Blood Biomarkers Determine Response in Cystic Fibrosis Patients

NJH ID: #08-22

Background
Cystic fibrosis (CF) is a genetic disorder that affects one out of every three thousand newborns. It is the most common lethal inherited disease in the western world. The life expectancy for this disease has increased to nearly 40 years and respiratory failure is the main cause of death in this patient population.

The major causal factors 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 pulmonary inflammation.  A clinical study performed in cystic fibrosis patients at National Jewish showed that circulating leukocytes transcripts of this gene panel is particularly useful in quantifying therapeutic response and predicting disease resolution. This blood-based diagnostic test is highly reproducible across patient groups and can differentiate between acutely ill and subsequent treated patients. This test is able to quantified reduction in pulmonary infection and inflammation following treatment with greater accuracy than standard measurements such as FEV(1) and CRP.

 

Potential Applications
Diagnostic test for measuring drug response in any lung related disease were inflammation, exacerbation and pulmonary infection are present

 

State of Development
The test has been validated in CF

 

Publications

  • 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

 

Patent Status
Issued U.S. Patents #8,101,361 and #8,465,923. Other US patents pending

 

Inventors
Milene Saavedra, MD and Jerry Nick, MD

 

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