Researchers in Australia have discovered a drug that may help patients with relapsed neuroblastoma— a fatal childhood cancer—overcome resistance to treatment.
This finding promises a boost for a disease that is the most common solid tumor in children outside the brain and which still kills nine out of ten young patients who relapse, say officials at Xinhua news agency.
According to the Garvan Institute of Medical Research in Sydney, the combination therapy can circumvent the cellular defenses that normally cause the tumors to rebound.
The team demonstrated that the already-approved lymphoma medication “romidepsin” provokes death of neuroblastoma cells through alternative mechanisms, sidestepping the blocked pathways that render conventional chemotherapy ineffective in children.
Their experiments showed that standard chemotherapy agents usually depend on a JNK‑driven “switch” to trigger cell death. In tumors that have returned, this switch often fails, making the drugs useless.
In animal studies, pairing romidepsin with usual chemotherapy halted tumor growth by engaging other cell‑death routes, bypassing the defective JNK pathway that is common in resistant cases.
The combination narrowed tumor sizes, lengthened survival times, and allowed lower doses of the chemotherapeutic agents—potentially reducing the toll on young patients, the researchers reported in Science Advances.
David Croucher, an associate professor at the Garvan Institute, said, “Finding a way to overcome the resistant state of relapsed high‑risk neuroblastomas has been a major goal for my lab.”
He added, “These tumours can be highly resistant to chemotherapy – and the statistics once patients get to that point are devastating for families.”
Since romidepsin is already approved for other cancers and has been tested for safety in children, its repurposing for neuroblastoma could speed up clinical development.
Nonetheless, Croucher cautioned that translating the results into patient care will require additional studies and clinical trials to confirm the combination’s safety and effectiveness.
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