On Apr. 18, 2019, St. Jude announced a cure for SCID-X1, commonly known as bubble boy disease. By combining gene therapy and low-dose chemotherapy with busulfan, immune function is restored in infants with the disorder. Major support for the gene-therapy work also came from the California Institute of Regenerative Medicine, the National Institutes of Health and ALSAC, the fundraising and awareness organization for St. Jude. Money from many private donors also helped fund the studies.

Findings published Thursday in the New England Journal of Medicine detailed how kids treated at St. Jude and a San Francisco hospital received gene therapy for the often-fatal disorder that had left them with little or no immune protection. The disease, caused by a mutation in a gene critical to normal immune function, drew national attention in the 1970s with the plight of David Vetter, who grew up sheathed in a plastic bubble.

The gene therapy, produced in the Children’s GMP, LLC, manufacturing facility on the St. Jude campus, involved use of a virus to transport and insert a correct copy of a gene into the genome of patients’ blood stem cells. Following the treatment, the children began producing functioning immune cells for the first time, according to St. Jude, and most patients were discharged from the hospital within one month.

St.ﾠJude Children’s Research Hospital was founded in 1962 by actor Danny Thomas.