Note:You must always seek professional medical advice about any treatment or change in treatment plans.
Some of the different medications being used in the research into treatment of Cystic Fibrosis include:
Possibly curable or rare types of Cystic Fibrosis include:
Some rare types of Cystic Fibrosis include:
Some of the more recent treatments for Cystic Fibrosis include:
Treatments to consider for Cystic Fibrosis may include:
CF research has accelerated sharply since the discovery of CFTR in 1989. In 1990, scientists successfully cloned the normal gene and added it to CF cells in the laboratory, which corrected the defective chloride transport mechanism. This technique --- gene therapy --- was then tried on a limited number of CF patients. However, this treatment may not be as successful as originally hoped. Further research will be required before gene therapy, and other experimental treatments, prove useful in combating CF. (Source: Genes and Disease by the National Center for Biotechnology)
Improvements in antibiotic therapy, clearance of lung secretions, nutritional support, and the collection of patients at centers for expert care have increased the mean survival of patients with CF from under 5 years to approximately 30 years. Since the identification of the CF gene in 1989, there has been a rapid increase in our understanding of the pathogenesis of CF and the challenge now is to translate this improved understanding into new approaches to therapy. While the discovery of the CF gene has stimulated research to find a cure for the disease, until this is achieved, the pulmonary infection and inflammation that ultimately leads to respiratory failure and premature death remain prime targets for therapy. Continued improvement in therapy directed at removing airway mucus and reducing infection and inflammation can preserve lung function until more definitive therapy is developed. In recent years NIDDK-supported researchers have made further progress in developing new treatment approaches to improve CF patients' length and quality of life. Some of the devices and drugs that have become available for therapy are described below.
The "Flutter" Device Helps Clear Airways
The "flutter," a small, hand-held device that looks like a pipe, allows patients to loosen the mucus that clogs their airways without having to endure conventional chest- and back-clapping therapy. When patients exhale through the flutter, a special valve causes rapid air pressure fluctuations in the patients' airways. The resulting vibrations dislodge the mucus from the airway walls and promote mucus movement.
In an NIDDK-sponsored study, three times more mucus was cleared with the flutter than after chest percussion and vibration by an experienced respiratory therapist or by vigorous voluntary coughing. Treatment with the flutter does not require the assistance of another person, giving the patient more independence. Further study is needed to determine whether the improved airway clearance may delay the onset of serious lung disease.
DNase Reduces Mucus Stickiness
One factor contributing to mucus stickiness is the DNA released from white blood cells that die while fighting bacterial infections. A naturally occurring enzyme called DNase can cut long DNA molecules into shorter pieces and reduce their stickiness. In 1993 the Food and Drug Administration approved the use of DNase for CF treatment. The enzyme is administered as an aerosol spray and is generally well tolerated, although patients may experience transient throat irritation or hoarseness. Treatment with DNase reduced the frequency of severe episodes of lung infection and slightly improved lung function after 24 weeks of therapy. Longer studies are needed to determine whether the small improvement in lung function seen at 24 weeks persists and whether this therapy will retard progressive loss of lung function.
New Antibiotic Therapy of Bacterial Infection
Pseudomonas bacteria are a leading cause of lung infection and death among CF patients. Until recently, Pseudomonas infections were treated by intravenous administration of antibiotics that were not available in oral form. This treatment required high antibiotic doses so that enough of the drug would reach the lung. Besides being expensive, the high doses could damage hearing and kidney function in patients.
An aerosol form of the antibiotic tobramycin significantly reduced Pseudomonas infections in CF patients. The inhaled drug directly reaches the infected lung tissue, reducing the dose required and the potential for side effects. Tobramycin by aerosol form is easier and less expensive to administer than by intravenous injection. In addition, there is now one oral anti-Pseudomonas antibiotic, Ciprofloxacin. For some patients, Ciprofloxancin effectively substitutes for a course of intravenous antibiotic, if the patient's germs are sensitive and the illness is mild.
Ibuprofen Prevents Loss of Lung Function
A clinical trial conducted at an NIDDK-supported CF Research Center recently showed that the anti-inflammatory drug ibuprofen, an ingredient in many over-the-counter painkillers, can preserve lung function in CF. To reduce the inflammation that contributes to progressive lung damage, CF patients received high, twice-daily doses of ibuprofen for four years. Patients who took the drug consistently maintained their lung function and body weight significantly better than control patients who received a placebo. The treatment was most effective in younger patients under 13 years of age. Researchers warn that ibuprofen treatment should be performed only under medical supervision because the high drug doses required must be determined individually for each patient.
Nutrition May Improve Patients' Health Status
If not corrected, malnutrition may contribute significantly to the deterioration of CF patients' health. Although the vast majority of CF patients now take supplements of pancreatic enzymes to compensate for pancreatic insufficiency, these supplements do not fully correct the malabsorption, and many children are underweight and shorter than would be expected based on parental height. Recently high doses of pancreatic enzyme supplements were found to be associated with development of colonic strictures in a few patients, causing physicians to be more cautious in dosing.
In recent years, increased attention to caloric needs, a balanced diet, and supplements of vitamins and other nutrients have contributed to the increasing longevity and well-being of CF patients. Appropriate nutritional therapy improves the patients' growth and development, strength and exercise tolerance and may improve resistance to bacterial infections. Researchers do not yet know to what extent better nutrition actually can delay progression of lung disease. Researchers are studying the causes and consequences of malnutrition in CF patients, and developing new strategies to prevent and treat malnutrition. (Source: excerpt from Cystic Fibrosis Research Directions: NIDDK)
Since CF is a genetic disease, the only way to prevent or cure it would be with gene therapy at an early age. Ideally, gene therapy could repair or replace the defective gene. Another option for treatment would be to give a person with CF the active form of the protein product that is scarce or missing.
At present, neither gene therapy nor any other kind of treatment exists for the basic causes of CF, although several drug-based approaches are being investigated. (Source: excerpt from NHLBI, Facts About Cystic Fibrosis: NHLBI)
Gene therapy for CF is not yet possible but impressive progress is being made in developing ways to treat the gene abnormality that causes CF. In the laboratory, scientists have been able to grow cells from the nasal passages of CF patients. By introducing the normal gene into these cells, researchers corrected the cells' chloride transport abnormality. The chloride defect has also been corrected in small regions in the nasal passages themselves by giving CF patients the normal gene in nose drops.
Scientists are still looking for answers to many questions about gene therapy. Some of these questions are: How should the gene be packaged? What are the best ways to get the gene-containing package into the patient's lungs? What will the long-term results of this treatment be? Can the abnormal chloride transport be corrected in other parts of the body? How long will the correction last? And, most importantly, can gene therapy cure or prevent the lung disease in CF? (Source: excerpt from NHLBI, Facts About Cystic Fibrosis: NHLBI)
The following medical news items are relevant to medical research for Cystic Fibrosis:
Some of the clinical trials for Cystic Fibrosis include:
Medical research papers related to Cystic Fibrosis include:
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