Woman with arms crossed wearing a lab coat and stethoscope. Icons representing Lungs and medical research.

Current and Future Research Needs in the Era of Highly Effective Modulator Therapies for Cystic Fibrosis

June 2 - 3, 2022
Virtual Zoom Workshop


Cystic fibrosis (CF) is a rare inherited disease caused by mutations in a gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Mutations in CFTR cause significant morbidity and mortality for patients with CF, primarily affecting the lungs and digestive systems. With no cure for CF, advances in treatment using highly effective modulator therapies (HEMT) are helping patients live longer lives with improvements in clinical outcomes.

On Thursday, June 2, and Friday, June 3, 2022, the National Heart, Lung, and Blood Institute (NHLBI), National Institute of Allergy and Infectious Diseases (NIAID) and National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) of the National Institutes of Health (NIH) in planning collaboration with the Cystic Fibrosis Foundation will host a virtual workshop with the objective to bring together experts in Cystic Fibrosis (CF) clinical-translational research to address critical questions and inform the field of the future research needs in the era of highly effective modulator therapies (HEMT) for the treatment of CF. The workshop will highlight challenges and opportunities for research in the context of rapidly shifting treatment options. The workshop is free, open to the public, and will be recorded.

REGISTER by May 30, 2022, to participate.

Individuals who require reasonable accommodation to participate in this event should contact Myesha Macdonald at and/or the Federal Relay (1-800-877-8339).

Tentative Agenda

Day 1: June 2, 2022
9:00 a.m. - 5:00 p.m. EDT

Welcome Address and Meeting Logistics
Welcome Address on behalf of NHLBI Dr. Jim Kiley, Ph.D., Director, Division of Lung Diseases
Introduction to Workshop and Meeting Logistics Dr. Marrah Lachowicz-Scroggins, Ph.D., Program Director, Division of Lung Diseases

Cystic Fibrosis Research Landscape in the Era of High Effective Modulator Therapies
Dr. J.P. Clancy, MD, Vice President of Clinical Research, Cystic Fibrosis Foundation (on behalf of CFF)
This session will set the stage for the meeting with an overview of the current cystic fibrosis (CF) research landscape and the development pipeline of CFTR-based therapies for all including novel modulators, genetic-based therapies, and readthrough agents. This session will also address how the outcomes and future research needs of those on highly effective modulator therapy (HEMT) will critically inform the research needed when disease modifying therapies are available and accessible to all individuals with CF.

Session 1: Animal Model Systems to Study High Effective Modulator Therapies
This session will introduce current animal models to study HEMT, discussing their capabilities and limitations. The goal is to allow the audience to understand current CF animal models that can enhance understanding of the CF human condition and inform clinical studies.

Session 2: Late-stage Cystic Fibrosis Lung Disease Pathophysiology and Processes
The session will discuss what lung pathology of CF is reversible in people with chronic and established lung disease, including structural changes and chronic infection. Cross-talk between endocrine systems, nutrition and metabolism will be addressed and how changes in these systems link to lung disease progression. Goal is to define clinical challenges for CF patients on HEMT with chronic lung disease.

Community Member Perspective

Breakout Sessions and Recap

Day 2: June 3, 2022
9:00 a.m. - 5:00 p.m. EDT

Welcome (Recap Day 1) and Patient Representative

Session 3: Concerns and Questions About People with Cystic Fibrosis and Early Lung Disease
This session will define clinical challenges for CF patients on HEMT with chronic lung. Topics include: additive therapies for children on HEMT when to start or stop? Disease monitoring in children starting HEMT includes bronchoscopy, imaging, LCI, pathogen monitoring, CFRD screening, and non-invasive biomarkers. Effects of HEMT on host-pathogen interactions and extrapulmonary organ systems—multiorgan interactions on the evolution of CF lung disease. Potential use of HEMT in utero and early models to predict disease course.

Session 4: Considerations for Modulator use in Special Populations 
This session will address modulator use and needs during pregnancy, post-transplant, in patients with ultra-rare mutations, and as patients age with risk of age-related co-morbidities (cancer, renal failure, etc.). This session will also address racial disparities in CF, including access to medication, representation in clinical trials, and known healthcare disparities pre-modulators as a primer for a path forward to high-quality clinical care for all patients with CF.

Session 5: Advancing Clinical Care and Outcomes for Patients with CF in the Post Modulator Era 
This session highlights the challenges of detecting outcomes and meaningful endpoints for research in the post-modulator era will include discussion of novel imaging, pathogen detection and remote data capture tools for monitoring lung disease progression in CF as well as defining and treating acute pulmonary exacerbations.

Breakout Sessions and Recap

Panel Discussion: What are the gaps and opportunities? What research programs are needed?


For programmatic questions, contact Marrah Lachowicz-Scroggins at For logistical questions, contact NHLBI Workshop Support at


REGISTER by May 30, 2022, to participate.