Cystic Fibrosis Research

In 2002, Cystic Fibrosis Foundation Therapeutics, Inc. the nonprofit drug development affiliate of the Cystic Fibrosis Foundation, selected Nationwide Children’s Cystic Fibrosis Care Center to be part of the Therapeutics Development Network (TDN).  Nationwide Children’s is one of 16 TDN centers in the United States. These centers are accredited to conduct clinical trials of possible new treatments for cystic fibrosis.

In 2009, Nationwide Children’s was selected to serve as one of 13 translational research centers for the Cystic Fibrosis Foundation’s Therapeutics Development Network. These translational research centers lead the newest Phase I clinical trials and provide scientific direction to the entire network.

Faculty Focused on Cystic Fibrosis Research

• Robert G. Castile, MD, MS
• Karen S. McCoy, MD
• Shahid I. Sheikh, MD
• Richard D. Shell, MD
• Rohan A. Thompson, MD

Current Projects

• Clinical Trials:  We are involved in several clinical trials aimed at preventing and improving treatment of cystic fibrosis.  Learn more about our cystic fibrosis clinical trials
• The Role of Macrophages in the Inflammatory Response: We are examining how macrophages (large white blood cells) are important in the exaggerated inflammatory response in cystic fibrosis. Understanding how these cells respond in cystic fibrosis may help us modify the abnormal inflammatory response to infection in cystic fibrosis.
• Early Biofilm Detection: The infectious agents common to cystic fibrosis are unusual and may progress to form special colonies called biofilms.  This change to biofilms allow bacteria to protect themselves from antibiotics. The ability to detect early biofilm formation may lead to better understanding of thsese special infections and allow us to better protect patients.
• How Viral and Bacterial Infections and Cigarette Smoke Affect Cystic Fibrosis:  We are collaborating on a study aimed at understanding how influenza infection and bacterial infections interact with cystic fibrosis. We are also examining the impact cigarette smoke has on cystic fibrosis.  We are examining the potentail use of vaccines to prevent pseudomonal infection.

Latest Cystic Fibrosis Research Findings

Inpatient Healthcare Trends Among Adult Cystic Fibrosis Patients in the U.S.

Adult cystic fibrosis (CF) patients are an expanding cohort that is taken care of in a variety of hospital settings including adult centers located within pediatric institutions. This study compared costs and discharge rates among adult CF patient hospitalizations in terms of location of hospitalization.

Access an abstract of this study: Inpatient healthcare trends among adult cystic fibrosis patients in the U.S. Pediatr Pulmonol. 2011 Sep 7. doi: 10.1002/ppul.21535. [Epub ahead of print]

Result of a Phase IIa Study of VX-809 in Adults with Cystic Fibrosis

This study details the finding of a phase II clinical study of VX-809, a potential CFTR corrector, in adults with cystic fibrosis.  

Access an abstract of this study: Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax. 2011 Aug 8. [Epub ahead of print]

Does Repeat AZLI Treatment Decrease Antibiotic Effectiveness?

Aztreonam for inhalation solution (AZLI) is an inhaled antibiotic for patients with cystic fibrosis (CF) and Pseudomonas aeruginosa airway infection.  This study investigated whether repeated treatment courses of AZLI led to decreases in P. aeruginosa susceptibility to aztreonam or other antibiotics.

Access an abstract of this study: Pseudomonas aeruginosa antibiotic susceptibility during long-term use of aztreonam for inhalation solution (AZLI).  J Antimicrob Chemother. 2011 Oct;66(10):2398-404.

Multicenter Evaluation of Infant Lung Function Tests as Cystic Fibrosis Clinical Trial Endpoints

The conducting of clinical trials in infants with cystic fibrosis (CF) has been hindered by lack of sensitive outcome measures.  This study evaluated the safety, feasibility, and ability to detect abnormalities in lung function of serial pulmonary function tests (PFTs) in infants with CF.

