ONC206 for children with brain tumors: a first-in-children trial
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-11535240 · Data updated 2026-11-12
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.
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) | $12,000 |
| NIH project number | 3R01CA266596-05S3 |
| Project ID | NIH-11535240 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are pediatric patients with malignant brain tumors, including diffuse midline gliomas, who can tolerate oral therapy and may participate in combination arms.
✕Less likely to help
Some participants may not experience benefit or may experience treatment-related side effects.
What this research studies
This trial will enroll pediatric patients to see if ONC206 is safe and provides early clinical signals of benefit. Researchers will measure safety, how the drug moves in the body, and preliminary anti-tumor activity, including in combination with radiation for some groups. The study builds on lab data showing ONC206 activates a mitochondrial pathway (ClpP) that can trigger tumor cell death. Four patient cohorts will be studied, including newly diagnosed and relapsed cases, to determine the recommended dose for future studies.
Why it matters
Potential benefit: If successful, children with certain brain tumors could have a new therapy option and potentially improved outcomes.
How similar studies have gone: Preclinical data show ONC206 activity and survival benefit in models, supporting translation to a pediatric trial.
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.