Cystic Fibrosis Research

New drugs to treat cystic fibrosis symptoms are currently in clinical trials. Additional research areas include:

Gene therapy

Gene therapy hopes to replace defective genes that cause cystic fibrosis with normal genes by safely and effectively delivering the normal genes directly to the airways of people with cystic fibrosis. Potential delivery systems (or vectors) include the following viruses:

• Adeno-associated virus (the smallest known human virus that is not known to cause disease).
• HIV virus (the virus that causes AIDS.).

So far, gene therapy using these viruses has been successfully used to treat mice with cystic fibrosis. The next step is to treat humans who have the disease.

Drug therapy

Researchers are developing a new drug for genetic disorders, such as cystic fibrosis. The drug is designed for conditions caused by genetic mutations that inactivate a gene's normal function. Clinical trials are currently investigating the drug's effect on chloride secretion in the airways.

Growth hormone

Growth hormone (somatotropin) is an organic compound that stimulates the growth of bone, cartilage, and tissue. It also influences the metabolism of protein, carbohydrate, and fat. Researchers are investigating the use of growth hormone for children with cystic fibrosis who exhibit malnutrition and poor growth.

Inhaled hypertonic saline

Several studies have shown that inhaling hypertonic saline several times a day can help reduce and clear mucus, and improve lung function.

Preimplantation genetic diagnosis (PGD)

PGD is a technique that is sometimes used for couples with serious genetic disorders (such as cystic fibrosis), who desire to have children free of an inherited disease. The female's eggs are surgically removed and mixed with the male's sperm in a laboratory dish. Fertilization typically takes place within a few days. Embryos free of genetic defects are identified, and then implanted into the female's uterus.

Restoring salt transport

The defective cystic fibrosis gene alters the body's ability to move salt (sodium chloride) in and out of cells. This change contributes to the development of thick mucus. Researchers are attempting to correct the defective salt transport by modifying the protein formed by cystic fibrosis cells.

Clearing mucus from the lungs

Researchers have discovered that the rhythmic motion of the lungs during normal breathing is critical to clearing bacteria and mucus from a person's lungs. The discovery helps explain how physical and specialized breathing techniques improve lung health for people with cystic fibrosis. This new knowledge should help develop new techniques of clearing mucus from the lungs.