US authorities investigate potential link between gene therapy and childhood cancer
US investigation into potential link between Zolgensma drug and tumors in children
Translated by Qiyas from الجريدة — Original source
The U.S. Food and Drug Administration (FDA) is investigating reports of children developing cancer after receiving gene therapy with Zolgensma, a drug used to treat spinal muscular atrophy (SMA), a rare genetic disorder that can lead to death before a child reaches the age of two.
The agency stated that it is reviewing reports of tumor development in several children who underwent treatment with the drug, which is produced by the Swiss company Novartis. The European Spinal Muscular Atrophy organization (SMA Europe), citing a letter from the company, noted that more than 6,000 patients have received the drug since its approval, and that the company has recorded nine reports of tumors in patients who underwent the treatment.
A few years ago, Zolgensma was the most expensive drug in the world, with a price of approximately two million euros. It received approval for use in the United States in 2019 and in Europe in 2020 for children under the age of two with spinal muscular atrophy.
The treatment involves administering a single dose via intravenous infusion, aiming to compensate for the missing or non-functional SMN1 gene, which is responsible for the disease. The process uses modified viruses known as adeno-associated viruses (AAV) to deliver a healthy copy of the gene to the body’s cells.
Analyses showed that the viral DNA used in the treatment was found within tumor tissues in two cases. According to the FDA, the tumors appeared in the central and peripheral nervous systems, as well as in the spinal column and kidneys.
SMA Europe, citing Novartis, stated that the two patients in question received the treatment and are still undergoing medical follow-up. The company confirmed that it highlights the potential risk of tumor development in the drug’s medical information.
The use of adeno-associated viruses is not limited to Zolgensma; they are also used in other gene therapies to deliver genes. The FDA pointed to two scientific reports recently published regarding cancer cases following similar gene therapies.
The agency stated that it is closely monitoring the risk of tumor development, including malignant tumors, among patients receiving gene therapies based on these vectors, and is examining whether regulatory actions are necessary. However, it emphasized that the benefits of Zolgensma treatment continue to outweigh its potential risks.
