Bluebird Bio’s gene therapy, known as Skysona, has been linked to the development of blood cancers in seven out of 67 children who received the treatment for a severe neurological disorder in clinical trials. This news comes after concerns were raised in June 2022 when three cases of cancer were reported, prompting the Food and Drug Administration to hold a hearing before approving the therapy.
Since then, four additional patients have been diagnosed with blood cancers, and sadly, one patient has passed away from complications of cancer treatment. Researchers anticipate that more children may develop cancer in the future, and they are closely monitoring all recipients of the gene therapy with regular blood tests to catch any potential issues early on.
Christine Duncan, a senior physician at Boston Children’s Hospital and the lead author of the new study, expressed the sentiment shared by many in the medical community, stating that everyone wishes there were no more cases of cancer associated with the treatment. However, she acknowledges that this may not be a practical expectation.
The data surrounding the link between Bluebird Bio’s gene therapy and blood cancers is alarming and raises concerns about the safety of the treatment. This information is crucial for both healthcare providers and patients to be aware of when considering this therapy for neurological disorders.
As the investigation and monitoring of patients continue, it will be essential for healthcare professionals to stay informed about any new developments or findings related to the gene therapy. Additionally, patients and their families should have open and honest discussions with their healthcare providers about the risks and benefits of this treatment option.
While gene therapies hold great promise for treating a variety of diseases and disorders, it is imperative that thorough research and monitoring are conducted to ensure the safety and efficacy of these innovative treatments. The findings related to Bluebird Bio’s gene therapy serve as a reminder of the importance of continued vigilance in the field of gene therapy research and development.