Study: Researchers successfully treat babies with 'bubble boy' disease using gene therapy

Patient4Life · Apr 18, 2019

Study: Researchers successfully treat babies with 'bubble boy' disease using gene therapy USA Today

St. Jude performed the therapy on infants newly diagnosed with X-linked severe combined immunodeficiency (SCID-X1) – a genetic condition also known as "bubble boy" disease – according to a study published in the New England Journal of Medicine's April 18 issue.

The disease prevents babies from developing an immune system to fight even routine infections. In January 2018, St. Jude researchers reported that babies in the trial developed fully functioning immune systems but would be monitored further to confirm its long-term benefits.
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The gene therapy works like this: A deactivated virus is inserted into the patient’s bone marrow, which delivers the correct gene copy into blood stem cells, replacing the defective one. These cells are then frozen and undergo testing.

“This virus is able to effectively deliver a healthy copy of the gene into a stem cell in a way that was not possible before,” Mamcarz said.

The patient then receives two days of low-dose busulfan, a chemotherapy drug that makes space in the marrow for the stem cells to grow, and the cells are then infused back into the patient.
It takes about 10 days from the time the cells are taken out to when they are infused into the patient, Mamcarz said.

The proper immune cells were found within three months of the treatment in all but one patient, who needed a second dose of gene therapy, St. Jude says.

“This novel approach has shown really outstanding results for the infants,” Downing said. “The treatment has fully restored the immune system in these patients, which wasn’t possible before, and has no immediate side effects.”
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Study: Researchers successfully treat babies with 'bubble boy' disease using gene therapy

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