The clinical research program at Washington University School of Medicine has grown substantially since our inclusion in the Cystic Fibrosis Foundation’s Therapeutics Development Network (TDN), and we have and are participating in network-supported clinical trials testing novel agents to modulate, correct or potentiate specific cystic fibrosis transmembrane conductance regulator (CFTR) defects; these agents may profoundly affect the clinical course of cystic fibrosis. The center is one of six primary sites of the National Institutes of Health (NIH) supported Genetic Disorders of Mucociliary Clearance Consortium, a clinical research network to study rare diseases of the airways, focusing on variant forms of cystic fibrosis, as well as related diseases such as primary ciliary dyskinesia. We are participating in the NIH and Cystic Fibrosis Foundation supported Cystic Fibrosis Liver Disease Research Consortium, a multicenter, longitudinal study utilizing diagnostic, clinical and outcome data to understand the pathogenesis of cystic fibrosis-related liver disease. The center also collaborates in investigator-initiated research on CF Related Diabetes, intestinal complications in CF and hearing loss in CF due to aminoglycoside therapy. The center continues its many quality improvement initiatives, including an Agency for Healthcare Research and Quality (AHRQ) supported program to develop a computer-based system for the detection of adverse drug events in cystic fibrosis patients during care transitions.

The Cystic Fibrosis Center has maintained collaborative relationships with Washington University investigators in clinical and basic science departments, which has allowed us to consider questions fundamental to our understanding of the disease. Several NIH-funded investigators are collaborating on projects that deal with host-pathogen interactions, pulmonary inflammation and airway injury, as well as cell biology of the airway epithelia. 

Basic science and translational research

The Division of Pediatric Allergy and Pulmonary Medicine has a core group of physician-scientists and clinical investigators, many of whom have extramural funding, including NIH and Cystic Fibrosis Foundation-supported grants that focus on pathophysiology relevant to cystic fibrosis lung disease. The center collaborates with investigators in clinical and basic science departments, including as a key collaborator in the St. Louis Children’s Hospital Children’s Discovery Institute Center of Excellence in Pediatric Pulmonary Diseases & Asthma.

Education & training

Washington University provides an Accreditation Council for Graduate Medical Education (ACGME)-accredited, three-year, training program in pediatric pulmonology. Fellows in this training program currently rotate through our clinical services and directly care for cystic fibrosis patients both in clinic and when they are hospitalized.

Several clinical conferences are regularly held for the pediatric pulmonology fellows, including a weekly lecture series that comprehensively reviews pulmonary physiology, pathophysiology, and clinical care; cystic fibrosis is extensively covered as part of the curriculum. Cystic fibrosis-related topics are also presented at the divisional research conference and Journal Club. Several research conferences are held weekly throughout the medical center, covering topics in pulmonary infection, inflammation, host defense and lung injury that relate to cystic fibrosis. Several NIH Clinical Training (T32) Grants, administered through the Departments of Pediatrics and Internal Medicine, are available to support our fellows during their research training.

The Department of Pediatrics at the Washington University School of Medicine consistently attracts talented house officers. Residents are intimately involved in both the inpatient and ambulatory care of our cystic fibrosis patients. As residents rotate through the Division of Pediatric Allergy and Pulmonary Medicine, which many do during their second- or third-year of training, they have the opportunity to participate in the care of cystic fibrosis patients in all stages of disease, particularly during rotations on the 7100 Respiratory Unit where a mixture of formal and informal teaching occurs regularly. They receive more formal education in the scheduled departmental clinical conferences, and members of the Division of Pediatric Allergy and Pulmonary Medicine are regularly recognized with teaching awards from the medical students and residents. Cystic fibrosis is often a topic for discussion in Department of Pediatrics Grand Rounds and Case Management Conference.

Washington University School of Medicine is distinguished as one of the most competitive in the nation, and has the largest Medical Scientist Training Program (MSTP) program in the country. Medical students are exposed to cystic fibrosis in pulmonary pathophysiology courses, and several pre-clinical medical students and undergraduates have been attracted to both clinical and basic cystic fibrosis research, in some cases supported by Cystic Fibrosis Foundation Student Traineeship Awards.

During their third-year clinical training, medical students are involved in the care of hospitalized cystic fibrosis patients on the 7100 in-patient unit, and the division faculty review cases with students rotating through pediatrics.  These students also participate in the departmental lectures that deal with cystic fibrosis.  As fourth-year medical students, trainees from Washington University and other medical schools can elect to rotate through the cystic fibrosis clinics.

Cystic fibrosis has been the topic of the Markey Scholar Program, a progressive educational program that exposes PhD students and post-doctoral fellows to important clinical problems.