Cystic Fibrosis
UCSF has been a major center for cystic fibrosis research for more than 25 years, receiving funding from the Cystic Fibrosis Foundation, National Institutes of Health, and other agencies. For example, the Cystic Fibrosis Research and Development program at UCSF, one of the few basic research centers sponsored by the Cystic Fibrosis Foundation, has long focused on understanding the biology of alterations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein and identifying molecular approaches to correct fundamental defects in CFTR that lead to disease.
In addition, UCSF physicians and researchers are engaged in many other areas of cystic fibrosis research. For adults with CF cared for at our Center, clinical research studies led by UCSF investigators provide opportunities for patients to participate as research study volunteers. Some areas of recent and ongoing research investigation are highlighted below.
- Clinical trials of new therapies for CF: The UCSF Adult CF Center, led by Mary Ellen Kleinhenz, MD and Yvonne Huang, MD, has been involved in pharmaceutical trials of new treatments for CF lung disease. These have included studies of inhaled ciprofloxacin and inhaled levofloxacin antibiotic regimens to manage lung disease in patients chronically colonized with the bacteria Pseudomonas aeruginosa.
- Microbiome studies in CF: Although the lungs of CF patients typically are infected by Pseudomonas aeruginosa, the entire microbial community present in the lungs is now known to be considerably more complex. Moreover, depending on the microbial milieu, organisms not traditionally thought to have pathogenic consequence may be detrimental and contribute to inflammation. Understanding components of this microbiome that contribute to lung inflammation and disease progression, as well as how the microbiome is affected by therapies, are all active areas of research interest. In addition, many CF patients suffer concurrently from upper respiratory problems like chronic sinusitis, and studies are ongoing to improve our understanding of the CF sinus microbiome.
- Airway mucus biology in CF: Abnormal mucus is a hallmark of CF lung disease as well as other airway diseases. Understanding the basis of abnormal mucus properties and hypersecretion in CF, as well as development of new approaches to reduce abnormal mucus in the airways, are active areas of investigation in the division.
- Quality and transition to adult CF: Spearheaded by a Cystic Fibrosis Foundation grant for quality improvement in CF care, we have ongoing initiatives to facilitate patient transition from pediatric to adult CF care, a challenge faced by all individuals with CF and their care providers. Through CFF support, we have established a program of communication including pre-transition meetings with pediatric patients and printed resources, to make the transition to adult CF care as seamless as possible.