Cystic Fibrosis
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Cystic fibrosis, also known as mucoviscidosis, is a genetically inherited, chronic disease that results in the buildup of thick, sticky mucus in the lungs, airways, and other organs. One pathological consequence of the altered CF mucus is the inhibition of mucociliary clearance, which leads to coughing, breathing problems, scarring (fibrosis), and lung infections. This disease affects about 30,000 people in the U.S. and 70,000 worldwide. With no cure, current modulator treatments can improve both the length and quality of life for cystic fibrosis patients.
People with CF are highly susceptible to infections from bacteria, viruses, and fungi because the thick, sticky mucus traps pathogens in the airways and does not have the same infection-fighting properties as normal mucus. CF mucus also provides an ideal environment for bacteria to form protective layers – known as biofilms – that make them much less susceptible to antibiotics and, therefore, more difficult to control. More than 60 percent of adults with CF battle Pseudomonas aeruginosa (PA) infections. Once PA is established in the airways, it becomes difficult to eliminate, with a cycle of recurring infections and inflammation that gradually degrades lung tissue and function. Aggressive treatment with antibiotics such as Tobramycin can delay the development of long-term infection, but over time these bacteria develop resistance to these drugs. As a result, the need for new drugs to treat mucus-containing biofilms while avoiding antibiotic resistance is significant. Vast is developing an inhalable nitric oxide-based compound to treat chronic PA infections in individuals with CF. Our lead drug candidate has demonstrated unprecedented in vitro activity against multiple strains of PA bacteria, including susceptible, non-susceptible, and multidrug-resistant isolates. |
