![]() ![]() Made famous by the television show “Seinfeld,” the real “bubble boy” was no joke. You can contact NPR science correspondent Richard Harris at. Now, with continual advances in treating SCID, "it's just nice to see another success for gene therapy," Kohn says. Kohn at UCLA says that for more than a decade, it seemed that the field of gene therapy was a dead end.Ĭlearly it has made a comeback and has been used to treat other rare diseases, including adrenoleukodystrophy, a neurological condition better known as Lorenzo's Oil disease, after a 1992 movie that highlighted a boy with the condition and his parents' hunt for a cure. It works in the laboratory, "and this really sets the stage then for a clinical trial, hopefully in the next 12 to 18 months," he says.Īll this makes the leukemia setback from the 1990s feel like a fading memory. "Sometimes their families don't understand just how profoundly affected their immune system is."įor example, instead of inserting a healthy gene into blood cells, Porteus, the Stanford pediatrician, has used a precision gene-editing technique called CRISPR to correct the genetic error in blood cells drawn from SCID patients. Screening for SCID is now done throughout the U.S., though its introduction was gradual and state by state.īefore screening was instituted, these children used to show up in the hospital with life-threatening infections, "and now we're seeing happy, bouncy little newborns who just look perfectly normal and they're never sick," Puck says. In her view, the key to treatment is finding these children early - through newborn screening - before they start to get life-threatening infections. ![]() Of course, she adds, they'll be watched carefully for signs of leukemia and to see if the effect of the therapy is wearing off. These children are "growing normally, they're getting colds like everyone else and they're getting over infections - so I would say that is a cure," Puck says. Jennifer Puck, a pediatrician at UC San Francisco and a collaborator in the latest study, says infants who got the gene therapy don't need that medication. As a result, patients require monthly infusions of antibodies called immunoglobulins. But that procedure often restores only part of a child's immune system. Standard treatment for SCID is a bone marrow transplant. They aren't any different from my daughters." Two more infants have been treated since the paper was prepared for publication, the team says. "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. ![]() Scientists went to work to figure out how to inject new genes into cells without triggering leukemia, a cancer of blood cells. He says 18 of those original patients are still alive today, but the leukemias put an understandable pall on the whole field of gene therapy. "But, over time, five of them went on to develop a leukemia." "Of the 20 patients, they all had immune recovery," says Donald Kohn, an immunologist at UCLA's Broad Center of Regenerative Medicine and Stem Cell Research. Those cells then build up the patient's immune system.Īt first, this treatment in the 1990s and early 2000s looked really promising. This process involves removing defective blood cells from a patient, inserting a new gene with the help of a virus and then putting the cells back into the body. In the 1990s, European scientists actually cured SCID in some patients, using a technique called 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. Simon Says Opinion: The Doctor And 'The Boy In The Bubble'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. ![]()
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