Discovery May Lead To New Cystic Fibrosis Treatments

March 11, 2010 - PRLog -- Discovery may lead to new cystic fibrosis treatments

A new discovery may give hope to the thousands of cystic fibrosis sufferers around the world.

According to a research team from the University of California, San Diego School of Medicine, they have identified a defective signalling pathway which increases the severity of cystic fibrosis, a condition which affects one in 2,400 people in the UK, and of which four per cent of the population are carriers.

In the report, published in the February 14th edition of the journal Nature Medicine, lead investigator Dr Gregory Harmon and study supervisor Dr Christopher Glass, professor of cellular and molecular medicine at the facility, say that the discovery may be able to significantly reduce symptoms in sufferers.

The specialists revealed that defective signalling for a protein called the peroxisome proliferator-activated receptor-y (PPAR-y) accounts for a portion of disease symptoms in cystic fibrosis, and that the correction of the defective pathway reduces symptoms of the disease in mice.

Dr Harmon pointed out that cystic fibrosis results from a genetic mutation in a membrane pore that facilitates the transport of chloride and bicarbonate electrolytes from inside the cell to the spaces outside the cell.

He added: "Loss of the cystic fibrosis pore channel results in inflammation and mucus accumulation. It also results in dehydration of the cell surfaces that make up the lining spaces inside the lungs and other affected organs, such as the intestinal tract."

Dr Harmon revealed that the fact a drug may be able to activate bicarbonate transport without affecting chloride transport is what could result in an improvement in the disease.

"The finding of the reduced PPAR-y activating prostaglandin in cystic fibrosis is exciting since it could serve as a marker to identify which patients might benefit from treatment,"  the expert concluded.


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