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

DATE: THURSDAY, September 1, 1994                   TAG: 9409010094
SECTION: NATIONAL/INTERNATIONAL                    PAGE: A-14   EDITION: METRO 
SOURCE: Associated Press WASHINGTON
DATELINE:                                 LENGTH: Medium

CF PATIENTS GET NORMAL LUNG GENE, TEMPORARILY

Scientists have proved they can safely transfer normal genes into the lungs of cystic fibrosis patients, and now are researching how to make those genes live long enough to fight the fatal disease.

In a study published today, scientists report they used a modified cold virus, called adenovirus, to give four cystic fibrosis victims' lungs a vital gene.

But those genes lived only about 10 days. So researchers are designing a new study to see how often they can repeat this gene therapy until CF patients experience a benefit.

``We know we can transfer a gene to the lung,'' said Dr. Ronald Crystal, who developed the treatment at the National Institutes of Health. ``Now the critical question is: Can we do it more than once?''

Cystic fibrosis patients inherit a defective gene that prevents salt from being filtered out of cells lining the airways. As a result, heavy mucus builds up and destroys the lungs, leading to death, usually by age 30.

In the journal Nature Genetics, Crystal detailed how he added a normal salt-filtering gene to adenovirus that was disabled so it wouldn't cause a cold, and dripped it into four patients' lungs.

Although the patients remain stable, the study didn't prove the gene worked, only that it could be transferred.

Crystal, now at Cornell University, previously disclosed some early results of the study in interviews and another journal. But his latest paper unveils three important findings:

The new gene, not permanent since it's merely carried by a virus, lived only 10 days.

Patients showed no signs of developing immunity to the gene-virus solution. So Crystal hopes to start a study this fall to see if he can safely give patients the gene once a month for six months.

The safe dose of the genetic solution is 5 milliliters, discovered after one patient who received four times that dose experienced moderate side effects.

``Although gene therapy for CF is still years away, this research provides another piece of a complicated puzzle,'' said Dr. Claude Lenfant, director of the National Heart, Lung and Blood Institute.

Crystal now has treated an additional five patients, and said he is seeing similar results.

CF affects one of every 3,000 babies born in the United States.

by CNB