Cystic Fibrosis

Cystic fibrosis and the new drug Kalydeco

Cystic fibrosis is genetic disease affecting the respiratory and digestive system, eventually leading to early death (Marieb, 2012). This disease is inherited from your parents who both must carry this gene (WebMD staff, 2012). According to Grossman and Grossman (2005) there are numerous variances that can occur to the gene CFTR resulting in the disease manifestation, but the majority has the defect F508. CFTR regulates the flow of chloride in and out of cells (Marieb, 2012). When this process is not working properly mucous is not thinned out appropriately becoming thick, sticky and clogging the lungs and pancreas (NCBI:Publicmed Health staff, 2012). Individuals with cystic fibrosis suffer from repeated episodes of bronchitis, pneumonia, lung infections and sinusitis (NHLBI staff, 2011). Mucus in the pancreas decreases the ability of the intestines to process fats and proteins resulting in bowel issues, stomach pain, malnutrition, pancreatitis, rectal prolapse, liver disease, gallstones, and diabetes (NHLBI staff, 2011).

Cystic fibrosis affects approximately 30, 000 individuals in the United States with around 1,000 new incidents diagnosed annually (CFF staff, n.d.). According to the Cystic Fibrosis Foundation the majority of cases are discovered by age two and currently over 45% of the cystic fibrosis population is older than 18 (CFF staff, n.d.). The average individual with cystic fibrosis lives into their 30s (CFF staff, n.d.).

A new drug, Kalydeco has been developed and approved by the F.D.A. that goes beyond treating symptoms (Pollack, 2012). This drug actually assists the mutant protein in being able to do a better job of processing the flow of salts and fluids in the lungs (CFF staff, 2012). It is effective specifically on the G551D mutation which is found in about 4% of those stricken with cystic fibrosis (Pollack, 2012).

Kalydeco’s estimated price tag per patient per year is approximately $294,000...