33rd Annual North American Cystic Fibrosis Conference

Oct 31, 2019 ‐ Nov 2, 2019

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P1--*Emerging Technologies for CFTR Restoration in All People With CF

Oct 31, 2019 4:30pm ‐ Oct 31, 2019 6:00pm

Genetic-based therapies are discussed in the news nearly every day. These innovative technologies including RNA therapies, gene therapies and gene editing technologies are moving towards the clinical realm at an unexpected pace. They hold the key for 'a cure for all with CF.' However, moving from the promise of these technologies to the reality of effective therapy and cure for all will require time, tenacity and vision. This plenary will take us down that path to review the power of genetic therapies, outline the obstacles/challenges ahead as these therapies move from the pre-clinical space to clinical trials, and discuss what success looks like as we move to a one-time cure.

Learning Objectives:

  • Review emerging genetic therapies and the promise they hold. 
  • Discuss/Describe the unique challenges we face regarding delivering these therapies in CF.
  • Discuss/Describe the incredible advantages and resources the CF community will harness to overcome obstacles.

Plenary Leader(s):

P2--*Entering the Era of Highly Effective Modulator Therapy

Nov 1, 2019 8:30am ‐ Nov 1, 2019 10:00am

In this session, Professor Jane Davies, will describe the progress and the promise of highly effective CFTR modulator therapies for people with CF. She will highlight the long-term benefits such therapy has 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 use 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 will be illustrated with pre-school and infant data. Whilst celebrating the success of highly effective modulator therapy, she will also discuss some of the challenges that remain, including developing treatments for people with rarer mutations and accessing CFTR modulators in certain regions of the world.

Learning Objectives:

  • Summarize the progress to date in CFTR modulator development, including the recent successful Phase 3 trials of triple-combination therapies for people with either a single copy of the F508del mutation (heterozygous) or two copies of F508del (homozygous). 
  • Illustrate the long-term benefits of highly effective modulator therapies, particularly the opportunities of initiating treatment early, by referencing the use of ivacaftor in people with gating mutations.  
  • Identify future challenges in ensuring that highly effective treatments are available to all people with CF.

Plenary Leader(s):
  • Jane Davies, M.D., MBChB, MRCP, Prof - Paed Respirology & Experimental Medicine

P3--*Evolution of CF Care: Innovation & Impact

Nov 2, 2019 9:00am ‐ Nov 2, 2019 10:00am

We count our success in lives, per Brent James, this is how we measure the quality of care we provide people with CF. In this plenary, Dr. Peter Mogayzel will walk 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.

Learning Objectives:

  • Recall how CF care has evolved to meet past challenges.
  • Describe foundations of the CF care model that have enabled us to meet past challenges.
  • Identify key factors to help shape how CF care evolves to meet the changing needs of patients and their families.

Plenary Leader(s): Panelist(s):

S01--CLIN: Endocrine Topics From the EnVision Group

Oct 31, 2019 2:00pm ‐ Oct 31, 2019 3:45pm

This symposium will address major endocrine concerns in CF, specifically CF related diabetes (CFRD), female reproductive health, CF bone disease (CFBD), and vitamin D deficiency. The emphasis will be on practical information related to diagnostic and therapeutic management of CF-endocrine disorders.

Learning Objectives:

  • Discuss the use of continuous glucose monitoring in CFRD.
  • Identify concerns related to female reproductive health for individuals with CF.
  • Describe management strategies for CF bone disease and vitamin D deficiency.

Symposium Chair(s): Symposium Speaker(s):
  • Christine Chan, M.D., Associate Professor of Pediatrics, Children's Hospital Colorado
  • Melissa S. Putman, M.D., M.Sc., Pediatric and Adult Endocrinologist
  • Tanicia J. Daley, M.D., M.P.H., Assistant Professor of Pediatrics, Emory University
  • Kara S. Hughan, M.D., Assistant Professor of Pediatrics, Children's Hospital of Pittsburgh
Symposium Chair(s):

S02--CLIN-APP&D-INF/MIC: *The Impact of Highly Effective CFTR Modulator Therapy on the Natural History of CF

Oct 31, 2019 2:00pm ‐ Oct 31, 2019 3:45pm

This symposium will feature presentations on the clinical outcomes of existing CFTR modulators, focusing on pulmonary and non-pulmonary outcomes.

Learning Objectives:

  • Describe longer-term pulmonary outcomes of CFTR modulator therapy.
  • Characterize non-pulmonary outcomes, including microbiologic and GI outcomes, of CFTR modulator therapy.
  • Discuss the impact CFTR modulator therapy may have on approaches to future treatment regimens in chronic CF care.

