Misfortune strikes a Bluebird patient, highlighting the safety risks that come with gene therapy

People get excited about gene therapy for its potential to cure some pretty horrible diseases, but the safety risks that patients take on with the preparation and administration of these treatments are often overlooked.

On Saturday afternoon, Bluebird Bio reported for the first time the diagnosis of myelodysplastic syndrome (MDS), a cancer-like disease of the bone marrow, in a single sickle cell disease patient who had undergone treatment with the biotech’s Lentiglobin gene therapy. Bluebird’s Chief Scientific Officer David Davidson tells me the case of MDS was likely caused by the chemotherapy regimen used to “condition” the patient’s bone marrow before the modified stem cells that comprise the sickle cell gene therapy are infused. More details and my take here.