Cystic Fibrosis (CF) Research
Since 1989, when the cystic fibrosis gene mutation was identified on chromosome 7, research to find a cure has increased dramatically. Within one year of the cystic fibrosis gene mutation discovery, researchers were able to correct in the laboratory the basic cystic fibrosisTR defect.
The following summaries represent some of the research currently underway to develop a cure for people with cystic fibrosis.
Activating Alternative Chloride Channels
Since it is known that with cystic fibrosis the chloride channels do not function properly, researchers have been exploring ways to activate alternative chloride channels.
In 1991, researchers administered the drug Uridine Triphosphate (UTP) by nasal spray to people with cystic fibrosis and observed that the exchange of salts (sodium and chloride) and water in their nasal cells had improved.
Further research is underway to examine the effectiveness of using aerosolized UTP in the lower respiratory tracts of people with cystic fibrosis.
Researchers believe that certain viruses may be able to enter the cells that line the respiratory system and correct the genetic material (DNA) that causes the chloride channel to malfunction.
Laboratory studies done in the early 1990s showed this method to be successful in correcting the chloride channel of the cells that line the nasal passages of people with cystic fibrosis.
Researchers are now examining different kinds of viruses to determine the most effective use of gene therapy to correct the defective chloride channel in the lungs of people with cystic fibrosis. Researchers are also looking at ways to use microscopic fat globules to deliver the normal gene to the cells.
Natural Airway Defenses Against Infection
Researchers recently identified defensin, a bacteria-fighting substance in the fluid that lines the airways of people with and without cystic fibrosis. Laboratory studies showed that defensins exposed to high concentrations of salt lost their abilities to destroy bacteria.
Since people with cystic fibrosis have higher levels of salt in their airway fluids, their defensins become inactivated, resulting in an increased risk for lung infections. These findings offer researchers direction for future studies of ways to correct or improve the lung immunity of people with cystic fibrosis.
Novel Nutritional Therapy
Fats, or fatty acids, in the body's cell walls are important for the cell to function normally. People with cystic fibrosis have been found to be deficient in these fatty acids.
Docosahexaenoic acid (DHA) is a particular fatty acid that recently has been found to correct the basic defect in the organs affected by cystic fibrosis. In experiments using mice with symptoms of cystic fibrosis, the mice improved when given DHA.
Clinical trials with people who have cystic fibrosis are underway to evaluate the safety and effectiveness of using DHA to treat cystic fibrosis.
Repair Protein Therapy
Laboratory studies of 8-cyclopentyl-1, 3-dipropylxanthine (CPX) have found that this new drug "repairs" the chloride channel defects found in the delta F508 gene mutation.
Clinical trials with people who have cystic fibrosis are currently underway to examine the safety and effectiveness of this drug, which can be taken orally.