an infant, described the improvements in his health in the three years since he began taking ivacaftor (Kalydeco™) - the first drug to target the root cause of the disease in people with certain rare CF gene mutations. 

Quigley, the married father of two children and a competitive sailor, told the audience, “I still have worries, but I worry a lot less. I look forward to this research, and hope that more medications like Kalydeco continue to come and that one day there is a cure.”

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