Early stem cell transplant may cure “bubble boy” disease

Posted: July 31, 2014 at 7:44 pm

"Bubble boy" David Vetter lived in a protective environment designed by NASA engineers. He died of complications after receiving a bone marrow transplant in 1984, at the age of 12. Baylor College of Medicine Photo Archives

Babies born with so-called "bubble boy" disease can often be cured with a stem cell transplant, regardless of the donor -- but early treatment is critical, a new study finds.

Severe combined immunodeficiency (SCID), as the condition is medically known, actually refers to a group of rare genetic disorders that all but eliminate the immune system. That leaves children at high risk of severe infections.

The term "bubble boy" became popular after a Texas boy with SCID lived in a plastic bubble to ward off infections. The boy, David Vetter, died in 1984 at the age of 12, after an unsuccessful bone marrow transplant -- an attempt to give him a functioning immune system.

15 Photos

Immune disorder forced David Vetter to live in bubble - but breakthroughs from his story now enable similar kids to live free

In the best-case scenario, a child would get stem cells -- the blood-forming cells within bone marrow -- from a sibling who is a perfect match for certain immune-system genes.

But that's not always an option, partly because kids with SCID are often their parents' first child, said Dr. John Cunningham, director of hematopoietic stem cell transplantation at the University of Chicago Comer Children's Hospital. He was not involved in the study.

In those cases, doctors typically turn to a parent -- who is usually a "half" match, but whose stem cells can be purified to improve the odds of success. Sometimes, stem cells from an unrelated, genetically matched donor can be used.

The good news: Regardless of the donor, children with SCID can frequently be cured, according to the new findings. But early detection and treatment is vital.

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Early stem cell transplant may cure "bubble boy" disease

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