Prime Highlights

• Australian researchers have identified a drug combination using romidepsinthat can bypass treatment resistance in relapsed neuroblastoma, offering new hope for affected children.
• The therapy could potentially reduce chemotherapy intensity and improve survival outcomes for a cancer that currently kills 90% of children after relapse.

Key Facts

• In animal studies, combining romidepsin with standard chemotherapy reduced tumour growth, extended survival, and worked without relying on the blocked JNK pathway.
• Romidepsin is already approved for lymphomaand has been tested for safety in children, potentially speeding up its pathway to clinical trials for neuroblastoma.

Background

Researchers in Australia have discovered a promising drug combination that could help treat relapsed neuroblastoma, a deadly childhood cancer. The breakthrough could improve survival rates for the most common solid tumour in children outside the brain, which currently kills nine out of 10 children after relapse, according to the Garvan Institute of Medical Research.

The team found that romidepsin, a drug already approved for lymphoma, can trigger neuroblastoma cell death through alternative pathways. Standard chemotherapy relies on the JNK pathway to kill cancer cells, but in relapsed tumours, this pathway often stops working, rendering treatments ineffective.

In animal studies, combining romidepsin with standard chemotherapy successfully bypassed the blocked JNK pathway. The combination reduced tumour growth, extended survival, and allowed for lower chemotherapy doses, which could reduce side effects for young patients. The findings were published in Science Advances.

David Croucher, Associate Professor at the Garvan Institute, stated, “My lab has been focused on identifying strategies to tackle the resistant nature of relapsed high-risk neuroblastomas.” He noted that these tumours are highly resistant and that current statistics for relapsed patients are devastating for families.

Researchers have already tested Romidepsin for safety in children, and it is approved for treating other cancers, which could help develop it faster as a treatment for neuroblastoma. However, they warn that clinical trials are needed to prove the drug combination is safe and effective in children.

If this approach works, it could give children with relapsed neuroblastoma a new chance, fight the cancer more effectively, and let them have less intense chemotherapy.