Access an abstract of this study: Multicenter evaluation of infant lung function tests as cystic fibrosis clinical trial endpoints. Am J Respir Crit Care Med. 2010 Dec 1;182(11):1387-97.

Using Volume-Monitored Chest CT to Obtain Images of Lung Volumes in Cystic Fibrosis Patients

This study describes a simple volume-monitored method for performing reproducible, montion-free full inspiratory and end expiratory chest CT exams in children with cystic fibrosis.

Access an abstract of this study: Volume-monitored chest CT: a simplified method for obtaining motion-free images near full inspiratory and end expiratory lung volumes. Pediatr Radiol. 2010 Oct;40(10):1663-9.

Current Cystic Fibrosis Research Grants

Possible Advantages of Conducting Bronchoalveolar Lavages During Sinus Surgeries in CF Patients, Cystic Fibrosis Foundation Therapeutics (Stephen Kirkby)

Infant Study of Inhaled Saline in Cystic Fibrosis – Isis002, University of North Carolina and CF Therapeutics Development Network (Karen McCoy)

A Phase 2, multi-center, randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ΔF508-CFTR Gene Mutation, Vertex Pharmaceuticals, Inc. (Karen McCoy)

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects with Cystic Fibrosis and the G551D Mutation, Vertex Pharmaceutical Inc. (Karen McCoy)

2 year study to assess the natural hx of progressive CF lung disease utilizing CT imaging, PFT’s and clinical scores, CF Therapeutics Development Network/Novartis (Karen McCoy)

Cystic Fibrosis Center, CFFT (Karen McCoy)

CF Therapeutic Development Network - Translational TDC Center, CF Therapeutics Development Network (Karen McCoy)

Prevention of Cystic Fibrosis Diabetes, Emory University/Food and Drug Administration
Phase 3b Randomized, Double-Blind, Placebo-Controlled Two-Part Trial to Assess the Safety and Efficacy of Continuous Aztreonam for Inhalation Solution (AZLI) in Subjects with Cystic Fibrosis (CF) and Chronic Burkholderia Species Infection, Gilead Sciences, Inc. (Karen McCoy)

An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX770 in Subjects with Cystic Fibrosis, Vertex Pharmaceuticals (Karen McCoy)

Phase 3 b, Randomized, Double-Blind, Placebo-Controlled Two-Part Trial to Assess the Safety and Efficacy of Continuous Aztreonam for Inhalation Solution (AZLI) in Subjects with Cystic Fibrosis (CF) and Chronic Burkholderia Species Infection, Gilead Sciences, Inc. (Karen McCoy)

Randomized, Placebo-controlled, Double-Blind, Multi-Center Study to Assess the Efficacy, Safety and Tolerability of Study Drug in Cystic Fibrosis Patients with Chronic Infection Due to Pseudomonas Aeruginosa, Insmed Inc. (Karen McCoy)

Prevention of Cystic Fibrosis Diabetes, Emory University (Karen McCoy)

CFTR regulation of caspase-1 and inflammatory cytokines during Burkholderia cepacia infection, The Research Institute at Nationwide Children’s Hospital (Benjamin Kopp)

A phase 3, multi-center multinational, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of MP-376 in stable cystic fibrosis patients, Mpex Pharmaceuticals (Karen McCoy)

Pulmonary vascular hemodynamic in patients with cystic fibrosis, Children’s Hospital Medical Center Cincinnati (Karen McCoy)

Changes in lung structure and function in children with cystic fibrosis, Cystic Fibrosis Foundation Therapeutics (Robert Castile)

iCARE: I change adherence and raise expectations, University of Miami (Karen McCoy)

A Prospective, 5-year Registry Study to Monitor the Susceptibility to Study Drug of PA Isolates from Patients with Cystic Fibrosis in the United States, Anonymous (Karen McCoy)

ALPINE-Open-Label Phase 2 Trial to Evaluate the Safety and Efficacy of Study Drug in Patients with Cystic Fibrosis, Anonymous (Karen McCoy)