Newly published data show that seven out of 67 children who received Bluebird Bio’s gene therapy for a devastating neurological disorder in clinical trials have since gone on to develop blood cancers.
That means four additional patients have developed blood cancers since June 2022, when concerns about three cancer cases prompted the Food and Drug Administration to hold a hearing of outside advisers before approving the treatment, marketed as Skysona. One patient died from complications of cancer treatment. Researchers expect more children will develop cancer in coming years and are closely monitoring recipients with regular blood draws.
“All of us who are in this space would give anything for there not to be [more cases],” said Christine Duncan, a senior physician at Boston Children’s Hospital and lead author on the new study. “But I think that that is not a practical likelihood.”
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