Doctors may have a way to treat a deadly disease through experimental gene therapy—and they got an assist from an unlikely source.

By Beth Ann Mayer
May 13, 2021
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A group of children who were born without working immune systems now have them—and doctors credit an unlikely helper: the AIDS virus.

Part of a study published in the New England Journal of Medicine earlier this week, doctors found that 48 of 50 children who were given new experimental gene therapy developed the ability to combat germs.

The children in the study were born with severe combined immunodeficiency syndrome (SCID), a genetic disease that prevents the bone marrow from making healthy red blood cells needed to form a working immune system. The disorder was put in public view when David Vetter, a child in Texas, spent 12 years living in a plastic bubble to keep him safe from germs. It's now known as "bubble baby" disease because it can affect any child regardless of gender. Without an intervention, kids born with bubble baby disease typically die before their second birthdays.

So, how does the new gene therapy work? Doctors removed some of the patients' red blood cells. Then, they use a disabled AIDS virus to give kids a healthy version of the gene they need to develop functioning immune systems and return the red blood cells with an IV. The children didn't report any complications with the treatment and said side effects were mild.

Though doctors can't say for sure whether it's a cure, Dr. Donald Kohn of UCLA Mattel Children's Hospital, who led the study, says the treatment has enabled the children to live more normal lives.

"They're basically 'free range'—going to school, doing normal things," without the worry that any infection could become life-threatening," Kohn says.

As reported in the Associated Press, Josselyn Kish of Las Vegas had the therapy when she was three years old and is now 11. Kish says she rarely gets sick. She did recently come down with COVID-19 but only had mild symptoms.

The other two youngsters received a bone marrow transplant after the experimental therapy. Until recently, this was the go-to treatment. But it's risky and doesn't always work—Vetter died when he got mono shortly after his bone marrow transplant. But it did help cure a pair of Arizona siblings in 2012, and doctors say the two children who were part of this study have done well so far.