LONDON (Reuters) - A three-year-old "bubble boy" undergoingpioneering gene therapy in London has developed leukemia,marking another setback for the experimental treatment.
Doctors at Great Ormond Street Hospital said on Tuesday theboy had been successfully treated for SCID-X1, or x-linkedsevere combined immunodeficiency, often known as "baby in thebubble syndrome," but had developed leukemia two years later.
The news is a blow to the treatment program at the Londonhospital, which has a worldwide reputation from treating sickchildren.
Although gene therapy has been linked to leukemia before,this is the first such case within the London hospital'sprogram. Doctors said they were now seeking improvedformulations of the genetic medicine for new trials next year.
Five years ago, similar cases of leukemia were seen in twoFrench boys given gene therapy. Researchers believe in thatcase the experimental treatment triggered a gene that causedtheir bone marrow to overproduce immune system cells calledT-cells.
Leonard Seymour, professor of gene therapies at theUniversity of Oxford, said the latest case showed the need tobalance the risks for patients, given that gene therapy isoften the only hope.
Childhood leukemia is generally treatable, with cure ratesof 90 percent or more. In contrast, children with SCID whocannot get a bone marrow transplant almost always die within ayear.
(Reporting by Ben Hirschler)
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