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

Beat Childhood Cancer Foundation Multi-Trial Grant

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 #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

EIN: 84-4322722

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