Skysona, a personalized gene therapy approved by the FDA for cerebral adrenoleukodystrophy (CALD), has brought significant changes to the field of gene therapy.
Recent data has raised concerns about the therapy's association with cancer, particularly in young boys. A study published in The New England Journal of Medicine reported seven cases of blood malignancies among 67 boys treated with Skysona, prompting questions about the balance between its benefits and risks.
Despite these concerns, the therapy has shown promising results in terms of neurological function scores, with most patients experiencing stable or improved function. However, families considering Skysona must carefully weigh the potential risks and benefits.
The therapy's safety profile has been a topic of discussion since its development, and the recent publication provides a more comprehensive assessment of the risks associated with Skysona. The therapy utilizes an engineered virus constructed from the patient's own stem cells, and the cancers observed in some patients are believed to be mediated by this virus. This raises concerns about the long-term safety of gene therapies, especially in vulnerable populations like children.
The recent findings regarding Skysona's safety profile could have broader implications for the acceptance and implementation of gene therapies in clinical practice. Bluebird bio, the developer of Skysona, also offers other gene therapies for different conditions, and the safety profiles of these therapies may be influenced by the specific lentivirus used.
The ongoing scrutiny of Skysona's safety profile may impact the company's ability to attract investment and support for its gene therapy programs. In conclusion, while Skysona represents a significant advancement in CALD treatment, the recent data on cancer risks highlights the need for careful evaluation of its use, considering the potential benefits and risks of gene therapies.