Beat Childhood Cancer Foundation - Multi-Trial Grant (DFMO Studies)

Beat Childhood Cancer Foundation Multi-Trial Grant

Funder: Little Warrior Foundation Inc (WI, US)
Amount: $700,000
Status: Awarded (announced October 2025)
Period: 2025-2026
Recipient: Beat Childhood Cancer Foundation (BCCF) / Beat Childhood Cancer Research Consortium (BCCRC)

Focus Areas

Supporting launch of three new clinical trials, with two focused on DFMO (difluoromethylornithine) for pediatric sarcomas:

Trial Details

#### Trial #1: BCC020 (NCT06465199)

Status: Now open for enrollment

Sites: Penn State Health Children's Hospital; expanding nationwide through BCCRC network

Phase: Phase I/II

Patient Population: Relapsed Ewing sarcoma, other sarcomas, CNS tumors, neuroblastoma

Treatment: DFMO in combination with AMXT 1501 (polyamine transport inhibitor)

Mechanism: Dual blockade - DFMO stops tumor polyamine production; AMXT 1501 stops tumor polyamine import

Lead Investigator: Dr. Giselle Sholler (Chair of BCCRC)

#### Trial #2: BCC023

Status: Pending FDA approval; expected to open late 2025/early 2026

Phase: Multi-institutional study

Design: Four arms (two for Ewing sarcoma, two for osteosarcoma)

Patient Population: Relapsed/refractory settings

Treatments: DFMO as maintenance therapy and in combination with chemotherapy

Co-Investigator: Dr. David Loeb (preclinical work, Johns Hopkins/Montefiore Einstein)

Scientific Rationale

DFMO Background:
• FDA-approved as IWILFIN for high-risk neuroblastoma
• Rare example of pediatric oncology drug developed specifically for childhood cancer (not adapted from adult use)
• Metabolic inhibitor blocking polyamine biosynthesis

Expansion to Sarcomas:

Strong preclinical activity in Ewing sarcoma and osteosarcoma models

Attacks disease at metabolic core

Blocks polyamine pathway fueling EWSR1::FLI1 fusion oncogene

Inhibits tumor cell division, spread, survival

Triggers ferroptosis (form of cell death)

Contact Information

Little Warrior Foundation PO Box 2124 Brookfield, WI 53008-2124 info@littlewarrior.org EIN: 84-4322722

Special Recognition: Battle Brynn Foundation contributed $10,000 to support these trials