Indianapolis is known for its speedway, but this weekend, scientists, physicians and advocates for advancements in cystic fibrosis (CF) therapies are gathering there for the 31st Annual North American Cystic Fibrosis Conference. Throughout the weekend, experts in the field will present findings of their latest research into the causes and (potential) cures for this chronic, life-threatening disease.
CF is a progressive genetic disease that causes persistent lung infections and chronic inflammation of pulmonary tissue leading to permanent lung damage and ultimately resulting in respiratory failure. CF is characterized by the accumulation of thick, sticky mucus in the lungs and clogged airways which impede breathing. Bacteria are not easily cleared and create protective biofilms that are difficult for antibiotics to penetrate and often lead to the emergence of multi-drug resistant bacteria.
More than 30,000 people in the United States, and a similar number in Europe, live with cystic fibrosis, according to the Cystic Fibrosis Foundation.
One of the presentations will be about a new class of inhaled therapeutics for the treatment of pulmonary disease. The drug, currently known as SNSP113, has been the focus of two studies led by the laboratory of Steven M. Rowe M.D. at the University of Alabama at Birmingham.
Results from one study presented suggest that SNSP113 has the potential to enhance pulmonary clearance by improving the viscosity and transport of mucins through structure alteration.
In addition, pretreatments of SNSP113 prevented Pseudomonas aeruginosa infection in 70% of wild-type rats, whereas all animals treated with control were infected. Enhanced pulmonary clearance is an important aspect of removing bacteria to prevent colonization and infection in the lung. SNSP113 is being developed by Synspira, a privately held company.
“People living with cystic fibrosis are challenged by abnormal mucus that precipitates chronic infection and dysregulated inflammation,” said Rowe. “The effects on both mucus and Pseudomonas bacterial infection demonstrated by SNSP113 are very promising for cystic fibrosis patients, no matter their underlying CFTR mutation.”
