Novartis Confirms Nine Cases of Tumors in Children Treated with Gene Therapy Zolgensma
"Two cases to date have shown evidence of vector integration into the DNA of the patients. In both cases, the patients underwent treatment and continue to be monitored,” Novartis said in a statement. The post Novartis Confirms Nine Cases of Tumors in Children Treated with Gene Therapy Zolgensma appeared first on GEN - Genetic Engineering and Biotechnology News .
Novartis has confirmed nine cases in which tumors developed in children who received its gene therapy Zolgensma for spinal muscular atrophy. Two of the cases have shown evidence of the virus used in the therapy integrating into the patients' DNA. Novartis stated that patient safety is its priority and that it notified health authorities in a timely manner.
Zolgensma, an adeno-associated virus vector-based therapy, is used to treat children under two with SMA caused by genetic mutations. The therapy replaces the function of a missing or nonworking SMN1 gene in patients' cells. While the risk of tumors due to vector integration is acknowledged, its clinical relevance is unknown. Novartis has been urged by patient advocacy groups to provide more transparent and timely information about these tumor cases.
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