4-Year-Old Girl Becomes First Gene Therapy Patient

A 4-year-old girl received an intravenous injection Friday and made history by becoming the first patient to receive gene therapy.

“The patient is doing beautifully,” said Dr. French Anderson, a scientist at the National Heart, Lung and Blood Institute.

“The family now has new hope for their child,” said another researcher, Dr. Kenneth W. Culver of the National Cancer Institutes.

During the 28-minute infusion procedure, the girl--who was not identified--sat up in her bed, played and put stickers on the lab coats of her doctors.

“I’m sure this will be one of the most closely watched human beings on the face of the earth today,” said Don Ralbovsky, a spokesman for the National Institutes of Health in Bethesda, Md., where the procedure was conducted.

The therapy involves inserting curative genes into human cells. Its first therapeutic use was given final approval only hours before by the Food and Drug Administration.

Success could lead to use of the procedure against cancer, hemophilia, sickle cell anemia, AIDS and other diseases in years to come.

The child suffers from a rare genetic disease called ADA Deficiency, which weakens her immune system so that it cannot fight infection and disease.

About 1 billion cells--approximately a small thimbleful--were mixed in a saline solution in the first infusion.

Anderson said he would not expect to see results for the first several months, but that within a year they should know whether the treatment is working.

The National Institutes of Health has approval to treat up to 10 patients with ADA Deficiency.