# Gallium maltolate for the treatment of difficult-to-treat high-grade pediatric brain tumors

> **NIH CA R01** · MEDICAL COLLEGE OF WISCONSIN · 2026 · $632,314

## Abstract

PROJECT ABSTRACT/SUMMARY
Neoplasms of the central nervous system (CNS) are the most frequently encountered solid tumors of childhood
and remain one of the top causes of death in children. Pediatric high-grade gliomas (pHGGs) and atypical
teratoid rhabdoid tumor (ATRT) are particularly aggressive pediatric CNS (pedCNS) tumors associated with
poor outcome. Therapy consists of extremely burdensome multi-modal treatment protocols with toxic profiles
that cause patients to suffer from detrimental effects, which significantly impact the quality of life during their
already limited lifespan. New therapeutic strategies are badly needed to increase the survival benefit and
quality of life of patients with pHGG and ATRT. To address this need, we performed initial studies that
demonstrated that primary CNS cancers display dysregulated iron homeostasis and that gallium maltolate
(GaM), an iron mimetic metallocompound, inhibits the growth of pHGG and ATRT cells in vitro and in an
orthotopic rat model, extending overall survival.
 Cancer iron metabolism is an attractive target for therapeutical intervention. Iron plays a vital role in the
pathobiology of many cancers, including brain cancer. Gallium acts as an iron mimetic, enabling it to hijack
common iron trafficking pathways to enter cancer cells. However, unlike iron, gallium cannot take part in
cellular redox reactions, thus disrupting critical iron-dependent processes and resulting in cell death. Our group
has demonstrated the effectiveness of gallium maltolate (GaM), a newer generation compound with high oral
bioavailability and therapeutic index, both in vitro and in vivo. In animal studies of adult glioblastoma, the most
aggressive type of primary brain tumors, we demonstrated a slower tumor growth rate, a doubling of survival,
and an improved quality of life with treatment. Preliminary studies in pHGGs and ATRTs suggest similar
benefits. Despite this promising initial step, questions regarding the efficacy of GaM remain

## Key facts

- **NIH application ID:** 11296733
- **Project number:** 1R01CA299189-01A1
- **Recipient organization:** MEDICAL COLLEGE OF WISCONSIN
- **Principal Investigator:** Mona M Al-Gizawiy
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** CA
- **Fiscal year:** 2026
- **Award amount:** $632,314
- **Award type:** 1
- **Project period:** 2026-04-01T00:00:00 → 2031-03-31T00:00:00

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11296733

## Citation

> US National Institutes of Health, RePORTER application 11296733, Gallium maltolate for the treatment of difficult-to-treat high-grade pediatric brain tumors (1R01CA299189-01A1). Retrieved via AI Analytics 2026-07-06 from https://api.ai-analytics.org/grant/nih/11296733. Licensed CC0.

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