New drug approach for diffuse midline glioma (DMG)
Project 2: Druggable dependencies in diffuse midline gliomas
This project is trying an oral drug called orludodstat that targets a DNA-repair weakness in H3K27M-mutant diffuse midline glioma, mainly affecting children.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11178590 on NIH RePORTER |
What this research studies
You would be offered a treatment based on lab work showing these tumors rely on an alternative DNA-repair pathway. Researchers identified brain-penetrant drugs and are opening a lead-in phase 1 trial of orludodstat, a DHODH blocker, to see how it behaves in people. The team completed preclinical studies showing activity in models and prepared the drug for clinical testing. If you qualify, you would receive the study drug with regular scans and labs to monitor safety and any tumor effects.
Who could benefit from this research
Good fit: Ideal candidates are children (and possibly young adults) with H3K27M-mutant diffuse midline glioma who meet phase 1 safety and eligibility criteria.
Not a fit: Patients without the H3K27M mutation, with other tumor types, or who are medically unfit for a phase 1 trial are unlikely to benefit.
Why it matters
Potential benefit: If successful, the drug could slow tumor growth or make tumors more vulnerable to other treatments, possibly improving survival or quality of life.
How similar studies have performed: Laboratory and animal studies showed promising activity for targeting the alt-EJ/DHODH pathway, but clinical benefit in humans has not yet been proven.
Where this research is happening
Boston, United States
- Brigham and Women's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Haas-Kogan, Daphne a. — Brigham and Women's Hospital
- Study coordinator: Haas-Kogan, Daphne a.
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 below.