Plenary Session 1: A Cure for All: Leaving No One Behind
Speakers: Eric Sorscher, M.D. and Mitchell Drumm, Ph.D.
Eric Sorscher, M.D., the Georgia Research Alliance Eminent Scholar and Hertz Professor in Cystic Fibrosis Research at Emory University in Atlanta, provided an overview of the development of potential treatments that would target the underlying cause of cystic fibrosis and its symptoms. If clinical trials for the second generation of CFTR modulators are successful, nearly 95 percent of people with CF will have a drug that targets the defective CFTR protein that causes their symptoms. For the remaining 5 percent of people with CF who do not produce any CFTR protein, researchers are testing compounds and exploring other potential treatments to ensure that no one is left behind. Dr. Sorscher said that working on treatments that reduce excessive inflammation, fight chronic infections and improve airway clearance remain top priorities until a cure can be found.
Mitchell Drumm, Ph.D., a professor of pediatrics and genetics for Case Western Reserve University School of Medicine, spoke about gene editing and other approaches to a one-time CF cure.
Watch plenary 1. (Duration: 1 hour, 37 minutes)
Plenary Session 2: Clinical Research: A Worldwide CF Community Effort
Speakers: Patrick Flume, M.D., Kris De Boeck, M.D., Ph.D. and Patricia Burks, M.A., R.N., C.C.R.C.
Patrick Flume, M.D., professor of medicine and pediatrics at the Medical University of South Carolina, said it is going to take a tremendous team effort between doctors, researchers and people with CF to advance new therapies through the more than 50 clinical trials underway. Because there are less than 35,000 people with CF in the United States, it is difficult to recruit enough people who meet all the eligibility criteria for a trial, he said.
Kris De Boeck, M.D., Ph.D., director of the Pediatric Pulmonary and Infectious Diseases program at the University of Leuven in Belgium, explained how the CF Foundation is helping to expand the pool of people who can enroll in trials by improving the performance of clinical trials in the United Kingdom, Europe and Australia.
Patricia Burks, M.A., R.N., C.C.R.C., director of Clinical Trial Affairs at the CF Foundation, moderated a panel discussion with three members of the CF community to discuss why they think it is important that everyone with CF has the opportunity to participate in clinical trials.
Watch plenary 2. (Duration: 1 hour, 9 minutes)
Plenary Session 3: You Can Observe a Lot Just Watching: Lessons from the CF Foundation Patient Registry
Speaker: Wayne Morgan, M.D.
Wayne Morgan, M.D., a pediatric pulmonologist at the University of Arizona in Tucson, talked about the connection between CF care and the Patient Registry, and introduced a new way for people with CF, along with their families, to help shape the research conducted using the Registry. Dr. Morgan reviewed the role of the Registry in improving our understanding of cystic fibrosis, the care of people with CF and opportunities to improve care. He also introduced a new initiative called the Insight CF Registry Research Project, in which anyone in the CF community can submit a research question that can be answered by mining data in the Registry.
Watch plenary 3. (Duration: 1 hour, 6 minutes)