New Metabolic Target Offers Hope for Treating Rare and Aggressive Childhood Cancer

A groundbreaking study has uncovered a vital metabolic vulnerability in Malignant Peripheral Nerve Sheath Tumor (MPNST), a rare and highly aggressive childhood cancer predominantly affecting teenagers and young adults. This form of cancer is characterized by rapid growth and a tendency to spread, with limited effective treatment options, especially once it metastasizes. Currently, there are no targeted therapies available for metastatic MPNST, making new treatment approaches critically important.

Researchers from the University of Iowa, led by Dr. Eric Taylor and Dr. Rebecca Dodd, employed advanced gene editing techniques to develop models that accurately replicate the genetic mutations found in patients. Using these models, they utilized cutting-edge genomic and metabolomic analyses to investigate the cancer’s fuel sources. Their findings, published in Science Advances, revealed that MPNST cells depend heavily on a specific metabolic pathway to survive oxidative stress and sustain tumor growth.

The study identified the Pentose Phosphate Pathway (PPP) as a key process in these tumor cells. This pathway metabolizes sugars to generate molecules essential for combatting oxidative damage. When researchers inhibited the PPP, the tumors grew more slowly and became more susceptible to chemotherapy, indicating a promising therapeutic target.

This discovery is significant because it links the PPP pathway directly to MPNST tumor progression for the first time, opening new avenues for treatment development. The collaborative research combined expertise in cancer biology and metabolism, led by graduate student Gavin McGivney, now a postdoctoral researcher at the University of Chicago.

Dodd emphasizes that targeting the PPP could lead to novel strategies to slow tumor growth and improve patient outcomes in this difficult-to-treat cancer. The findings suggest that future therapies might incorporate metabolism-based approaches to enhance current treatments. This research marks a vital step toward more effective interventions for children and young adults affected by MPNST.

Source: https://medicalxpress.com/news/2025-08-metabolic-pathway-rare-aggressive-childhood.html