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Bluebird Bio Gene Therapy Benefits Marred by 7 Children Developing MDS and AML

Oct 9, 2024, 09:25 PM

Bluebird Bio's gene therapy for cerebral adrenoleukodystrophy has shown long-term benefits for patients, with most of the 32 treated patients remaining free of major disability after a median of 6 years. However, concerning findings have emerged, with seven children developing blood cancer, specifically myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), 14 to 92 months post-treatment. These cases were previously disclosed to investigators and the FDA, leading to an update in the Skysona label in April. The study, published in the New England Journal of Medicine (NEJM), highlights the difficult choices faced by families of affected children. The research involved lead and senior authors from Dana-Farber.