A gene therapy that makes use of an unlikely helper, the AIDS virus, gave a working immune system to 48 babies and toddlers who were born without one, doctors reported Tuesday.
Results show that all but two of the 50 children who were given the experimental therapy in a study now have healthy germ-fighting abilities.
“We’re taking what otherwise would have been a fatal disease” and healing most of these children with a single treatment, said study leader Dr. Donald Kohn of UCLA Mattel Children’s Hospital.
“They’re basically ‘free range’ — going to school, doing normal things,” without the worry that any infection could become life-threatening, he said.
The other two children who weren’t helped by the gene therapy later had successful bone marrow transplants. Doctors say it will take longer to know if any of the 50 are cured, but they seem to be well so far.
The children had severe combined immunodeficiency syndrome, or SCID, which is caused by an inherited genetic flaw that keeps the bone marrow from making healthy versions of the blood cells that form the immune system. Without treatment, it often kills in the first year or two of life.
It became known as “bubble boy disease” because of a case in the 1970s involving a Texas boy who lived for 12 years in a protective plastic bubble to isolate him from germs.
It’s now called “bubble baby disease” because roughly 20 different gene defects, including some that affect girls as well as boys, can cause it.
Results of the UCLA-led study were published Tuesday by the New England Journal of Medicine and presented at an online American Society of Gene & Cell Therapy conference.
Grants from U.S. and British government health agencies and the tax-supported California Institute for Regenerative Medicine paid for the work. Kohn is an inventor of the treatment and an adviser to the company now developing it, London-based Orchard Therapeutics.
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