Recent data released by Bluebird Bio Inc. has shed light on a concerning trend: seven out of 67 children who received their gene therapy, BLUE, for a severe neurological disorder in clinical trials have developed blood cancers. This represents a significant increase from June 2022, when three cancer cases prompted the FDA to convene an advisory panel before approving the therapy, now marketed as Skysona. Sadly, one child succumbed to complications from cancer treatment.
The STAT News report highlighted the continued concerns surrounding the therapy, with researchers anticipating more cancer cases in the future. They are actively monitoring the patients through regular blood tests.
The FDA’s approval of Skysona in September 2022 for treating early, active cerebral adrenoleukodystrophy (CALD) in boys aged 4-17 years was met with mixed reactions. Further, the FDA included a black box warning in the label for Bluebird Bio’s Lyfgenia (lovotibeglogene autotemcel (lovo-cel)), a gene therapy for sickle cell disease, acknowledging the potential risk of hematologic malignancy (blood cancer) in treated patients.
These recent developments come amidst a period of restructuring for Bluebird Bio. In April 2022, the company laid off approximately 30% of its workforce to extend its cash runway and save $160 million over the next two years. This strategic move reflects the company’s commitment to financial sustainability and further emphasizes the importance of addressing the safety concerns associated with its gene therapies.
The emergence of these blood cancer cases has undoubtedly raised concerns about the long-term safety of Bluebird Bio’s gene therapies. While the company continues to research and develop these groundbreaking treatments, the FDA and researchers will need to closely monitor the situation and ensure patient safety remains paramount.
