The US Food and Drug Administration (FDA) is weighing the need for regulatory action for gene therapy Bluebird Bio’s BLUE to treat a rare neurological disorder, the company said Wednesday, as the agency investigates additional reports of blood cancer cases linked to the drug’s use.
Bluebird’s Skysona was approved by the FDA in 2022 for the treatment of cerebral adrenoleukodystrophy (CALD) and cost $3 million at the time of launch.
CALD, which affects approximately one in 20,000 to 50,000 people worldwide, typically occurs in boys between the ages of 3 and 12.
Skysona’s prescribing information already includes a warning about blood cancers, including leukemia and myelodysplastic syndromes, a group of cancers that occur when the bone marrow produces immature blood cells instead of healthy cells.
Patients should consider alternative therapies such as a stem cell transplant from a suitable donor before deciding to treat a child with Skysona, the FDA said, adding that it is studying the “known risks” associated with gene therapy, which include Hospitalizations and death include.
“The agency’s FOCUS today was not prompted by new cases of malignancy or other security updates,” Bluebird told Reuters. The risk of blood cancer is known, it said.
Eric Schmidt, an analyst at Cantor Fitzgerald, said the FDA’s investigation was “not a surprising development given the recent article in the New England Journal of Medicine that highlighted this risk.”
Patients with this disease have a very poor prognosis, so the FDA will likely consider the risk or benefit of Skysona treatment to be favorable, Schmidt said.
Massachusetts-based Bluebird, which has expressed doubts about its ability to continue as a going concern in recent years, also wants to bring its other gene therapies to market. In September, the company laid off about 25 percent of its workforce in a second major restructuring (link) in two years to cut costs.
* **What are the key ethical considerations surrounding gene therapies that aim to treat rare diseases with potentially high costs and unknown long-term effects?**
## Navigating the Risks and Rewards of Gene Therapy: An Interview
Welcome to World Today News’ insightful interview series. Today, we delve into the complex world of gene therapy, spurred by recent news regarding the FDA investigation into Bluebird Bio’s Skysona. Joining us are:
* **Dr. Emily Chen:** A leading expert in pediatric neurology and gene therapy research.
* **Ms. Sarah Jones:** A patient advocate representing families impacted by cerebral adrenoleukodystrophy (CALD).
**I. Understanding CALD and the Promise of Gene Therapy**
**Interviewer:** Dr. Chen, could you provide our viewers with a better understanding of CALD? What are its devastating effects, and what makes it such a challenging condition to treat?
**Interviewer:** Ms. Jones, could you share your experience with CALD? What hope did treatments like Skysona bring to families facing this devastating diagnosis?
**II. The Skysona Controversy: Weighing Risk and Reward**
**Interviewer:** Dr. Chen, Skysona was hailed as a groundbreaking treatment for CALD when it was approved in 2022. However, the FDA is now investigating reports of blood cancerlinked to its use. How do we reconcile this potential risk with the desperate need for effective CALD treatments?
**Interviewer:** Ms. Jones, how do families struggling with CALD grapple with the news of these potential risks? What factors do they consider when making difficult treatment decisions for themselves or their loved ones?
**Interviewer:** Ms. Jones, some argue that the high cost of Skysona, at $3 million per treatment, creates an additional layer of burden for families. What are your thoughts on the financial accessibility of such innovative therapies?
**III. Moving Forward: The Future of Gene Therapy**
**Interviewer:** Dr. Chen, what are the implications of the FDA’s investigation for the future of gene therapies? How can researchers and regulators ensure that the potential benefits of these treatments outweigh the potential risks?
**Interviewer:** Ms. Jones, what message do you have for policymakers and pharmaceutical companies regarding the development and accessibility of gene therapies for rare diseases?
**Concluding Remarks**
The conversation surrounding gene therapy is complex and multifaceted. While therapies like Skysona hold immense promise for treating devastating diseases, it’s crucial to ensure patient safety and accessibility. Open dialogue and collaboration between researchers, regulators, patients, and families are essential to navigate the exciting but challenging landscape of gene therapy.
