
                         Roanoke Times
                 Copyright (c) 1995, Landmark Communications, Inc.

DATE: THURSDAY, March 19, 1992                   TAG: 9203190083
SECTION: NATIONAL/INTERNATIONAL                    PAGE: A-6   EDITION: METRO 
SOURCE: Associated Press
DATELINE: BOSTON                                LENGTH: Medium

CYSTIC FIBROSIS `SPRAY' EFFECTIVE

Cystic fibrosis patient Ian Ferguson says he usually would become winded running up a flight of stairs. But after receiving a genetically engineered protein, he could bound up three flights without losing his breath.

Ferguson, a 27-year-old architect from Rockville, Md., was one of 16 people who tested the new medicine researchers say is the first drug to effectively break up the infected, lung-clogging mucus that is a major hazard of cystic fibrosis.

About 50,000 Americans have the inherited illness, which is often fatal by age 30. Until now, it has been treated mostly by giving antibiotics to prevent infections and pounding on patients chests to loosen mucus buildup.

The medicine, deoxyribonuclease, or DNase, is a copy of a natural human protein and administered in an aerosol spray that is breathed through the mouth. Researchers from the National Heart, Lung and Blood Institute in Bethesda, Md., conducted the first test of the medicine's effectiveness. They reported their findings in today's New England Journal of Medicine.

"It's not a cure, but there is no question it works," said Dr. Ronald Crystal, who directed the study.

The medicine's maker, Genentech Inc., plans to seek federal permission to sell the drug in early 1993. It is now available only in carefully controlled studies.

Ferguson took the medicine for just six days during the preliminary study, and he hopes it will be approved quickly for routine use.

DNase is being tested on 900 people at 50 hospitals around the country.

"This is very exciting," said Dr. Robert Beall of the Cystic Fibrosis Foundation. "There was a very significant improvement in pulmonary function. We think this could have an impact on the disease."

The new treatment is a natural human protein, produced in quantity by gene splicing, that breaks down the genetic material DNA.

In people with cystic fibrosis, the cells lining the lungs absorb too much water. This dries out the mucus, making it thick and hard to cough up. Bacteria grows in the mucus.

White blood cells fight this infection. When these cells die, they release their DNA into the mucus. This makes the mucus even thicker and stringy, severely hindering breathing.

When breathed in a nasal spray, DNase attacks this leftover DNA, thinning out the mucus.

by CNB