New York Post

HIV’S CURE POWER

Kills ‘bubble’ disease

- By AMANDA WOODS With Wire Services awoods@nypost.com

Eight babies suffering from a rare immunodefi­ciency virus commonly known as “bubble boy disease” have been cured with an unlikely treatment — gene therapy based on the HIV virus, a new study has revealed.

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

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.

It became known as “bubble boy disease” following the famous 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 day care.”

The treatment, pioneered by St. Jude physician 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 latest boy to be treated. He received the therapy last 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 Associated Press, 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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