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Dual Enzyme Inhibitors Convert Neuroblastoma Tumors into Healthy Neurons in Mice

Plans for pediatric trials are under way following the mouse model breakthrough.

Overview

  • A PNAS study by Karolinska Institutet and Lund University shows that inhibiting PRDX6 and GSTP1 causes some tumor cells to die and others to differentiate into active neurons in mice.
  • Current retinoic acid differentiation therapy fails in about half of patients, highlighting the need for alternative strategies.
  • High PRDX6 and GSTP1 levels in neuroblastoma correlate with poorer survival, identifying these enzymes as promising treatment targets.
  • One of the inhibitors holds orphan drug designation from the US FDA, potentially speeding its development for pediatric use.
  • Researchers aim to launch clinical trials to assess the treatment’s safety and efficacy in children with neuroblastoma.