STAT+: New tumor cases show common gene therapy tool can come with risk
New cases show AAV gene therapies like Zolgensma carry tumor risks, raising key concerns for SMA patients and gene therapy researchers in general.
Novartis has notified of seven previously unrecorded instances where tumors emerged in children treated with its spinal muscular atrophy (SMA) gene therapy. The revelation, made public on Thursday by a patient advocacy group and subsequently shared on a Facebook page, has spurred concerns among families, medical professionals, and patient support organizations.
Earlier in the week, Novartis had disclosed the information to neurologists. The advocacy group SMA Europe announced that regulatory agencies are now investigating the cases.
In response to the disclosure, Novartis issued its own statement. According to the company, since the approval of its treatment, Zolgensma, in 2019, it has received nine reports of tumors in patients who had previously undergone the therapy. Out of these nine cases, only two have shown indications that the tumors might be associated with the treatment itself.
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- Novartis Confirms Nine Cases of Tumors in Children Treated with Gene Therapy Zolgensma genengnews.com
- STAT+: Pharmalittle: We’re reading about a Novartis gene therapy, faster U.K. drug reviews, and more statnews.com
- Novartis reveals cancer cases possibly linked to gene therapy Zolgensma biopharmadive.com
