Overview

• In PNAS research, combining inhibitors of PRDX6 and GSTP1 in mouse models caused some neuroblastoma cells to die while others differentiated into healthy neurons, reducing tumor growth
• Retinoic acid differentiation therapy fails in nearly half of patients due to nonresponse or resistance, driving the search for new treatment options
• One of the enzyme inhibitors already holds orphan drug designation from the US FDA for an unrelated adult condition, which could accelerate its repurposing for pediatric trials
• Harsh current treatments often leave survivors with lifelong cognitive impairments, underscoring the need for less damaging therapeutic strategies
• Researchers at Karolinska Institutet and Lund University intend to launch clinical trials to assess the safety and efficacy of the dual-inhibitor approach in children