Eight for eight. That's the record scientists at St. Jude Children's Research Hospital are thrilled about after using gene therapy to cure eight baby boys of the rare disorder commonly known as "bubble boy" disease, reports the BBC. What's more, the researchers used HIV to do it, notes the AP. The infants had the most common form of severe combined immunodeficiency disease, or SCID, one called SCID-X1. Essentially, they were born without a functioning immune system, meaning even a common cold could prove deadly. The solution? Researchers extracted blood cells from the babies, inserted a genetic fix into the cells, then put them back into the infants, explains NPR. In this case, the genetic fix was delivered via a modified version of HIV, one that can't give the babies AIDS.
“It’s a game changer,” an immunologist at Children’s Hospital of Philadelphia who was not involved in the study tells the Washington Post. “It’s exciting to see this wave of treatments actually becoming a reality.” Gene therapy has been tried before on SCID with some degree of success, but many of the previous patients developed cancer. The latest experiment, published in the New England Journal of Medicine, has safeguards to prevent that, including a small amount of chemotherapy. Bone-marrow transplants also have proved successful in treating the disease, but the best-case scenario—a "tissue-matched sibling donor"—is not available in most cases, say researchers in a statement. All eight babies in the study are now toddlers with healthy immune systems. (More discoveries stories.)
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