Archives

Defining the New CF in the Era of Highly Effective Modulators

Speaker: Jennifer L. Taylor-Cousar, M.D., MSCS, ATS

In this session, the latest research into highly effective CFTR modulators will be highlighted, providing and in-depth overview of the CFTR modulator research landscape one-year post-approval of Trikafta. The speaker will describe the ongoing and upcoming studies looking at the holistic impact of highly effective CFTR modulation on the lives of people with CF. Finally, there will be a review of on-going and upcoming studies for the development of new modulators as well as the potential impact of modulators on complications.

"Emerging Technologies for CFTR Restoration in All People With CF"

Speaker: Marie Egan, M.D.

Genetic therapies, including RNA therapies, gene therapies, and gene editing, hold the key to a cure for cystic fibrosis and are moving toward the clinical realm at an unexpected pace. Moving from the promise of these technologies to the reality of effective therapies for the underlying cause of the disease for all people with CF and a cure for CF will require time, tenacity, and vision.

Marie Egan, MD, from the Yale School of Medicine, reviews the power of genetic therapies, outlines the obstacles and challenges that lie ahead, and discusses what success looks like as we move toward therapies that address the underlying defect for all people with CF and a cure for cystic fibrosis. 

"Entering the Era of Highly Effective Modulator Therapy"

Speaker: Jane C. Davies, M.D.

In this session, Professor Jane Davies from the National Heart & Lung Institute, Imperial College in London, describes the progress and the promise of highly effective CFTR modulator therapy for people with CF. She highlights the long-term benefits such therapies have had on the relatively small proportion of the CF community for whom a highly effective modulator has been available for several years -- those with the gating mutation G551D who are on ivacaftor -- and uses this evidence to describe the impact we might expect from a triple-combination drug in a larger population. The additional opportunities posed by introducing CFTR functional restoration in early life is illustrated with pre-school and infant data.

"Evolution of CF Care: Innovation & Impact"

Speaker: Peter Mogayzel, M.D., Ph.D.

In this session, Dr. Peter Mogayzel walks us through past challenges and how our care model has met those challenges and built upon what we've learned to prepare for the future. As we enter a new era of therapeutics, now is the moment to consider what should shape how CF care evolves to meet the changing needs of people with CF and their families. Following his presentation, Sue Sullivan, RN, from Oregon Health & Science University; Terri Laguna, MD, MSCS, from Ann and Robert H. Lurie Children's Hospital of Chicago; and Cindy Brown, MD, of Indiana University, join Dr. Mogayzel for a panel discussion about the factors that should influence how CF clinical care evolves to support the needs of everyone with CF.

"Improving Outcomes of Infections in the Age of CFTR Modulators"

Speaker: Lisa Saiman, M.D., M.P.H.

In this session, Dr. Lisa Saiman will address the challenges of infections in people with CF and discuss why infections remain a major focus of the CF community, even in the era of effective CFTR modulators. She will provide an overview of CF infections, describe ongoing studies to increase our understanding of CF microorganisms and their optimal treatment, and discuss current strategies to develop new anti-infective agents to treat even the most difficult infections.

"Anti-inflammatories & Mucociliary Clearance Therapies in the Age of CFTR Modulators"

Speaker: Felix Ratjen, M.D., Ph.D.

This plenary will review the effect of CFTR dysfunction and CFTR restoration on both inflammation and mucociliary clearance in individuals with CF. Current and future therapeutic strategies to address inflammation and mucociliary clearance will be discussed, as well as how these strategies have potential to benefit both those patients using CFTR modulators and those not eligible for modulators.