A HER2 drug kept two rare brain tumors steady
Original title: Durable treatment response to Trastuzumab deruxtecan in two patients with recurrent grade III anaplastic ependymoma.
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.
It was used in only two people outside a trial, so it is not proven yet.
The short version
Two people whose rare brain tumor kept coming back stayed stable for many months on a drug called T-DXd.
What was studied. Doctors report on two people with anaplastic ependymoma, a rare and fast growing brain tumor. The tumors had come back many times, tests showed HER2 activity, so each person was given the HER2 drug T-DXd off label.
What they found. In the first person, the tumor stopped growing and stayed under control for 26 months. The second person first tried two other drugs, and the tumor kept growing. After switching to T-DXd, that tumor stayed stable for 14 months. Side effects hit blood counts and the liver, and doctors handled them with extra care, lower doses, and short breaks.
What this means, and what it doesn't
What it could mean: If your ependymoma keeps coming back and tests show HER2, this drug may be worth asking your care team about. It did not make the tumors go away. It held them steady for a time.
What it doesn't mean: This is a report on just two people, not a trial. It does not show the drug works for most people with this tumor. T-DXd is not approved for ependymoma, so it was used off label. It is not a cure. Real trials in more people are needed before this becomes normal care.
Source: PubMed, October 7, 2026 · Read the original
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