New Treatment Approach Transforms Neuroblastoma Cells into Healthy Neurons in Mice

Researchers from Karolinska Institutet and Lund University in Sweden have developed a promising new treatment for neuroblastoma, a severe form of cancer affecting children. By utilizing a combination of two antioxidant enzyme inhibitors, they have successfully managed to convert neuroblastoma cells in mice into functioning nerve cells.

The findings of this research were published in the Proceedings of the National Academy of Sciences. Neuroblastoma is recognized as the most common cause of cancer-related fatalities in young children and primarily impacts the nervous system. While some patients exhibit a favorable prognosis, those with metastatic forms of the disease often face grim outcomes, even with the latest treatment modalities such as surgery, radiation, chemotherapy, and immunotherapy.

Marie Arsenian Henriksson, a professor at the Department of Microbiology, Tumor and Cell Biology at Karolinska Institutet, emphasized the urgent need for innovative treatment options, stating that survivors frequently endure lasting cognitive impairments due to the aggressive nature of conventional therapies.

One existing treatment approach, known as differentiation therapy, seeks to convert cancerous cells into healthier, more mature cells. However, the current standard treatment using retinoic acid has limitations, as many patients either do not respond to it or develop resistance over time.

In collaboration with Lund University researchers, Henriksson's team has identified that inhibiting two specific enzymes, PRDX6 and GSTP1, presents a viable alternative to retinoic acid for treating neuroblastoma. The aggressive metabolism of neuroblastoma cells induces significant oxidative stress, making these tumors reliant on antioxidant enzymes such as PRDX6 and GSTP1 to survive. Elevated levels of these enzymes correlate with poorer prognoses.

The researchers observed that by blocking these enzymes in both cell cultures and mouse models, they could induce cell death in some tumor cells while others transitioned into active, healthy neurons, thereby hindering the growth of the tumor.

Looking ahead, the next phase involves conducting clinical trials to assess the safety and efficacy of this treatment in children. Notably, one of the inhibitors has already been granted orphan drug designation by the US Food and Drug Administration for a different condition in adults, heightening its potential as a therapeutic candidate.

For further information, refer to the study by Liaño-Pons et al. in the Proceedings of the National Academy of Sciences.