Improving the Life of Cystic Fibrosis Patients

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In cystic fibrosis (CF), thick mucus clogs air passages in the lungs and interferes with breathing. The disease also affects digestion by blocking ducts in the pancreas that secrete enzymes needed to break down food. In the past, children with this inherited disorder faced the prospect of problematic lung infections and a reduced life expectancy.

UW researchers made the first direct link between the thick mucus and the defective transport of chloride ion that are the hallmarks of this disease (see Of Slug Slime, Spider Silk, and Mollusk Shells ). And now that the genetic defect has been identified, researchers at the UW and elsewhere are developing vectors to deliver the corrected CF gene back into the lungs of these patients. Until gene therapy becomes available, however, other therapies are being developed to improve the quality of life of these patients. UW researchers have made major strides in preventing the recurrent infections that damage the lungs.

Aerosol methods to deliver medication directly into the lungs of CF patients have been developed by BonnieW. Ramsey and colleagues. Ramsey, UW pediatrics professor, serves as director of the Cystic Fibrosis Center at Children's Hospital and Medical Center in Seattle.

Aerosol delivery of antibiotics offers a potentially more effective and less expensive way to treat lung infections than conventional methods of oral or intravenous delivery. In addition, as a means to improve the function of the lungs, the researchers have tested the aerosol delivery of deoxyribonuclease, an enzyme that breaks down the proteins and DNA that help make mucus thick.

"With medical treatment," says Ramsey, "we have almost doubled the life expectancy of most patients. Twenty years ago, most of these patients died before the age of 10. Now, one of two patients will reach age 30," she says. "Many patients are enjoying a good quality of life, and some are living even longer than expected. We think that in the near future, having CF will not be incompatible with a long and fruitful life."

  1. "Brighter Future Ahead for Cystic Fibrosis Patients," Julie Garner, Medicine Northwest, Summer 1987, p. 17.

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