A discovery from Australian researchers could lead to better treatment for children with neuroblastoma, a cancer that currently claims 9 out of 10 young patients who experience recurrence. The team at the Garvan Institute of Medical Research in Sydney, Australia, found a drug combination that can bypass the cellular defenses these tumors develop that lead to relapse. Read more: https://medicalxpress.com/news/2025-11-drug-combination-sidesteps-resistance-aggressive.html

Cancer treatment balances on a razor’s edge. On one side, physicians must use enough of a drug to kill tumor cells effectively. On the other side, a high drug concentration can be harmful to a patient’s normal cells. In children, the difference between the two, called the therapeutic index, is small.  Read more: https://www.stjude.org/research/progress/2025/finding-balance-between-efficacy-and-toxicity-in-pediatric-cancer-treatment.html

A research team from the Children's Hospital of Chongqing Medical University, China, reported in Pediatric Discovery  that thiostrepton can significantly suppress rhabdomyosarcoma progression by targeting the PI3K-AKT signaling pathway. Read more: https://www.news-medical.net/news/20251027/Study-finds-evidence-for-repurposing-thiostrepton-as-an-anti-rhabdomyosarcoma-drug.aspx

The FDA granted rare pediatric disease designation to iopofosine-131 for treating relapsed or refractory pediatric high-grade glioma.   Read more: https://www.curetoday.com/view/fda-grants-rare-pediatric-designation-for-iopofosine-131-in-glioma-subset

The FDA has granted orphan drug designation (ODD) to AMXT 1501 in combination with difluoromethylornithine (Iwilfin; DFMO) for the treatment of patients with neuroblastoma. Read more: https://www.targetedonc.com/view/amxt-1501-and-dfmo-combination-achieve-fda-orphan-drug-designation

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