Background Cystic fibrosis (CF) is a genetic disorder that affects approximately one out of every three thousand newborns in populations of European descent. It is the most common lethal inherited disease in the western world. The median life expectancy for this disease has increased to 46 years, for patients born between 2015-2019 (Cystic Fibrosis Foundation 2020 Patient Registry). Respiratory failure continues to be the main cause of death in this patient population.
The major complications in CF are airway obstruction with mucus, chronic endobronchial infection with pathogens such as Pseudomonas aeruginosa, and severe airway inflammation. Acute pulmonary exacerbations are also common in CF and contribute to loss of lung function.
There is a critical need for new effective anti-microbial and anti-inflammatory therapies to mitigate the progression of this disease. However, assessment of treatment outcomes is currently limited to standard practices based on Forced Expiratory Volume (FEV1) and C-Reactive Protein (CRP) measurements. This approach lacks sensitivity and therefore is not suitable to reliably determine the efficacy of drug treatments in CF patients or other diseases associated with airway inflammation.
Technology Physician scientists at National Jewish have identified a novel panel of leukocyte gene biomarkers associated with CF pulmonary inflammation. A clinical study performed in CF patients at National Jewish showed that circulating leukocyte expression of this gene panel is particularly useful in quantifying therapeutic response and predicting disease resolution in response to treatment of acute pulmonary exacerbations. This blood-based diagnostic test is highly reproducible across patient groups and can differentiate between acutely ill and subsequently treated patients. This test is able to quantify reduction in pulmonary infection and inflammation following treatment with greater accuracy than standard measurements such as FEV1 and CRP.
Potential Applications Diagnostic test for measuring drug response in any lung related disease where inflammation, exacerbation or pulmonary infection is present
State of Development The test has been validated in CF
Saavedra, M. T. et al. "Circulating RNA Transcripts Identify Therapeutic Response in Cystic Fibrosis Lung Disease." American Journal of Respiratory and Critical Care Medicine 178.9 (2008): 929-38. PMID: 18723435.
Nick, J. A. et al. "Blood MRNA Biomarkers for Detection of Treatment Response in Acute Pulmonary Exacerbations of Cystic Fibrosis." Thorax 68.10 (2013): 929-37. PMID: 23783371.
Licensing Status This technology is available for licensing
For Further Information, Contact: Emmanuel Hilaire, PhD Director Technology Transfer Office National Jewish Health 1400 Jackson St., Room M206b Denver, CO 80206 Voice: 303.398.1262 Fax: 303.270.2352 HilaireE@njhealth.org