Challenge
Cystic fibrosis is a life-threatening disease that affects the lungs and digestive system and impacts about 70,000 people worldwide.
Cystic fibrosis (CF) is the most common inherited disease in the Caucasian population, and affects about 40.000 patients in Europe and 80.000 worldwide. CF is caused by mutations in the CFTR ion channel, leading to stagnant mucus in most mucus-secreting organs, with mucus retention, inflammation, chronic bacterial infections and tissue damage. The lungs are the predominant organ involved which causes significant morbidity and life expectancy of less than 40 years in Western Europe.
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).
EUROPEAN CLINICAL TRIAL NETWORK
The clinical trial will include 120 CF patients from 45 European sites in coordination with the European Clinical Trial Network.
6
Partners
45
European sites
Funding
This project has received funding from the European Union’s Horizon 2020 research and innovation programme part “Health, demographic change and well-being” (call H2020-SC1-2016-2017) under grant agreement No 755234.