A Drug Aimed at a Gene Change Helped a Baby With a Brain Tumor
Original title: Infant-Type Hemispheric Glioma With NTRK Fusion Responds to Larotrectinib.
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 one child treated in real care, not a trial, so it is not proven for others.
The short version
Doctors found a gene change in a baby's brain tumor, and a drug aimed at that change helped.
What was studied. This is a report about one baby, four months old, with a large brain tumor. Doctors looked at the tumor under a microscope and tested its genes.
What they found. The tests showed a type of brain tumor called infant-type hemispheric 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. The gene tests found a change called a TPM3::NTRK1 fusion. After surgery, the baby was given a drug that targets NTRK. The tumor responded, and the response lasted, both in the clinic and on scans.
What this means, and what it doesn't
What it could mean: Gene testing of a tumor can change what treatment is offered. When a tumor has an NTRK change, there may be a drug aimed at it. It is fair to ask the care team whether the tumor has had gene testing.
What it doesn't mean: This is one child's story, not a study of many people. One case cannot tell us how often this drug works or who it will help. It does not say how long the good result will last. It is not a cure, and it is not proof that it works for other people. This kind of tumor and this gene change are rare.
Source: PubMed, August 6, 2026 · Read the original
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