Posted December 20, 2010 14:41:00

Cystic fibrosis patients could benefit from a new experimental drug being trialed in the United States.

Cystic fibrosis is a genetic disease that affects a number of organs in the body – especially the lungs and pancreas – by clogging them with thick, sticky mucus.

The new medication is called denufosol and acts to help prevent the formation of the mucus.

Doctors say the drug can be given early in the progression of the condition and may delay the disease getting worse.

The medication works by normalising the surface of the airways.

In patients with cystic fibrosis, mucus builds up in the patient’s airways causing obstruction and respiratory infections.

Dr Frank Accurso, professor of paediatrics at the University of Colorado School of Medicine in Denver, says the new drug helps enhance the hydration of the airways and can aid in clearing mucus.

“The drug is different from other cystic fibrosis medications which primarily treat the symptoms rather than the underlying causes,” he said.

Researchers enrolled 352 cystic fibrosis patients and they were given either the new drug three times daily for 24 weeks or a placebo.

At the start of the study most patients had mild impairment of their lung function.

Patients’ lung functions were measured and they found those who received the active drug had better exhalation rates than the placebo group.

Both groups had a similar number of adverse events and the active drug group had fewer headaches and sinus problems.

Dr. Jay Kolls, Professor and Chair of Genetics at LSU Health Sciences Center New Orleans, and colleagues wanted to identify the factors that determine why only some develop the allergy and what factors regulate tolerance or sensitisation to the mould resulting in the development of ABPA (Allergic Bronchopulmonary Aspergillosis).The environmental mould is one of the most prevalent fungal organisms inhaled by people. In asthmatics and in patients with Cystic Fibrosis (CF), it can cause significant allergic symptoms.CF causes the body to produce thick, sticky mucus that clogs the lungs, leads to infection, and blocks the pancreas, which stops digestive enzymes from reaching the intestine where they are required in order to digest food.The researchers discovered that heightened Th2 (the hormonal messengers of T-helper cells that produce an allergic response) reactivity in the ABPA group correlated with a lower average blood level of vitamin D. “We found that adding vitamin D not only substantially reduced the production of the protein driving an allergic response, but it also increased production of the proteins that promote tolerance,” notes Kolls.”The next step in our research is to conduct a clinical trial to see if vitamin D can be used to treat or prevent this complication of asthma and Cystic Fibrosis,” Kolls said. The work will be published in September 2010 issue of the Journal of Clinical Investigation.