"To be able to see these babies in my clinic now as toddlers is very rewarding," she says. "I am thrilled to see these outstanding results," says Ewelina Mamcarz, a transplant physician and first author on the new paper. The babies developed apparently healthy immune systems, according to the new study. This helped the new cells take up permanent residence. Jude's Children's Research Hospital in Memphis modified the procedure for SCID by giving the infants a short course of chemotherapy before introducing the new gene. Gene therapy has been used successfully over the past decade. That engineered virus can't cause AIDS and it's been further tweaked to reduce the risk that it could trigger leukemia. The gene to correct the problem was inserted into a modified version of HIV, the virus that causes AIDS. The latest advance, reported in the New England Journal of Medicine Wednesday, details a study of eight infants who have a type of SCID called SCID-X1. Since then, there has been gradual improvement in gene therapy. "This is one of those diseases in which there's probably more doctors and scientists studying the disease than patients who have the disease," Porteus says. The disease remains a source of great interest to researchers. He died at the age of 12 in 1984.Īll babies born in the United States are now screened for this condition, and the best treatment today - a bone marrow transplant - succeeds more than 90 percent of the time. "It was made famous in the mid '70s when the 'Bubble Boy' was described in a documentary, and I think it captured the imagination of a lot of people," says Matthew Porteus, a pediatrician at Stanford University.ĭavid Vetter was the boy who spent most of his short life inside a plastic bubble to protect him from infection. The disease is called severe combined immunodeficiency, or SCID. These infants are born without a functioning immune system. That's been the case with a condition that strikes fewer than 100 babies a year in the United States. Sometimes rare diseases can let scientists pioneer bold new ideas.
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