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

Momentum is building for PROTAC treatments that eliminate disease-causing proteins, including those responsible for difficult-to-treat...

The Leukemia & Lymphoma Society (LLS) announced today the first pediatric patient has received treatment in a new subtrial of its...

Asparaginase (ASNase) remains a cornerstone of chemo regimens for ALL. But there’s a quiet threat that’s often missed: hypersensitivity...

The FDA has granted Rare Pediatric Disease Designation for NEO100 in the treatment of pediatric-type diffuse high-grade gliomas, a type...

Our SIOPEL-6 study showed that sodium thiosulfate (Pedmarqsi) almost halves the percentage of children and teenagers who lose hearing as...

“Patient response to revumenib has been good in other clinical trials,” explains Cuglievan. “But it’s also been short-lived when used...