In Brief: UCLA Researchers Say Gene Therapy Can Defeat “Bubble Boy” Disease

Gene therapy can be used to restore the immune systems of children who suffer from the so-called “bubble boy” disease, condemning them to a life of antiseptic isolation.

The 11-year study of 10 children with ADA-deficient severe combined immunodeficiency by UCLA researchers concluded that a combination of chemotherapy and gene therapy restored an immune response in three of the six children who received it. A more refined version of the therapy will be used in future studies, officials said.

“Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease,” said Donald Kohn, M.D., a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. 

Currently, the only treatment available for children with the condition is twice-weekly enzyme injections.