Babies with ‘bubble boy’ disease cured with HIV treatment — study

By Amanda Woods

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(NEW YORK POST) — Eight babies suffering from a rare immunodeficiency virus commonly known as “bubble boy disease” have been cured with an unlikely treatment — gene therapy made from the HIV virus, a new study reveals.

The treatment, conducted at St. Jude Children’s Research Hospital in Memphis. Tenn., was detailed in a study published by the New England Journal of Medicine Thursday.

As part of the study, scientists managed to alter the HIV virus so that it wouldn’t cause disease — and then used it to deliver a gene that the infants lacked.

The babies suffered from severe combined immunodeficiency syndrome, or SCID, which is caused by a genetic flaw — and is often called “bubble boy disease” following a 1970s case of a Texas boy who lived for 12 years in a plastic bubble to shield him from germs.

The illness affects one in 200,000 newborns — almost all males — and often causes death within the first year or two of life without treatment.

But the tots treated as part of this study now have fully functioning immune systems, researchers say.

“The children are cured,” Dr. Ewelina Mamcarz, one of the study leaders at St. Jude, told NBC News. “They came to us as little infants, some of them as young as 2 months, with severe infections. Now they are home, living normal lives, attending daycare.

“The treatment, pioneered by St. Jude doctor Brian Sorrentino who recently died, involves removing some of the babies’ blood cells, using the altered HIV to insert the gene that the patients lack — and returning the cells through an IV. Before the cells are returned, the babies are given a drug to destroy some of their marrow, which grants the modified cells more space for growth.

The research is ongoing, and Omarion Jordan, who will turn 1 later this month, is the 10th boy to be treated. He received the therapy back in December.

“For a long time we didn’t know what was wrong with him. He just kept getting these infections,” his mom Kristin Simpson, told the AP, calling her son’s condition at the time “just heartbreaking.”

But that has turned around, she said.

“He’s like a normal, healthy baby,” Simpson said. “I think it’s amazing.”

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