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

Chemo clears way for transplanted cells to help children develop immune systems

FRIDAY, May 21 (HealthDay News) -- Scientists report that they've successfully used gene therapy to treat a small number of patients with the condition known as "bubble boy" disease, which robs children of the ability to fight off germs.

The research is preliminary, and another phase of testing is needed before the therapy can get federal approval. Also, the treatment only appears to work in patients with a particular strain of the disease.

Even so, the findings highlight the fact that the disease is becoming easier to treat and the prognosis is improving for these children, said lead investigator Dr. Donald B. Kohn.

"We're talking about 70 to 90 percent surviving and doing well, and we're hoping to get that even higher as gene therapy gets more effective," said Kohn, a pediatric bone-marrow transplant doctor at the University of California at Los Angeles David Geffen School of Medicine.

When they're born, babies have natural resistance to disease, thanks to immunity that they inherit from their mothers. Over time, if things go well, they develop immune systems of their own. But in some cases the immune systems don't develop properly and the children become especially vulnerable to disease.

The condition is rare, but it became widely known in the 1980s because of the case of the "Bubble Boy," a child named David Vetter who lived in a plastic bubble to avoid being infected by germs. He died at the age of 12, and his treatment remains controversial to this day.

Currently, children with the different types of the immune deficiency condition are treated with stem-cell transplants and gene therapy.

In the new study, researchers treated 10 children who had a form of the condition called adenosine deaminase severe combined immunodeficiency that affects 20 percent of those with bubble boy disease.


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