Two targeted drugs helped one man with a rare brain tumor
Original title: Leptomeningeal Dissemination in TERT Promoter-mutant Anaplastic Pleomorphic Xanthoastrocytoma Responding to BRAF-MEK Inhibition: A Case Report.
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 is a report on one patient, not a study in a group of people.
The short version
In one man, a rare brain tumor shrank for a while on two targeted drugs.
What was studied. Doctors reported on one patient, a 22-year-old man with a rare brain tumor called pleomorphic xanthoastrocytoma. The tumor came back, turned more aggressive, and later spread to the lining of his spine.
What they found. Tests on his tumor found two gene changes, called 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 and TERT promoter mutation: A change in the switch that controls the TERT gene. It keeps tumor cells from aging out. It is one of several findings your team reads together to name the tumor. See the glossary promoter. After radiation to his spine, he started two drugs, dabrafenib and trametinib. Scans showed the tumor partly shrank and his symptoms got better. It grew again 10 months after he started the drugs, but he was still alive when the report was written.
What this means, and what it doesn't
What it could mean: For 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 gene change, these two drugs may help for a time. Gene testing of a tumor can show if that change is there. It is worth asking your care team about testing.
What it doesn't mean: This is one person's story, not a trial. One case cannot show that a drug works for other people. The help did not last, and the tumor grew back. This is not a cure. There is still no set treatment when this tumor spreads along the spine.
Source: PubMed, August 14, 2026 · Read the original
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