ONC206 for children with dangerous brain tumors
Official title: Phase 0/1 trial of ONC206 - a novel imipridone for children with diffuse midline gliomas and recurrent malignant brain tumors
Funded at University of California, San Francisco · NIH-11535238 · Data updated 2026-11-12
A first-in-children study tests the safety and early effectiveness of an oral drug, ONC206, for kids with high-risk brain tumors that have spread or recurred.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | September 7, 2022 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $46,141 |
| NIH project number | 3R01CA266596-05S1 |
| Project ID | NIH-11535238 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children with malignant brain tumors, including diffuse midline gliomas, who may be eligible for a phase 0/1 dose-escalation and preliminary efficacy study.
✕Less likely to help
Some patients may not experience benefit or may experience side effects that preclude continued treatment.
What this research studies
This study will enroll pediatric patients with malignant brain tumors, including diffuse midline gliomas, to determine a safe starting dose and explore early signs of benefit. Researchers will give ONC206 by mouth and monitor how the drug behaves in the body, along with any side effects. The trial also explores how combining ONC206 with radiation might improve outcomes for newly diagnosed and relapsed patients. Data from four patient groups will help identify whether ONC206 could become a future treatment option for children with these difficult tumors.
Why it matters
Potential benefit: If successful, ONC206 could become a new treatment option that slows tumor growth or improves survival in children with malignant brain tumors.
How similar studies have gone: Preclinical data and early studies in models suggest ONC206 can trigger tumor cell death and may improve survival in relevant brain tumor models.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Mueller, Sabine — University of California, San Francisco
- Study coordinator: Mueller, Sabine
Related research
Other NIH-funded projects on the same topics.
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ONC206 for children with brain tumors: a first-in-children trial
A first-in-children trial will test the safety and early signs of effectiveness of ONC206, taken by mouth, in kids with malignant brain tumors like diffuse midline gliomas.
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ONC206 for children with malignant brain tumors
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Oral bacteria and brain metastasis: how the mouth may influence brain tumors
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Understanding how epigenetic changes drive pediatric brain tumor recurrence and how reversing them could help
A pediatric brain cancer research project is exploring how an epigenetic change (H3K27M) and tumor environment contribute to tumor coming back, using a reversible mouse model to study potential ways to prevent recurrence.
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SleepCARE: a patient-friendly way to help caregivers of brain tumor patients sleep better
A small grant is testing a telehealth sleep program designed to help caregivers of people with primary malignant brain tumors sleep better and feel less stressed.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.