Ibuprofen stalls advance of cystic fibrosis.

Heavy daily doses of a leading over-the-counter drug for arthritis can dramatically retard the progressive and ultimately lethal lung deterioration that characterizes cystic fibrosis, a new study finds. However, the novel treatment's benefits appear to be restricted largely to children.

Respiratory infections plague individuals with cystic fibrosis (CF), a genetic disease that fills the lungs with mucus. To fight the bacteria and other microbial invaders behind those infections, the body recruits a host of different agents, including neutrophils -- a class of white blood cells that functions as the immune system's rapid-deployment commandos. Unfortunately, lung tissue often succumbs to friendly fire from those neutrophils.

About 10 years ago, a team of Cleveland-based researchers began investigating the potential for ibuprofen, a nonsteroidal, anti- inflammatory drug, to shield the lungs of CF patients from the neutrophils' ravages. In the March 30 New England Journal of Medicine, they report its success in a double-blind study involving 85 patients between the ages of 5 and 39.

Reasoning that the anti-inflammatory therapy would work best where little permanent structural damage had occurred, the researchers restricted their trial to patients with only mild lung deterioration. Throughout the 4-year study, they charted lung function with a series of assays. Chief among them was FEV1, the volume of air an individual can forcefully expel in 1 second.

Healthy children should experience no FEV1 change from year to year, notes pediatric pulmonologist Michael W. Konstan of Case Western Reserve University in Cleveland, but "most CF patients have close to a 4 percent decline per year." That's about the rate of FEV1 decline his team witnessed in that half of the patients who received up to 16 inactive tablets daily (molded to look like the real drug). Declines were much smaller among those who received ibuprofen for 4 years -- 88 percent smaller for patients who were under 13 when the study began.

"We don't know if this treatment will benefit patients with more severe disease," Konstan says. But he says the findings "look promising enough that most CF centers are going to institute this as a therapy." The Bethesda, Md.-based Cystic Fibrosis Foundation, which helped fund the research, is drawing up guidelines to help physicians determine daily dosages of the drug.

While ibuprofen's long-term benefit "can only be surmised" from a 4-year trial, the new data strongly support the drug's efficacy, argues Harvey R. Colten of Washington University in St. Louis in an accompanying editorial.

In the United States, CF is the most common fatal genetic disorder. Some 1,000 new cases are diagnosed each year. Gene therapy to cure the disease (SN: 9/3/94, p.149) -- currently the holy grail of CF research -- will likely benefit only those patients with mild lung damage, Konstan believes.

"What's so nice about ibuprofen therapy," he says, "is that it will delay the progression of the disease so that when gene therapy becomes a reality, more patients will be able to benefit from it."