Challenge

In spite of intensive research during the recent decades including various gene therapy approaches, there is at present no cure for cystic fibrosis. Existing treatment options can ease symptoms, reduce complications and prolong life. Standard treatment in CF today includes the use of CFTR correctors and potentiators for subgroups of patients (although these remain inaccessible to many patients due to their high cost), preventing and combating bacterial lung infections, and promoting mucus clearance by increasing airway surface hydration with osmotic or mucolytic agents. Stagnant sputum of high viscosity, caused by the impaired chloride and bicarbonate transport resulting from a dysfunctional or downregulated CFTR protein, is a primary cause of elevated morbidity rates in CF. The stagnant sputum leads to recurrent infections and inflammation, which in turn gradually damage lung tissue and eventually leads to early death of patients (life expectancy of around 40 years). Acute infections described as “pulmonary exacerbations” (PEs), lead to hospitalizations, and are associated with sustained reduced lung function. There is therefore an unmet medical need for alternative mucolytic therapies that reduce airway mucus problems.

“Only three mucolytic agents are available for patients with CF, and ibuprofen is the only anti-inflammatory agent that is recommended for use. Mucolytic agents that are more effective and longer lasting, and anti-inflammatory agents that are safe to use and can effectively regulate chronic inflammatory responses in the lungs of CF patients, will be highly welcomed by the CF community”.

From Global Data report on Cystic fibrosis, July 2014 (5).