LIFE

HIV used to fix ‘bubble boy’ disease

Gael Jesus Pino Alva, who was treated for ‘bubble boy disease’, with his mother Giannina Alva in Memphis.APPeter Barta  

They were born without a working germ-fighting system, every infection a threat to their lives. Now eight babies with “bubble boy disease” have had it fixed by a gene therapy made from one of the immune system’s worst enemies HIV, the virus that causes AIDS.

A study out on Wednesday details how scientists turned this enemy virus into a saviour, altering it so it couldn’t cause disease and then using it to deliver a gene the boys lacked.

“This therapy has cured the patients,” although it will take more time to see if it is a permanent fix, said Dr. Ewelina Mamcarz, a lead doctor at St. Jude Children’s Research Hospital in Memphis.

Omarion Jordan, who turns one later this month, had the therapy in December to treat severe combined immunodeficiency syndrome, or SCID.

“For a long time, we didn’t know what was wrong with him. He just kept getting these infections,” said his mother, Kristin Simpson. Learning that he had SCID “was just heartbreaking ... I didn’t know what was going to happen to him.”

Omarion now has a normal immune system. “He’s like a normal, healthy baby,” Ms. Simpson said.

Study results were published by the New England Journal of Medicine . The treatment was pioneered by a St. Jude doctor who recently died, Brian Sorrentino.

SCID is caused by a genetic flaw that keeps the bone marrow from making effective versions of blood cells that comprise the immune system. It affects 1 in 2,00,000 newborns, almost exclusively males. Without treatment, it often kills in the first year or two of life.

“A simple infection like the common cold could be fatal,” Dr. Mamcarz said.

The nickname “bubble boy disease” comes from a famous case in the 1970s a Texas boy who lived for 12 years in a protective plastic bubble to isolate him from germs.