GRANT AWARDED FOR PHASE1A CLINICAL TRIAL
New Grant Advances Targeted Radioimmunotherapy Trial for Children with DMG
The Children’s Brain Tumor Project at Weill Cornell Medicine has been awarded a $50,000 grant from The Cure Starts Now Foundation and its collaborative partner organizations to support the launch of a groundbreaking clinical trial for children and young adults with diffuse midline glioma (DMG), including DIPG.
The study — “A Phase 1a Open-label Study Evaluating the Safety of 177Lu-DTPA-omburtamab Delivered by CED in Non-Progressive DMG Patients Previously Treated with External Beam Radiation Therapy” — represents the next evolution of more than a decade of pioneering work in convection-enhanced delivery (CED) led by Dr. Mark Souweidane.
This next iteration of the trial will evaluate a targeted radioactive antibody, 177Lu-DTPA-omburtamab, delivered directly into the tumor using CED — a highly specialized technique designed to bypass the blood-brain barrier and achieve concentrated treatment within the tumor while minimizing exposure to the rest of the body. The therapy targets B7-H3, a protein highly expressed on DMG tumor cells but largely absent from normal brain tissue.
Importantly, this study builds upon previously published peer-reviewed clinical research demonstrating the feasibility and safety of delivering radiolabeled omburtamab directly into the brainstem of children with DIPG. Those landmark findings, recently published in Neuro-Oncology, established the scientific foundation for advancing this next-generation therapeutic strategy.
Unlike traditional systemic therapies, this approach combines precision-targeted radioimmunotherapy with real-time imaging capabilities, allowing investigators to monitor drug distribution and dosimetry after treatment. Researchers hope the study will help define a safe repeat-dosing schedule while also informing future applications of localized radiopharmaceutical therapy across pediatric brain tumors.
The proposed trial reflects the growing momentum behind collaborative, translational approaches to pediatric neuro-oncology and highlights the importance of sustained philanthropic investment in innovative, high-risk, high-impact research.
The team also emphasized the importance of collaborative science and data sharing across the pediatric brain tumor community, including participation in the DIPG/DMG Collaborative and future dissemination of study findings through presentations, publications, and shared clinical updates.
This work would not be possible without the generosity and vision of donor-supported organizations committed to accelerating progress for children facing devastating brain tumors. We are deeply grateful to The Cure Starts Now Foundation and its affiliated funding partners for helping move this promising therapy one step closer to children and families in need.