A look at ONC201, a drug approved for one hard brain tumor
Original title: ONC201/dordaviprone for H3 K27M-mutant tumors: Discovery, mechanisms, therapeutic potential, and future directions.
How far along is this research?
- Lab cells
- Animals
- Review
- Tested in people
This looks across many earlier studies rather than running a new one.
The drug is approved for one type of brain tumor that has come back, but other uses are still being tested.
The short version
Experts review what is known about ONC201, a drug now approved for one type of brain tumor that has come back.
What was studied. This is a review of past research, not a new study. It looks at how the drug ONC201, also called dordaviprone, works and how it has done in tests.
What they found. ONC201 got a fast approval to treat diffuse midline glioma: A tumor that starts in the glial cells, the support cells of the brain and spinal cord. Gliomas are given a grade from 1 to 4 describing how the tumor is expected to behave. See the glossary with the H3 K27-altered: A change in a histone, one of the proteins that DNA wraps around. When a diffuse glioma has it, and sits in the middle of the brain or spine, it is called a diffuse midline glioma. See the glossary change that has come back. It was the first drug approved this way for this disease. In trials it seemed safe, and it is a pill taken once a week, but some tumors may learn to resist it.
What this means, and what it doesn't
What it could mean: If your tumor has the H3 K27-altered: A change in a histone, one of the proteins that DNA wraps around. When a diffuse glioma has it, and sits in the middle of the brain or spine, it is called a diffuse midline glioma. See the glossary change and has come back, you can ask your doctor if this drug is right for you. Researchers are also testing it with other drugs and in other cancers.
What it doesn't mean: This paper is a review, not a new result. A fast approval means more proof is still being gathered. The drug is only approved for one tumor type. It is not a cure, and it may stop working for some people over time.
Source: PubMed, June 24, 2026 · Read the original
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