New Clinical Trial Aims to Improve Outcomes for Pediatric Bone Cancers

The study will evaluate the drug eflornithine (DFMO) in treating Ewing sarcoma and osteosarcoma.

Apr. 16, 2026 at 3:02am

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A ghostly X-ray image revealing the internal structure of a human bone with a faint outline of a cancerous tumor, conceptually representing the new clinical trial's focus on improving treatment options for pediatric bone cancers.A new clinical trial aims to evaluate a promising drug that could help prevent relapse in children and young adults battling aggressive bone cancers.Penn State Erie Today

A new clinical trial led by the Penn State College of Medicine-based Beat Childhood Cancer Research Consortium will evaluate whether the drug eflornithine (DFMO) can help prevent relapse and improve outcomes for patients with Ewing sarcoma and osteosarcoma - two aggressive bone cancers that have seen few meaningful treatment advances in decades.

Why it matters

Ewing sarcoma and osteosarcoma are rare but aggressive cancers that primarily affect children, adolescents and young adults. While survival rates have improved for some pediatric cancers in recent decades, outcomes for patients with relapsed or metastatic sarcomas remain poor, making the development of new treatment options crucial.

The details

The Phase II clinical trial, known as BCC023, will assess the safety and effectiveness of DFMO in combination with standard treatments, as well as DFMO used alone in certain patient groups, for Ewing sarcoma and osteosarcoma. DFMO works by blocking an enzyme called ornithine decarboxylase (ODC), which plays a key role in cell growth and division, potentially slowing or stopping the growth of cancer cells.

  • The BCC023 study is expected to begin enrolling patients this month.
  • The trial is designed to enroll approximately 369 patients through the consortium's international network of more than 50 hospitals.

The players

Giselle Saulnier Sholler

Chair and founder of the Beat Childhood Cancer Research Consortium, Four Diamonds Endowed Chair for Pediatric Oncology Research in the Department of Pediatrics, and a professor of neuroscience and experimental therapeutics at Penn State College of Medicine.

David Loeb

Pediatric oncologist at Children's Hospital at Montefiore and study chair of the BCC023 study.

Beat Childhood Cancer Research Consortium

An international network of more than 50 hospitals that conducts collaborative research to develop new treatment options for pediatric solid tumors.

Penn State College of Medicine

The institution leading the new clinical trial and home to the Beat Childhood Cancer Research Consortium.

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What they’re saying

“The DFMO approval represented years of collaborative work focused on improving outcomes for children with neuroblastoma. What is especially exciting about the new clinical trial is the opportunity to extend what we've learned about DFMO to help children and young adults facing other devastating cancers.”

— Giselle Saulnier Sholler, Chair and founder of the Beat Childhood Cancer Research Consortium

“There have been few recent advancements in standard of care for Ewing sarcoma and osteosarcoma. Based on results from our clinically relevant translational mouse model, we anticipate that DFMO will prevent future disease relapse in children and young adults with osteosarcoma and Ewing sarcoma once radiographically free of disease.”

— David Loeb, Pediatric oncologist at Children's Hospital at Montefiore and study chair of the BCC023 study

What’s next

Researchers will evaluate several outcomes from the BCC023 study, including event-free survival, relapse-free survival and disease control at 12 months.

The takeaway

This new clinical trial represents a significant step forward in the fight against rare and aggressive pediatric bone cancers, as researchers work to expand the potential of the DFMO drug to improve outcomes for children and young adults facing these devastating diseases.