Seven of the 67 children who received Bluebird Bio’s gene therapy for a serious neurological disease in a clinical trial went on to develop blood cancer, according to newly released data.
This means that four more patients have developed blood cancer since June 2022. In June 2022, concerns over three cancer cases prompted the Food and Drug Administration to hold a hearing with outside advisers before approving the treatment, sold as Skysona. One patient died from complications of cancer treatment. Researchers expect many more children to develop cancer in the coming years and are closely monitoring recipients with regular blood draws.
“All of us in this space are willing to do anything to do nothing.” [more cases]” said Christine Duncan, a senior physician at Boston Children’s Hospital and lead author of the new study. “But I don’t think that’s a realistic possibility.”
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