Gene Therapy Success in Trial on “Bubble Boy” Disease

Great news from the uncontroversial biotech file: “Bubble Boy” disease, named after David Vetter who became famous because he had to be isolated behind plastic shields to prevent infection from a genetic disease that causes severe defects in the immune system, has been effectively treated in human trials using gene therapy. From the story:

Gene therapy seems to have cured eight of 10 children who had potentially fatal “bubble boy disease,” according to a study that followed their progress for about four years after treatment. The eight patients were no longer on medication for the rare disease, which cripples the body’s defenses against infection. The successiful treatment is reported in Thursday’s issue of the New England Journal of Medcine and offers hope for treating other diseases with a gene therapy approach…

Researchers removed marrow cells from the patients, equipped the cells with working copies of the gene for the enzyme, and injected the cells back into the patients. In most cases, that was done before age 2. The journal article reports the outcome two to eight years later, with an average of four years. All 10 patients were still alive, but two needed further treatment. None showed signs of leukemia or other health problems from the therapy, the researchers said…

The new findings are good news for the idea of using gene therapy to treat some other blood cell disorders, including sickle cell disease, said Kohn, who didn’t participate in the new study.

This is good. And it seems a form of adult stem cell therapy since the stem cells in the reinjected bone marrow thereafter produced immune cells that were healthy.

This is a very serious disease: David Vetter died at age 12. Gene therapy is an ethical hope for a more healthy future for humankind.