New Directions in CF Therapies

Volume 8, Issue 12

New Directions in CF Therapies

In this issue:

A truly effective cystic fibrosis (CF) disease modifying therapy needs to decrease the rate of lung function decline and improve both quality of life as well as life expectancy. The introduction and exceptional success of the CFTR protein potentiator ivacaftor began an exciting new era of progress in treating not just the symptoms of CF, but also the actual underlying mechanisms of the disease itself.

In this issue, Dr. Jennifer Taylor-Cousar from National Jewish Health and Dr. Shijing Jia from the University of Michigan analyze the current literature describing the newest frontiers of CF therapy.

Learning objectives:

Describe the potential clinical benefits and limitations of CFTR modulator therapy
Discuss the development of therapies targeted at dysregulated inflammation in CF
Explain the rationale for potential CFTR mRNA modifying therapies

Authors:

Jennifer Taylor-Cousar, MD, MSCS

Medical Director, Clinical Research Services
Co-Director and CF TDC Director, Adult CF Program
Interim Associate Vice President of Diversity, Equity and Inclusion
Professor, Departments of Internal Medicine and Pediatrics
Divisions of Pulmonary, Critical Care and Sleep Medicine and Pediatric Pulmonary Medicine
National Jewish Health
Denver, CO

Shijing Jia, MD

Assistant Professor
Department of Pulmonology
Michigan Medicine Pulmonary Clinic – Taubman Center
Ann Arbor, MI

Guest Faculty Disclosures

Dr. Taylor-Cousar has indicated that, as faculty at an institution that is part of the CF TDN, she has been a site PI on studies for Bayer, Celtaxys, Proteostasis, and Vertex Pharmaceuticals Incorporated. She has also received consulting fees from Gilead Sciences, Inc and Santhera. She has done clinical trial consulting and/or advisory boards for Gilead Sciences, Inc., Proteostasis, Protalix, Santhera, and Vertex Pharmaceuticals Incorporated. She has received grant funding from Gilead Sciences, Inc.

Dr. Jia has indicated that she has no financial interests or relationships with a commercial entity.

Unlabeled/Unapproved Uses

The authors have disclosed that they will be discussing the use of ivacaftor for rare mutations of CF. These methods have not been officially approved for this purpose.

Program Directors:

Peter J. Mogayzel, Jr., MD, PhD

Professor of Pediatrics
Director, Cystic Fibrosis Center
Johns Hopkins University School of Medicine
Baltimore, MD

Noah Lechtzin, MD, MHA

Director, Adult Cystic Fibrosis Program
Associate Professor of Medicine
Johns Hopkins University School of Medicine
Baltimore, MD

Suzanne Sullivan, RN, BSN

Senior Clinical Nurse
The Johns Hopkins University
Baltimore, MD

Guest Faculty Disclosures:

Dr. Jia has indicated that she has no financial interests or relationships with a commercial entity.

Unlabeled/Unapproved Uses:

The authors have disclosed that they will be discussing the use of ivacaftor for rare mutations of CF. These methods have not been officially approved for this purpose.

Length of activity:

1.0 hour Physicians
1.0 contact hour Nurses

Launch date: August 30, 2019
Expiration date: August 29, 2021

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