Symposium Chair(s): Speaker(s): Symposium Chair(s): Symposium Speaker(s):

S03--ET-NT-GMS: *Fixing What Modulators Won't

Oct 31, 2019 2:00pm ‐ Oct 31, 2019 3:45pm

Currently available modulator therapies target the defective CFTR protein to restore function. Patients with genotypes that are refractory to treatment with extant, and on the horizon, modulators are a challenge for these small molecule approaches - as nonsense and other changes can prevent production of the CFTR protein, which these modulators target. This symposium will focus on emerging science and technologies that point a way forward for this population. The theme will be to: 1) discuss the cells that require CFTR restoration, 2) new ways to deliver non-small molecule payloads to those cells, 3) methods to evaluate success, and 4) an example of such a non-traditional approach in development.

Learning Objectives:

  • Summarize the cell types necessary to restore CFTR. 
  • Evaluate delivery methods for restoration of CFTR. 
  • Identify outcomes for success.

Note: Speaker Daniel Seigwart's presentation was not recorded at his request.

Symposium Chair(s): Symposium Speaker(s):

S04--INF/MIC-NT: Bacteriophage Therapy for Airway Infection: What is the Potential for CF?

Oct 31, 2019 2:00pm ‐ Oct 31, 2019 3:45pm

This session will explore the potential for bacteriophage therapy as an anti-bacterial therapy for airway infection in CF. The session will include three presentations that will focus on (i) the fundamentals of bacteriophage biology, (ii) a brief history of the use of bacteriophages as anti-infective therapy in humans, and (iii) potential obstacles to the use of bacteriophage therapy in CF.

Learning Objectives:

  • Review the basics of bacteriophage biology and ecology with respect to their antibacterial activity.   
  • Describe the history of bacteriophages as antibacterial therapy for human infection. 
  • Summarize the potential obstacles that challenge the use of bacteriophages for therapy of airway infection in CF.

Symposium Chair(s): Symposium Speaker(s):
  • David Pride, M.D., Ph.D., Associate Director of Microbiology
Symposium Chair(s): Symposium Speaker(s):

S05--NRS: Caroline McPherson Symposium: Now That I Am Older

Oct 31, 2019 2:00pm ‐ Oct 31, 2019 3:45pm

Advances in the treatment CF have increased life expectancy, with the median age now up to 46 (CFF Patient Registry, 2017). This number is likely to increase with the advent of CFTR modulators. With these victories also come the realities & challenges associated with increased age, many of which patients with CF have not experienced. This session will review and discuss issues that may accompany aging. Topics will include fertility, contraception and pregnancy. Peer Connect will join us to offer insight regarding the mentoring tools, resources and support available for patients . Finally, participants can anticipate hearing from an adult living with CF who will tell her experience on this journey into adulthood.

Learning Objectives:

  • Explain and examine of the process of fertility.
  • Review pregnancy in relation to barriers, planning and treatment.
  • Identify more with adult patients and the struggles on their journey including utilizing CF Peer Connect.

Symposium Speaker(s): Symposium Chair(s): Symposium Speaker(s):

S06--NT-APP&D: Where Mechanism Meets Therapy Beyond CFTR

Oct 31, 2019 2:00pm ‐ Oct 31, 2019 3:45pm

This session will focus on the mechanistic basis for the development of novel, non-CFTR directed small molecule therapies that may improve mucociliary clearance in CF.

Learning Objectives:

  • Discuss the mechanistic basis of non-CFTR-mediated anion (Chloride and Bicarbonate) transport in the CF airway, and how small molecules directed at alternate anion transporters may bypass mutant CFTR in the CF airway.
  • Discuss the mechanistic basis of hypothesized defects in sodium transport in the CF airway, and how targeting this defect with small molecules may improve the pathophysiology of the CF airway.
  • Discuss the mechanistic basis of distorted mucociliary clearance in the CF airway in the context of how utilization of osmotic and mucolytic interventions may lead to improvements.

Note: Speaker Ronald Rubenstein's presentation was not recorded at his request.

Symposium Chair(s): Symposium Speaker(s):

S07--PT&RT-CLIN: Pulmonary Function Testing: Utilization & Testing Methodology in Clinical Practice, Clinical Research, & Global Application

Oct 31, 2019 2:00pm ‐ Oct 31, 2019 3:45pm

Pulmonary function studies have been a mainstay in the assessment and treatment in CF. This session will explore the clinical applications of pulmonary function studies throughout the lifespan of the person with CF. The session will review research to practice topics that have implications in the care of CF. Additionally, it will investigate modes of testing on the horizon that may have impact in CF pulmonary function testing.

Learning Objectives:

  • Describe various pulmonary function modalities that are being used globally in both a clinical and research capacity in CF.
  • Describe how these tests are being performed, evidence for clinical utility and identify research applications and their role in CF.
  • Describe how results of these tests are interpreted and how interpretation may be used to inform research and/or guide the care of patients with CF.

Symposium Chair(s):
  • Michael E. Cantine, BSAST, R.R.T., CPFT, Lead Respiratory Therapist, Atlantic Health, Morristown Medical Ctr.
  • Renee Jensen, R.R.T., Respiratory Therapist, The Hospital for Sick Children
Symposium Speaker(s):