A Novel Assay to Quantify Treatment Response in Cystic Fibrosis (CF)

Inflammation is present in the Cystic fibrosis (CF) airway from the time of infancy, and
worsens with the onset of chronic infection. Therapies with proven benefit are associated
with decreased airway inflammation. Thus, sensitive and reproducible biomarkers of airway
inflammation have long been sought as a necessary component to improved clinical care and to
facilitate therapeutic trials for CF. FEV1, the standard outcome measure in CF, is recognized
as an insensitive outcome measure. the investigators have identified a panel of 10 genes
which sensitively predict resolution of pulmonary inflammation, in response to therapy of an
acute pulmonary exacerbation. With the goal of yielding a technically simple but unique CF
biomarker assay, the investigators have tested whether proteins signified by these genes show
large changes in expression following treatment of acute pulmonary exacerbations. Protein
quantifications are among the most common measurements performed in clinical laboratories
around the world. Based on preliminary findings that changes in white blood cell proteins
mirror changes seen in the genes, the investigators propose to identify top candidate
proteins, from the investigators gene panel, which change in response to exacerbation
therapy. Once identified, these proteins will be quantified directly with a new blood test
which is inexpensive and simple to perform. The investigators propose to validate this blood
test in a single site trial. If successful, this proposal will yield a biomarker assay that
will be available to validate in a multi-site trial and provide unique insights into
mechanisms that regulate white blood cell activation and recruitment in CF lung disease.