![]() 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. SCID is a test case for all those scientists working to develop better gene therapy techniques.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. This latest advance is not only encouraging news for these rare patients. It targets a different mutation that causes SCID, but the technique is very much the same, including the brief dose of chemotherapy. A similar treatment, called Strimvelis, has already been approved by Europe's equivalent of the Food and Drug Administration. It has an exclusive licensing agreement with Mustang Bio to develop a product. Jude hopes to commercialize its treatment. Shots - Health News Parents Lobby States To Expand Newborn Screening Test For Rare Brain Disorder "Sometimes their families don't understand just how profoundly affected their immune system is." 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.
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