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BlueBird Bio - Finished

















































Tragic. The nest has been reduced to ashes.​

Seven children develop cancer after receiving Bluebird gene therapy for rare neurological disease​

Seven of the 67 children who received Bluebird Bio’s gene therapy Skysona have developed blood cancer, new research shows — and one patient has died from complications stemming from the cancer treatment.

In all seven cases, it seems as though the lentivirus used to ferry the therapy throughout the patients’ bodies disrupted a gene linked to cancer, STAT’s Jason Mast writes. Six patients developed myelodysplastic syndrome, an early malignancy that can progress into acute myeloid leukemia, or AML. One patient has developed AML. It’s still unclear why this particular treatment is so prone to triggering blood cancers.

But Skysona is still a critical treatment for cerebral adrenoleukodystrophy, a deadly neurodegenerative disorder, and doctors still intend to offer the treatment.

“This disease is awful, and until we have better, I think families and patients need to have choices,” the physician who detailed these cases in the New England Journal of Medicine told STAT.

Read more.
 








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