Cystic fibrosis is a genetic disease, which means that it is a result of the gene expression in an individual\’s DNA. The only way to cure, or prevent, the disease would be with gene therapy at an early age. In an ideal world gene therapy would replace or repair the defective gene.
At the present time, there is neither a gene therapy nor any other type of treatment that addresses the basic cause of cystic fibrosis, although there are several approaches being investigated for treatment protocols. In the meantime, physicians are able to recommend treatments which will slow the progression of the disease and improve the individual\’s quality of life. Treatments are usually a combination of antibiotic therapy, respiratory therapy, physical therapy and digestive enzymes.
At one time cystic fibrosis was a terminal illness in childhood. Today, with better treatment protocols available, the average lifespan of individuals has increased to nearly 30 years. As the major focus of treatments is the obstruction in the respiratory system that causes frequent lung infections. Chest physical therapy, consisting of bronchial or postural drainage, is taught to families and parents at home. This method uses percussion and vibration to dislodge the mucus and help it move out of the airway.
Younger children must have this performed by their parents but older children and adults can learn to do this on their own. Mechanical aids are available to help individuals performed chest percussion independently. Physicians also recommend exercise to help loosen mucus and stimulate coughing.
Patients may also use aerosolized, or mist medications to help dilate the bronchial tubes. Other medications will help to thin the mucus and reduce the swelling. Recent advances a new medication, and inhaled enzyme treatment, is used to thin mucus by digesting the cellular material.
Digestive problems and individuals who suffer from cystic fibrosis are less serious and more easily managed than those problems which present themselves in the respiratory system. Patients are encouraged to follow a well-balanced, high calorie diet which is low in fat and high in protein. Individuals often take pancreatic enzymes which help in the digestion of their food.
Children and adults who suffer from cystic fibrosis should use supplements of vitamins A, D, E and K to ensure good nutrition. Physicians will recommend mucolytic agents,and exercise to help prevent and treat intestinal blockages.
Some individuals qualify for a lung transplantation if they suffer from severe breathing problems or life-threatening pulmonary complications. Determining whether or not an individual is a good candidate for the procedure will depend upon a number of factors including overall health, lifestyle factors and availability of donor organs.
There has been major progress in the research and development of treatment protocols for individuals who have fibrosis since 1989. It was at this time that a genetic mutation that cause the disease was identified. Scientists have been studying ways to is her copies of the normal gene into cells of the respiratory tract. The challenge has been to find a reliable delivery method. Several methods have been tempted including using modified viruses, fat capsules and synthetic vectors.
RESOURCES
National Heart Lung and Blood Institute: How is Cystic Fibrosis Treated?
Cystic Fibrosis Foundation: Treatments
MayoClinic: Cystic Fibrosis Treatments
University of New Mexico: How is Cystic Fibrosis Treated?
Keck Hospital of USC: Cystic Fibrosis Treatment Options
Cystic Fibrosis Foundation: Therapies
New England Journal of Medicine: A CFTR Potentiator in Patients with Cystic Fibrosis