Bluebird case reminds us of gene therapy's risks

When gene therapy was first tested in a clinical setting, it unintentionally triggered an adverse reaction that led to the patient’s death. This week at ASH, we were reminded that the safety risks for these cutting-edge treatments are still very much present: Bluebird Bio reported that a patient who received its Lentiglobin gene therapy for sickle cell disease developed myelodysplastic syndrome, a cancer-like disease of the bone marrow.

Bluebird told STAT’s Adam Feuerstein, who is on site at ASH, that it may have been triggered by the chemotherapy regimen used to condition patients before they receive the gene therapy.