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Children's Health

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Cystic Fibrosis Advance Shows Promise

Inexpensive Treatment Cuts Infections, Boosts Lung Function

The Real Goal: Treating Infants

What really excites cystic fibrosis researchers is the possibility that very young children might be able to use hypertonic saline. Why? There's the chance that the treatment could keep kids with cystic fibrosis from getting germ-filled mucus plugs in the first place.

"I think younger patients are going to be key," Ratjen says. "If you have established mucus deep in your lungs, in these areas already plugged by mucus there will be no effect of treatment. But if you start treatment in infancy, that would be a different story. You could potentially have a much more pronounced effect on cystic fibrosis lung disease."

Tests are already under way.

"We are already testing the safety and efficacy of inhaled hypertonic saline in 4- and 5-year-olds," Donaldson says. "And we have a plan to start testing in infants, newborns, and 1- to 2-year-olds. The idea is to stop them from getting mucus plugs, rather than have the infectious cascade happen."

Not an Easy Treatment

Hypertonic saline has its limitations. It's certainly no cure for cystic fibrosis. Patients can only inhale the salty mist so far into their lungs, leaving deep mucus plugs untouched.

The saline solution used is usually about 7% salt. Human blood is only 1% salt. The Atlantic Ocean is 4% salt, while the Dead Sea is 12% salt. At first, patients have a hard time getting used to the treatment.

"The first time you inhale it you feel like you are drowning," Boucher says. "Patients really do mobilize secretions, so they get a funny feeling in the chest. It is different. It is different tasting, a different sensation, but the lung will feel different, too."

The stuff tastes terrible, Ratjen says. And treatment takes a long time -- 15 minutes, twice a day at least. Still, only about one in 20 patients finds it intolerable. Most patients get used to it, and some can tolerate even more-concentrated salt solutions.

In fact, Boucher says, "patients love it." Once they've been on the treatment for a while, he says, they don't want to enroll in clinical trials that don't guarantee hypertonic saline treatment.

And hypertonic saline may not be the best way to restore the lung's protective layer of water. Researchers are experimenting with a powdered sugar compound that tastes better -- and would reach deeper into the lungs. And there's hope that in the future, science may find a drug that restores surface water to cystic fibrosis lung cells.

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