By Weiss, R.
Science News , Vol. 138, No. 12
First Human Gene-Therapy Test Begun
Culminating years of laboratory studies, legal wrangling, and scientific and ethical debate, researchers at the National Institutes of Health last week performed the first federally approved infusion of therapeutic, genetically engineered cells into a patient.
A billion or so gene-altered immune-system cells, suspended in about 2 ounces of sterile saline solution, made medical history as they dripped down a plastic tube into the vein of a 4-year-old girl with an inherited, life-threatening immune deficiency.
Scientists and physicians involved in the case say that because of the experimental treatment's conservative design, they expect to detect few if any benefits for the young patient within the next year or more. Nonetheless, by providing the girl's circulatory system with a small army of cells bearing the critical gene she has lacked since conception, medical practitioners took their first tentative steps into the world of gene therapy.
That world's landscape stands in sharp contrast to the familiar scenery of current medical practice. Its landmarks are not the body's organs and cells or the microorganisms infecting them, but the basic molecular and genetic elements from which all inherited traits arise. Scientists expect gene therapy to prove valuable against a range of medical problems, including inherited diseases, cancer, cardiovascular diseases and possibly AIDS.
"Gene therapy is potentially a major, new therapeutic option that should have significant clinical effects in the next century," says NIH researcher W. French Anderson. "This should provide cures for what today are incurable diseases."
Anderson and co-workers R. Michael Blaese and Kenneth W. Culver directed the infusion, nearly 3-1/2 years after first submitting to NIH officials their unprecedented proposal to correct genetic errors in the cells of patients with an inherited disease. That highly technical, several-inches-thick document -- dubbed "The Phone Book" because of its size -- went through seemingly endless major revisions over the years. The changes reflected requests by various federal agencies and scientific advisory boards for additional information relating to the procedure's risks, potential benefits and ethical implications.
Final FDA approval arrived at 8:59 a.m. on Sept. 14. Within hours, the young patient -- whose identify remains unpublicized at the family's request -- was wheeled into the pediatric intensive care unit at the NIH clinical center in Bethesda, Md. There, over a period lasting about 30 minutes, her left arm received the nation's first infusion of gene-altered, therapeutic cells. …