BioPharma Dive October 10, 2024
Seven young boys given Bluebird’s Skysona later developed blood cancers, findings that could shape how doctors balance the treatment’s risks against its benefit.
About two years ago, the Food and Drug Administration approved a personalized gene therapy for an ultra-rare childhood brain disorder despite concerns treatment might inadvertently trigger cancer.
Those concerns are now back in the spotlight, as new study data published Wednesday in The New England Journal of Medicine document seven cases of blood malignancies in young boys given the therapy, called Skysona.
The cases are among 67 boys with cerebral adrenoleukodystrophy, or CALD, who were enrolled in studies of Skysona. In the months and years after treatment, six developed myelodysplastic syndromes, or MDS, a form of bone...
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