US Investigation into Potential Link Between Gene Therapy and Pediatric Tumors
The US Food and Drug Administration is investigating medical reports linking the gene therapy Zolgensma, used for spinal muscular atrophy, to cancer cases in children.
The US Food and Drug Administration (FDA) has launched an investigation into medical reports concerning a potential link between a gene therapy and the development of cancer in children. The investigation focuses on the drug Zolgensma, which is used to treat spinal muscular atrophy.
Novartis, the Swiss manufacturer of the drug, reported nine cases of tumors. Since the treatment received regulatory approval, more than 6,000 patients have received the therapy.
Medical analyses identified the DNA of the virus used in the treatment within two of the reported cases. The tumors were discovered in the patients' nervous systems, spines, and kidneys.
The manufacturer acknowledged that current medical information indicates a potential risk of developing these tumors. Meanwhile, the FDA continues to monitor risks associated with similar gene therapies.
Despite these findings, the FDA emphasized that the therapeutic benefits of the drug continue to outweigh its potential risks.