ONC206 — an oral drug for children with diffuse midline glioma and recurrent malignant 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-11410955 · Data updated 2026-11-12
This work will see if the oral drug ONC206 is safe and shows early signs of benefit for children with diffuse midline glioma and other recurrent malignant brain tumors.
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 | 2025 |
| Award (this year) | $36,720 |
| NIH project number | 3R01CA266596-04S2 |
| Project ID | NIH-11410955 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with diffuse midline glioma (including H3K27M-mutant tumors) or other recurrent malignant pediatric brain tumors who meet the study's medical and safety criteria and can attend visits at the study site.
✕Less likely to help
Patients without these tumor types, those who do not meet safety or organ-function requirements, or those unable to travel to the study location are unlikely to benefit from participation.
What this research studies
If you join, you would receive oral ONC206 in carefully monitored dose groups to find a safe dose and learn how the drug behaves in the body. The trial includes four cohorts testing ONC206 alone and in combination with radiation for newly diagnosed or re-irradiated diffuse midline glioma and other recurrent malignant pediatric brain tumors. Doctors will collect blood and tumor-related markers to measure drug effects on the mitochondrial target ClpP, monitor side effects, and look for early signs of tumor response. The main goals are to define a recommended phase 2 dose, characterize safety and pharmacokinetics, and gather preliminary evidence of benefit.
Why it matters
Potential benefit: If successful, ONC206 could provide a new treatment option that crosses the blood–brain barrier and may shrink or slow tumors in children with these aggressive brain cancers.
How similar studies have gone: Related imipridone drugs such as ONC201 have shown early signals of activity in some brain tumors, but ONC206 is newer and its pediatric safety and effectiveness are not yet established.
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
A first-in-children trial tests the safety and early effectiveness of a pill that can cross the brain barrier to treat pediatric brain tumors.
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ONC206 for children with dangerous brain tumors
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.
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Oral bacteria and brain metastasis: how the mouth may influence brain tumors
A study exploring how oral microbes may affect brain metastases and patient outcomes, with potential to guide new microbiome-based approaches.
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Bioengineered brain barrier chip to improve drug screening for brain tumors
A small, patient-friendly brain barrier chip is being developed to test drugs for glioblastoma more accurately before they reach clinical trials.
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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.
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.