Bluebird Bio's Skysona for CALD Linked to Blood Cancer Risks in Children

Skysona, developed by Bluebird Bio and approved by the FDA, is a personalized gene therapy designed to treat cerebral adrenoleukodystrophy (CALD), a rare degenerative brain disorder in children. However, recent studies have highlighted significant safety concerns, revealing that seven out of 67 boys treated with Skysona developed blood cancers, including six cases of myelodysplastic syndrome (MDS) and one case of acute myeloid leukemia (AML)[1][2]. These occurrences have been linked to clonal vector insertions and somatic mutations, potentially due to the lentiviral vector technology used in the therapy. Despite these risks, many patients achieved stable neurological function, indicating benefits that might outweigh the potential dangers, though this balance remains under scrutiny[1][2].