Roanoke Times
Copyright (c) 1995, Landmark Communications, Inc.
DATE: SATURDAY, April 6, 1991 TAG: 9104060089
SECTION: NATIONAL/INTERNATIONAL PAGE: A-2 EDITION: METRO
SOURCE: Associated Press
DATELINE: BETHESDA, MD. LENGTH: Medium
National Institutes of Health scientists, updating an advisory panel on the status of the experiment, said the youngster has been improving steadily and has had few side effects from the treatment.
"I'm delighted at the way things are going," said Dr. R. Michael Blaese, a National Cancer Institute scientist and co-researcher in the gene therapy experiment. "The patient's immune system seems to be improving."
The patient, who suffers from a severe immune disorder that leaves her defenseless against infections and cancers, was given her sixth treatment on Thursday. She began the treatments last September, becoming the first person to undergo gene therapy. She will have two more before researchers do an extensive evaluation of her condition, Blaese said.
A second child is also being treated for the same genetic deficiency, but it is too early to evaluate any changes in her condition, he said. Another child may be started on the experimental therapy in a few months, he said.
Dr. French Anderson, co-researcher on the project, said the first patient's improvement in immune function "has gone on long enough" for him to be confident that it's due to the gene therapy and not some other factor.
But Blaese cautioned that while the treatment appears to be working, "whether it's going to be an effective treatment is another issue."
"This is a very complicated process and it has many ramifica The experimental therapy inserts the missing . . . gene into the patient's blood cells and then the blood is returned to the patient. tions," he explained. "We're not going to be in a position of making conclusions about whether this is a worthwhile treatment for a long time.
The children in the experiment were born without a gene that produces an enzyme called adenosine deaminase, or ADA, which is essential for a healthy immune system.
The experimental therapy inserts the missing ADA gene into the patient's blood cells and then the blood is returned to the patient. In the bloodstream, these genetically altered cells begin secreting ADA.
Blaese said the first patient's ADA level was less than 1 percent of normal when the therapy began. It is now up to about 18 percent of normal, he said.
The only side effect the 4-year-old suffered during treatment was a fever, which persisted for several hours after two treatments, Blaese said.
by CNB