Cystic Fibrosis
There is probably no other condition that causes more suffering in children than cystic fibrosis. Cystic Fibrosis is a genetic disorder of the the exocrine system. Each year 1000 children with the disease are born with CF and 30,000 have the disease. Advances have given those with CF a much greater chance of living a life into adulthood.
That life is far from normal. CF affects cells that produce mucus. Salt is excreted in a greater amount than normal in these patients and the mucus these patients produce is much thicker and tenacious than normal. In the lungs mucus normally protects lung tissue from foreign bodies such as dust and diseases. Because the mucus in CF patients is much thicker than normal lung tissue isn't protected. The mucus is also a breeding ground for frequent infections which becomes an unfortunate pattern among those who have this disease. The increased infections, after awhile begin to damage lung tissue, causing a decrease in lung function and after time, respiratory failure. Lung failure is the leading cause of death among cystic fibrosis patients.
Other organs are affected by CF. The pancrease, an organ behind the stomach secreates enzymes necessary to digest food. But in patients with CF the ducts that secreate digestive enzymes become blocked and the patient will have a hard time receiving nutrituon from food. This compounds problems when children and others with this condition have fight off respiratory infections.
Many improvements have been made that have helped Cystic Fibrosis patients live a longer, more productive and enjoyable life. Removing the thick, tenacious secretions from the lungs has been a primary concern with those who work with cf patients. Not too many years ago this was accomplished by a process called CPT or chest physiotherapy. The patient would lie in a position such that the bronchial tube and the lobe of the lung that it feeds would be nearly perpendicular to the earth. Another person would use their hands to clap on patients chest and thus aid them in removing secretions. Various machines and flutter devices have many times taken the place of CPT.
Medicines are availble to help CF patients clear secretions . Mucomyst can be used to clear secretions by chemically breaking the secretions into smaller pieces thus making it easier to cough up. Mucomyst has drawbacks. It is can irritate the lungs and cause bronchospasm. It is also has this terrible smell of rotting eggs. Since 1993 a new drug has been used as a replacement for mucomyst. It is dNase or pulmozyme. It seems to be better tolerated than Mucomyst, and has been shown to reduce lung infections and increase lung functions slightly.
Improvements have been made in nutritional suppliments and antibiotics. Tobi is an inhaled antibiotic which has been used in the treatment of pseudomonas infections which plague CF patients. Suppliments of pancreatic enzymes are being used to help offset digestive defficiencies. These are essentially only stopgap measures till progress is made toward finding a cure.
Gene Therapy - Nothing has given more hope to cystic fibrosis patients than the possibilities of gene therapy. Cystic Fibrosis is a disorder of the genes in the person's body. More specifically it is a number of disorders of the long arm of the # 7 chromosome. This problem causes a change in one of the proteins which help regulate sodium and chlorine atoms moving in and out of an epithelial cell. Too many sodium ions end up in the cell, and the result is that cells that produce mucus produce a mucus that is too viscus to do any good. The hope with gene therapy is that if you can change the defect in the number 7 chromosome, the change in the protein will not occur and mucus will return to a normal state.
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Last Updated 5/27/2006 12:35:06 AM
