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

DATE: THURSDAY, March 8, 1990                   TAG: 9003081993
SECTION: NATIONAL/INTERNATIONAL                    PAGE: A/6   EDITION: EVENING 
SOURCE: Associated Press
DATELINE: WASHINGTON                                 LENGTH: Medium

FIRST HUMAN GENE-TRANSFER THERAPY URGED

The first human gene-transfer therapy could begin within a year if other government panels join a National Institutes of Health committee in approving the experimental treatment to combat a rare immune deficiency in children.

Six other federal committees must concur with Wednesday's decision by the NIH Institutional Biosafety Committee before the experimental therapy can be tried.

The treatment would be the first time that a human illness had been directly treated by the process of gene transfer. The process would insert curative genes into the blood cells of children suffering adenosine deaminase deficiency.

If approved, clinical use could begin by late fall or winter.

The proposal was presented by Dr. French Anderson of the Heart, Lung and Blood Institute, one of the National Institutes of Health, and Dr. Michael Blaese of the National Cancer Institute.

Blaese said the clinical plan is to remove lymphocytes - white cells - of the blood of children with the immune deficiency and insert into them the gene they lack.

Lymphocytes are the major infection-fighting element of the immune system.

The treated cells then would be cultured until they increase in number by about a thousandfold, and then transferred back into the patient's bloodstream.

Adenosine deaminase is an enzyme that performs an essential function in the immune system. Without the enzyme, the body's ability to fight infection is crippled.

The gene that would be inserted would program the cells to produce the missing enzyme.

The therapy would not be a permanent cure, Blaese said, since the enzyme would be produced only as long as the genetically manipulated cells live.

"We don't know how long these cells last," he said. "Some lymphocytes, however, can live for years."

The researcher said the therapy might allow patients to live longer than they would otherwise. However, scientists hope to develop a system where the gene could be placed in the cells of the bone marrow, which would permanently correct the enzyme deficiency.

Adenosine deaminase deficiency is "very, very rare," said Blaese. "There are only 20 to 30 patients in the whole world."

The sufferers are born without the required gene. Some are treated successfully by bone marrow transplants, but for many the disorder means death within the first year or two of life by some infectious disease.

by CNB