Pilot Study Shows Promise for New Cystic Fibrosis Treatment


2016-01-11

HAMPTON ROADS, Va.—A recently published pilot study from doctors at Eastern Virginia Medical School and Children’s Hospital of The King’s Daughters may have a significant impact on the health and treatment options for patients with cystic fibrosis.

Dr. Laura Sass, Dr. Tushar Shah, Dr. Neel Krishna and Dr. Kenji Cunnion are working together to determine how the immune system of a patient with cystic fibrosis plays a role in lung damage.

Cystic fibrosis is a genetic disease that causes sticky mucus to build up in the lungs. The presence of mucus makes it difficult to breathe and leaves patients susceptible to bacterial infections. Cystic fibrosis moves in a cycle where the lungs become obstructed by mucus, then infected by bacteria, then inflamed. This repetitive cycle grows worse and worse as it damages the lungs.

In response to bacteria, the patient’s immune system activates proteins that cause inflammation that damages lungs. Researchers tested sputum (a mixture of saliva and mucus) samples from patients at the CHKD Cystic Fibrosis Center for the presence of these proteins.

The team found that cystic fibrosis patients have almost five times the amount of a protein called C5a in their sputum than patients without cystic fibrosis. Furthermore, the amount of C5a corresponds with how sick the patient is. When the patient’s sputum contains high levels of this protein, they tend to have a lower body mass index, a sign that cystic fibrosis is getting worse.

Since C5a causes inflammation that damages lungs, researchers used a new type of medicine, called a complement modulator, to see if they could get rid of the protein. Dr. Cunnion and Dr. Krishna developed their own complement modulator at EVMS. When they put the medicine in the sputum samples, the generation of C5a was blocked. Reducing the amount of C5a in lungs could possibly help lessen lung damage.

Through a grant from the Cystic Fibrosis Foundation, the doctors are conducting a larger study which could have a major impact on how cystic fibrosis is treated including the use of complement modulators to reduce inflammation and damage to patients’ lungs, helping them feel healthier and live longer.

CHKD is the clinical home of the Eastern Virginia Medical School Department of Pediatrics and the only freestanding children’s hospital in Virginia. The hospital serves the medical and surgical needs of children throughout greater Hampton Roads, the Eastern Shore of Virginia and northeastern North Carolina.