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:: Treatments for Cystic Fibrosis

  • Date : 2009/01/26 (Rev: 2010/06/26)
  • Kent Pinkerton
  • Synopsis : Presently only symptomatic management is possible for Cystic fibrosis but there are very promising gene therapy trials under way.

Main Document

Cystic fibrosis, which affects 30,000 American children and adults, is a multi-system disease caused by a defective gene. Presently only symptomatic management is possible, but there are very promising gene-therapy trials under way.

In the respiratory system the thin mucus lining becomes thick and sticky. In cystic fibrosis management, the primary treatment of the system is to thin or clear this mucus. Bronco dilators like albuterol are used to clear the clogged airways. Mucus thinning drugs delivered by aerosol, like pulmozyme, are helpful.

The most effective way of clearing this mucus is by mechanically dislodging it. Clapping on the chest and back, with the head tilted on the edge of a table, is quite effective. There is an electrical clapper that does the job safely.

There is an electrical inflatable vest that vibrates and dislodges the mucus.

Infection is an ever-present risk with cystic fibrosis patients. Regular shots for pneumonia and influenza are very important. Bacterial infection is fought with newer antibiotics like TOBY, which delivers the medicine directly into airways with aerosols.

Because of the blocked ducts of the pancreas and liver, the enzymes and bile do not reach the intestine. Though the patient eats normally or even in excess, the fats and proteins are not digested. So the cystic fibrosis patient needs to have the enzymes supplemented with oral pancreatic enzymes. There is also the need to take vitamins, especially the fat-soluble ones. If lung function is completely damaged, the only alternative would be lung transplantation. This would involve many factors, like the availability of a donor and the patient's present health, prior to undergoing major surgery.

The Cystic Fibrosis Foundation has 115 centers all over America, and provides guidelines and advice. They support many studies to find gene therapy to cure cystic fibrosis. The target is to add normal genes to the cells of the airways to prevent further damage. Biotech laboratories produce healthy genes, which need to be delivered to the appropriate cells. These vectors, or delivery systems, are the key areas of research to combat cystic fibrosis. One method that is actively pursued is to modify the common cold virus to carry the healthy genes into the correct cells. On another track, the DNA molecules are compacted to their minimum size and delivered directly to the relevant cells without the need for a carrier such as a virus. This technology is called PLAS min.

The primary aim of medical and scientific research on cystic fibrosis is to understand, treat and cure cystic fibrosis. The research also focuses on serious symptoms and complications of the disease.

Cystic fibrosis research on gene therapy and protein therapy has made new developments. Gene therapy using liposomes is the technique of adding a healthy copy of the gene. Protein therapy is used to repair the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Drugs for replacing the function of CFTR protein have been developed. Research groups are engaged in developing new drugs to move this protein to the membrane.

Cystic fibrosis research has spread to the area of improving symptom control which includes controlling lung infections, reducing inflammation and improving nutrition.

Another fertile area of interest is regarding the cause of cystic fibrosis. The research on genetic disorders explains the factors that regulate channel activity and their effect on the plasma membrane. This research proves that the inherited disorders of cystic fibrosis include chronic pulmonary disease and hypertension. Research done on the regulation of gene expression identifies the factors that control gene expression. The study is conducted on the effect of alterations in these factors. Researchers have developed optical imaging aids for cystic fibrosis study. Optical sensing tools are used to investigate adenosine triposphate release and its role in cystic fibrosis.

Many researches are conducted to improve the treatment of cystic fibrosis. New medicines have been invented to remove the secretions in the lungs. The removal of secretions can be measured by inhaling a small amount of radioactive gas. New methods are developed to assess the deposition and clearance of the radioactive gas. Cystic fibrosis research has proved that the effect of physiotherapy is not uniform in all patients. Physiotherapy made a positive effect in majority of patients. But some patients experience deterioration in ventilation. Researchers on this disease investigate the disordered breathing and the effect of exercise on the patients. Arm exercises result in over-expansion of lungs and this may cause breathlessness in severe cystic fibrosis patients.

As this gene therapy experiments are at an advanced stage, cystic fibrosis sufferers can hope for a permanent cure in the near future.



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