A Targeted Drug for Children With BRAF Brain Tumors
Original title: Efficacy Study of Vemurafenib in Children With Recurrent or Progressive BRAFV600E- or BRAFInsT-Mutant Brain Tumors: PNOC002.
How far along is this research?
- Lab cells
- Animals
- Review
- Tested in people
This was tested in people. That is the most reliable kind of research we share.
This was an early trial in people, mostly to find the right dose.
The short version
A drug called vemurafenib helped many children whose brain tumors came back.
What was studied. Thirty young people, ages 3 to 23, took vemurafenib. All had brain tumors with a BRAF: A gene in a pathway that tells cells to divide. Some tumors carry a change in it. There are drugs made to target that change, so your team may test for it. See the glossary gene change that had come back or kept growing. 22 had low grade 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 and 8 had high grade glioma.
What they found. At 6 months, 80% of the whole group had no tumor growth. For low grade 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 that number was 86%. For high grade glioma it was 63%. Tumors stayed the same size in 13 patients and shrank in 13 others.
What this means, and what it doesn't
What it could mean: This drug may help some children whose tumor has this BRAF: A gene in a pathway that tells cells to divide. Some tumors carry a change in it. There are drugs made to target that change, so your team may test for it. See the glossary change. Side effects were seen as manageable by the study team. Still, 57% had a bad skin rash. And tumors grew back in 3 of 13 children with low grade 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 who stopped the drug.
What it doesn't mean: This was an early study in people. It was small, and its main goal was to find a safe dose. It is not a cure, and it is not standard care you can ask for today. It only looked at tumors with the BRAF: A gene in a pathway that tells cells to divide. Some tumors carry a change in it. There are drugs made to target that change, so your team may test for it. See the glossary change. It also does not promise the tumor will stay away after the drug stops.
Source: PubMed, September 23, 2026 · Read the original
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