Diet and medication combo interrupts growth of aggressive childhood neuroblastoma tumors, study finds (Arginine removal)

Medical Xpress / Children's Hospital of Philadelphia / Nature ^ | Sept. 25, 2025 | Sarah Cherkaoui et al

[ConservativeMind]

Researchers found that combining a specialized diet with an approved medication interrupts the growth of high-risk neuroblastoma, a deadly pediatric cancer, by reprogramming tumor behavior.

Neuroblastoma originates from primitive cells meant to form nerve tissues but that remain "undifferentiated," indicating cancer cells that haven't specialized, often suggesting a more aggressive and unfavorable prognosis. These tumors rely on a steady supply of chemicals called polyamines that are essential for rapid cell growth and tumor progression.

A medicine called difluoromethylornithine (DFMO) was approved by the Food and Drug Administration (FDA) to treat children with high-risk neuroblastoma, as DFMO blocks polyamine production. However, researchers sought to improve the effectiveness of the drug by using it at high doses and combining it with a diet that is depleted of the nutrients used by the body to make polyamines (arginine). This two-step approach was anticipated to lower polyamines substantially more than low dose DFMO alone.

"Our findings show that this treatment reduced polyamines in tumors to roughly 10% of their usual levels. This reduction greatly slowed tumor growth, and in many cases, completely eliminated the tumors," said Michael D. Hogarty, MD. "Notably, the treatment altered the way the tumor cells make proteins, making it harder for them to grow."

Hogarty and his team used a preclinical model to mimic MYCN-driven neuroblastoma, directly addressing the strong association between extra MYCN gene copies and aggressive neuroblastoma with poor prognosis.

Animal models with tumors were divided into groups: one fed a normal diet and the other lacking amino acids for polyamine production. Each group either received DFMO in their drinking water or did not. The special diet or DFMO alone partially lowered polyamines and extended survival, but the combination had the most significant impact on tumors due to the profound polyamine depletion it caused.

[ConservativeMind]

Difluoromethylornithine is available today and is used for this purpose, but combining that same drug with a diet missing arginine completely eliminated tumors, in many cases.

The authors believe this could work with other cancers with MYC gene activation.

[ConservativeMind]

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