Cystic Fibrosis

Cystic fibrosis, also known as mucoviscidosis, is the most common genetically inherited disorder among Caucasians. According to the Cystic Fibrosis Foundation, 30,000 Americans and total of 70,000 people worldwide suffer from the disease.

Cystic Fibrosis is an autosomal recessive genetic pulmonary disorder characterized by the abnormal transport of chloride and sodium ions across the pulmonary epithelium, leading to the accumulation of thick, viscous secretions or mucus in the lungs. This, in turn, leads to a chronic underlying inflammation that leads to persistent bacterial infection that can lead to excessive inflammation and fibrosis. Consequently, the cystic fibrosis airway is exposed to a vicious cycle of obstruction, infection, and inflammation, all intimately linked. Airway inflammation begins early in the disease course, with increased neutrophil infiltration present in infants as young as 4 weeks of age. The inflammatory process persists throughout the patient's life, gradually leading to bronchiectasis, irreversible damage to airways, and progressive decline in lung function. Pulmonary disease accounts for most of the morbidity and mortality in patients with cystic fibrosis although other organs, such as the pancreas and liver, are also affected by inflammatory damage in cystic fibrosis. According to the Cystic Fibrosis Foundation, the current life expectancy of a patient, with Cystic Fibrosis, is just 40 years old.

The importance of inflammation (both underlying and infection-derived) is universally accepted as contributing to the disease progression, but current anti-inflammatory treatments present a paradox: while treatment with systemic steroids and high doses of ibuprofen (a common NSAID) are effective at improving the symptoms of the disease, the side effects associated with these drugs are severe, and as a result, are infrequently used.

There is a strong consensus that a safe, effective chronic anti-inflammatory therapy will be of tremendous value to patients potentially impacting the quality of their lives as well as extending it.

For more information, please refer to the Cystic Fibrosis Foundation: www.cff.org


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