An ongoing study will explore tailored medical care and treatment for patients with cystic fibrosis.
A life-threatening, genetic disease, cystic fibrosis progressively damages the lungs and the digestive system. Although DNA screening detects most cases of cystic fibrosis at birth, the disease has a spectrum of severity. These early screening tests cannot currently predict disease severity. Despite recent diagnosis and treatment advances there is still no marker to determine which patients will barely show symptoms and which are at risk for persistent lung infections that can lead to respiratory failure.
“Now we’re flying blind,” says Hara Levy, MD, a physician-scientist at Ann & Robert H. Lurie Children’s Hospital of Chicago, who is trying to map out the molecular underpinnings of what patients with cystic fibrosis experience clinically.
In an ongoing study, she is using samples from cystic fibrosis patients to analyze how sets of genes are expressed over time in relation to disease severity, clinical course, treatment response, as well as a slew of other variables such as age, family history, and specific mutations in the cystic fibrosis gene. Her lab is the only one in the cystic fibrosis field to be doing this kind of cutting-edge research.
“Our ultimate goal is to be able to tailor medical care to individual patients according to their molecular profile differences,” explains Dr Levy, Director of Pulmonary Research at Stanley Manne Children’s Research Institute, within the Human Molecular Genetics Program, affiliated with Lurie Children’s, and Associate Professor of Pediatrics at Northwestern University Feinberg School of Medicine. She envisions that this tailored medical care could begin as soon as cystic fibrosis is diagnosed.