Combination of Special Diet and Medication Halts Growth of High-Risk Childhood Neuroblastoma Tumors

Researchers at Children’s Hospital of Philadelphia (CHOP) have discovered that combining a targeted dietary approach with a proven medication significantly impedes the progression of aggressive neuroblastoma in children. Neuroblastoma, a deadly pediatric cancer, originates from primitive nerve cells that remain undifferentiated, contributing to its rapid growth and poor prognosis. These tumors rely heavily on polyamines—chemicals vital for cell proliferation and tumor development.

The medication difluoromethylornithine (DFMO), approved by the FDA, inhibits polyamine synthesis. The study aimed to enhance its effectiveness by pairing high-dose DFMO with a diet specifically depleted of arginine, a nutrient essential for polyamine production. This combination drastically reduced tumor polyamine levels—by approximately 90%—leading to significant tumor suppression and, in some cases, complete tumor elimination.

Lead researcher Dr. Michael D. Hogarty explained that this dual therapy not only slows tumor growth but also prompts tumor cells to differentiate, making them less aggressive and easier to target. The team employed preclinical models mimicking MYCN-driven neuroblastoma, a subtype linked to a worse prognosis due to extra copies of the MYCN gene. They observed that while either high-dose DFMO or the special diet alone produced modest benefits, their combination yielded the most profound effects.

The promising results suggest that this strategy could be translated into clinical trials to test safety and effectiveness in children. Ultimately, the goal is to augment existing treatments and improve outcomes by targeting the tumor's metabolic dependencies. Since polyamines are involved in various cancer types with frequent MYC gene activation, this approach holds potential for broader cancer therapy applications.

Published in Nature, this research opens new avenues in pediatric cancer treatment by leveraging nutritional and pharmacological synergy to reprogram tumor behavior and combat neuroblastoma more effectively. Future studies are planned to confirm these findings and explore their potential for wider clinical use.