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Gene Therapy Might Treat 'Bubble Boy' Disease
Date:5/21/2010

Researchers, who began the study in 2001, assigned four patients to receive regular treatment with enzyme replacement therapy. Another six stopped that therapy and received chemotherapy designed to create room in their bone marrow for transplanted cells.

"It would be like if you're in a garden with a high density of plants, and you want to put some new plants in. The best chance for them to grow would be to remove some of the old plants," explained Dr. Mark Kay, head of the gene therapy program at Stanford University School of Medicine.

Researchers hoped the transplanted cells would create a new immune system and, in half of the six patients, they did just that.

The treatment gives the patients a "key missing piece of genetic information" so their bodies can create a normal immune system, Kohn said. And the cells come from the patient's own body so there should be less risk that the body will reject the cells or vice versa, he explained.

The study results came in a phase 1 trial, the first of three study phases that treatments must undergo to get federal approval. The second phase of research has already begun.

The treatment is costly, Kohn noted, requiring months of hospitalization.

Kay said the gene therapy treatment may become the standard way to treat bubble boy disease. It could, he said, allow children to undergo just one treatment -- a "lifelong cure" -- instead of repeatedly taking the enzyme treatment.

The study is scheduled to be released Friday at the annual meeting of the American Society of Gene & Cell Therapy, in Washington D.C.

More information

The Nemours Foundation has more about immune deficiency.



SOURCES: Donald B. Kohn, M.D., professor, department of microbiology, immunology and molecular genetics and dep
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