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W02--CFTR: *CFTR 2019
09:45am - 11:05am Central - October 31, 2019 | Room: 104
Estelle Cormet-Boyaka, Ph.D., Workshop Chair; Amit Gaggar, M.D., Ph.D., Workshop Chair; Nael A. McCarty, Ph.D., Workshop Speaker; Carleen Mae Sabusap, Ph.D., Workshop Speaker; Jae Seok Yoon, Ph.D., Workshop Speaker; Margarida Quaresma, M.Sc., Workshop Speaker; Kyu Shik Mun, Ph.D., Workshop Speaker
Track: Workshop

This workshop will focus on CFTR biology, from molecular structure/function to impact in disease. The session will introduce the earliest known CFTR by describing its structure and function. It will then be presented how proteostasis network profiling can help predict response to correctors. Additionally, there will be description of how the rate of translation of NBD1 can cause structural changes and affect function of mature CFTR protein. We will also learn about how CF airway epithelial cells exhibit altered localization of epithelial markers leading to a less differentiated and more proliferative state. Finally, a human pancreas-on-a-chip model will be presented as a way to study CF-related diabetes (CFRD).

Learning Objectives:

  • Define how sequence evolution and protein translation can affect CFTR function.
  • Evaluate new proteostasis profiling to predict response of CFTR mutants.
  • Assess the effect of CFTR on new considerations related to epithelial cell differentiation and models of CF-related diabetes.

Note: Speaker Jae Seok Yoon's presentation was not recorded at his request.

W03--CLIN: *Pulmonary Advances
09:45am - 11:05am Central - October 31, 2019 | Room: 207
Shijing Jia, M.D., Workshop Chair; Jamshed Kanga, M.D., Workshop Chair; Christopher Goss, M.D., M.Sc., Workshop Speaker; Carli Lehr, M.D., M.S., Workshop Speaker; Anne Stephenson, M.D., Ph.D., Workshop Speaker; Kathleen J. Ramos, M.D., M.Sc., Workshop Speaker; Jenna Gibilaro, M.D., Workshop Speaker
Track: Workshop

This session highlights the current work in CF pulmonary disease. Discussions will include an exploration of oral versus intravenous antibiotic therapy for pulmonary exacerbations, barriers and outcomes of lung transplantation for advanced lung disease, and a look at a single center's experience in extracorporeal membrane oxygenation.

Learning Objectives:

  • Evaluate the efficacy of home monitoring of FEV1 in identifying pulmonary exacerbations in cystic fibrosis.
  • Examine barriers to and outcomes of lung transplant for advanced lung disease.
  • Analyze outcomes of ECMO prior to lung transplant.

S02--CLIN-APP&D-INF/MIC: *The Impact of Highly Effective CFTR Modulator Therapy on the Natural History of CF
02:00pm - 03:45pm Central - October 31, 2019 | Room: 104
Gregory Sawicki, M.D., M.P.H., Symposium Chair; David Nichols, M.D.; Tara Barto, M.D., Symposium Chair; Daniel H Leung, M.D., Symposium Speaker; Pradeep Singh, M.D., Symposium Speaker
Track: Symposium

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.

S03--ET-NT-GMS: *Fixing What Modulators Won't
02:00pm - 03:45pm Central - October 31, 2019 | Room: 207
Susan Birket, Ph.D., Pharm.D., Symposium Chair; Joseph Zabner, M.D., Symposium Chair; Lindsey Plasschaert, Ph.D., Symposium Speaker; Patrick Sinn, Ph.D., Symposium Speaker; Daniel Siegwart, Ph.D., Symposium Speaker; John Paul Clancy, M.D., Symposium Speaker
Track: Symposium

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.

P1--*Emerging Technologies for CFTR Restoration in All People With CF
04:30pm - 06:00pm Central - October 31, 2019 | Room: KFD Ballroom
Marie E. Egan, M.D., Plenary Leader
Track: Plenary

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.

P2--*Entering the Era of Highly Effective Modulator Therapy
08:30am - 10:00am Central - November 1, 2019 | Room: KFD Ballroom
Track: Plenary

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.

S09--APP&D-NT: *How Can We Minimize Lung Damage in CF From Infancy?
10:30am - 12:15pm Central - November 1, 2019 | Room: 104
Emanuela Bruscia, Ph.D., Symposium Chair; Jennifer S Guimbellot, M.D., Ph.D., Symposium Chair; Jeffrey Wine, Ph.D., Symposium Speaker; Marcus Mall, M.D., Symposium Speaker; James F. Chmiel, M.D., M.P.H., Symposium Speaker; Felix Ratjen, M.D., Ph.D., Symposium Speaker
Track: Symposium

Infants and young children with cystic fibrosis have abnormal lungs at birth, with congenital defects of the airways, mucus glands, and airway surface liquid already apparent. Infection and inflammation are independent, yet interrelated causes of early childhood pulmonary disease, even in the absence of symptoms. Mucolytics, anti-infectives, and anti-inflammatory therapies are all challenging to implement in treatment of infants and young children for a variety of reasons. In this session, attendees will learn about the latest updates in the use of anti-infectives, mucolytics, and anti-inflammatory strategies.

