LWF Grants $700,000 to Help Launch Multiple New Trials
In 2025–2026, the Little Warrior Foundation (LWF) is proud to announce a $700,000 grant to the Beat Childhood Cancer Foundation to support the launch of three new clinical trials, including two focused on DFMO with the Beat Childhood Cancer Research Consortium (BCCRC.)
DFMO: From Grit to Groundbreaking
The story of DFMO (difluoromethylornithine) in childhood cancer is one of pure perseverance — a group of parents and physicians who refused to accept “no” for an answer. What started as an uphill fight to manufacture and supply the drug became a movement that led to FDA approval of DFMO (IWILFIN) for high-risk neuroblastoma.
Their drive and resilience inspired LWF from day one. That same “whatever it takes” spirit — the willingness to do the hard work for a chance at more tomorrows — is exactly what fuels our mission.
A New Chapter: DFMO Moves Into Ewing Sarcoma and Osteosarcoma
DFMO, now FDA-approved as IWILFIN, marks a rare milestone in pediatric oncology — one of only a small number of drugs ever approved for the treatment of a childhood cancer subtype. Unlike most oncology drugs, which are first developed for adult cancers, IWILFIN was designed and studied specifically to prevent relapse in children with high-risk neuroblastoma.
And now, DFMO’s story is expanding. Thanks to the groundbreaking preclinical work from Dr. David Loeb and Dr. Rachel Offenbacher (Montefiore Einstein Comprehensive Cancer Center / Johns Hopkins), DFMO is showing strong preclinical activity in Ewing sarcoma and Osteosarcoma models.
Their research revealed that DFMO isn’t just slowing cancer growth — it’s attacking the disease at its metabolic core. DFMO acts as a metabolic inhibitor, blocking polyamine biosynthesis, a key pathway that fuels EWSR1::FLI1, the fusion oncogene driving Ewing sarcoma. By cutting off this pathway, DFMO helps stop tumor cell division, spread, and survival — even triggering ferroptosis, a form of cell death in cancer cells that refuse to quit.
Dig deeper into this breakthrough research.
Check out Dr. Loeb’s Episode of Ewing’s U to learn more!
Collaboration Creates Momentum
LWF helped bring together two powerhouses in pediatric cancer research: Dr. Giselle Sholler, Chair of BCCRC and one of the world’s most experienced DFMO clinicians, and Dr. David Loeb, whose lab has pioneered DFMO’s expansion into sarcomas.
The result? Two new clinical trials.
Trial #1 — BCC020 (NCT06465199):
Now open for enrollment at Penn State Health Children’s Hospital and available to hospitals nationwide within the BCCRC network, this Phase I/II trial is testing DFMO in combination with AMXT 1501, a cutting-edge polyamine transport inhibitor.
DFMO stops the tumor from making polyamines.
AMXT 1501 stops the tumor from importing them.
Together, they create a one-two punch — shutting down both the internal and external lifelines the tumor needs to survive. This trial is open for warriors with relapsed Ewing sarcoma, as well as other sarcomas, CNS tumors, and neuroblastoma.
Trial #2 — BCC023:
Currently pending FDA approval and expected to open in late 2025 / early 2026, this multi-institutional study will feature four arms — two for Ewing sarcoma and two for osteosarcoma — testing DFMO both as maintenance therapy and in combination with chemotherapy in relapsed/refractory settings. More details to come as BCCRC works with the FDA to open this trial to create the best possible opportunity to impact safety, response, and survival.
The Road Ahead
We know progress never moves fast enough when a child’s life hangs in the balance. But with each new collaboration, each new trial, and each new dollar put to work, we’re moving the needle — together.
To every Little Warrior family, donor, and supporter, thank you. Your belief powers these breakthroughs. You make trials like these possible.
We fight because they can’t. And we’ll keep going until childhood cancer is a story with a different ending.
Swords Up — Always.
SPECIAL THANKS: We’re so grateful to our friends at the Battle Brynn Foundation for contributing $10,000 to the Little Warrior Foundation to help make this grant and these trials possible!
Brynn’s mission was clear: “I never want another kid to go through what I am going through. I want to make it better.”
She bravely participated in research, knowing it wouldn’t help her but could save lives in the future. Now, we carry that mission forward—together. This is what impact looks like. This is what community looks like. This is what Brynn’s legacy is all about.