Ten newborns with the rare genetic disorder known as “bubble boy” disease were cured with gene therapy, researchers revealed Wednesday. The treatment appears to have completely rid the babies of their immune disorder with no side effects or complications — a result scientists have sought for decades through painstaking research and heartbreaking setbacks.
An earlier attempt to use gene therapy to treat severe combined immunodeficiency disease, or SCID, was halted in 2003 after researchers realized the therapy was giving the children cancer. The treatment unveiled Wednesday does not appear to carry such calamitous side effects, and experts hope it could help advance cures for other rare genetic disorders such as sickle cell disease.
“It’s a game changer,” said Jennifer Hemall, a pediatric immunologist at Children’s Hospital of Philadelphia, who was not involved in the study. “For immunologists following this disease, gene therapy has always been out there as the hope of the future. It’s exciting to see this wave of treatments actually becoming a reality.”
Infants with SCID are essentially born without a functioning immune system. Without treatment, they rarely survive past their first birthday and can be killed by infections as innocuous as the common cold. Such children were once kept isolated in sterile environments, giving rise to the term “bubble boy.” Their unusual predicament has caught national attention and been featured in movies and TV shows…