Learning Objectives:

  • Discuss the latest concepts and progress in treatment strategies for infection, inflammation, and mucus stasis.
  • Describe the challenges to therapeutic development and implementation of early treatment strategies in infants and young children.
  • Define the challenges with overcoming early inflammation as new treatments (including anti-infectives, mucolytics, and modulators) are implemented in the youngest patients.

S12--CLIN-NRS-SW/PSYCH: *Sustaining Daily Care in the Era of Precision Medicine
10:30am - 12:15pm Central - November 1, 2019 | Room: 207
Emily Muther, Ph.D., Symposium Chair; Michelle Prickett, M.D., Symposium Chair; Kristin A. Riekert, Ph.D., Symposium Speaker; Martin Wildman, MRCP, M.Sc., Ph.D., Symposium Speaker; Lisa Opipari-Arrigan, Ph.D., Symposium Speaker; Kristin Dunn, Symposium Speaker; Carla A. Frederick, M.D., Symposium Speaker
Track: Symposium

Performing daily treatments is complex, time consuming and poses a significant daily challenge for many persons with CF. This session will explore methods allowing patients and health care teams to better achieve goals for care through assessment, collaboration, and technological advancement. These solutions will be all the more important as we encounter more specific therapeutic options in an expanding healthcare system.

Learning Objectives:

  • Illustrate a standardized way to assess the challenges and barriers to performing current CF related treatments and examine international methods to improve CF health outcomes through ease of care delivery.
  • Explain the role of technology and care team-patient/family partnerships in improving nutritional health for children living with CF.
  • Examine the opportunities for collaborations among persons with CF and their providers to design new strategies for sustained care.

W15--NRS-CLIN: *Pulmonary Exacerbations: Managing Slow Progression & Acute Drops in Lung Function Decline
02:15pm - 03:35pm Central - November 1, 2019 | Room: 104
Stacy Bichl, A.P.N., CPNP, AE-C, Workshop Chair; Cori Daines, M.D., Workshop Chair; Edith T. Zemanick, M.D., Workshop Speaker; Sharon McNamara, R.N., M.N., CCRC, Workshop Speaker; Jordana Hoppe, M.D., Workshop Speaker; Kristina Montemayor, M.D., Workshop Speaker; Lauren Castner, M.D., Workshop Speaker
Track: Workshop

This workshop examines current challenges in the management of pulmonary exacerbations. Consideration for identification of exacerbations using the FEV-1 indicated exacerbation signal (FIES), treatment options related to treatment setting, gender, targeting anerobes, and the influences on recovery of baseline lung function will be presented as well as the role of patients and families in decision making related to exacerbation treatment.

Learning Objectives:

  • Discuss priorities for research and quality improvement in the treatment of pulmonary exacerbations.
  • Define the concept of FEV1 indicated Exacerbation signal (FIES) and it's role in the identification and treatment of Pulmonary exacerbations.
  • Identify two factors influencing recovery of lung function to baseline in the treatment of pulmonary exacerbations

W17--NT-ET: *New & Emerging Therapies to Correct the Basic Defect
02:15pm - 03:50pm Central - November 1, 2019 | Room: 207
William T. Harris, M.D., Symposium Chair; Jennifer L Taylor-Cousar, M.D., M.Sc., Symposium Chair; Eyleen Poel, Ph.D., M.Sc., Workshop Speaker; Alessandra Murabito, M.S., Workshop Speaker; Matthew Goddeeris, Ph.D., Workshop Speaker; Patrick Flume, M.D., Workshop Speaker; Raksha Jain, M.D., M.Sc., Workshop Speaker; Jonathan Zuckerman, MD, Workshop Speaker
Track: Workshop

During this session, we will explore data supporting the development of therapies to address the basic defect in CF including gene editing, mRNA read-through, and CFTR protein modulation. The session will cover CFTR therapeutic development along the entire pathway from pre-clinical technical advancements and target identification to results of Phase I, II and III clinical trials.

Learning Objectives:

  • Identify appropriate molecular targets for CFTR-directed therapeutics.
  • Review safety of potential new CFTR-directed therapies.
  • Critically evaluate efficacy data from CFTR modulator clinical trials.

Note: Speaker Matthew Goddeeris' presentation was not recorded at his request.