A targeted drug helped a baby with a rare spinal cord tumor
Original title: A Case of Primary Intramedullary Infantile Fibrosarcoma With an RBPMS::NTRK3 Fusion and Response 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 is a report on one baby, not a trial in a group of people.
The short version
One baby with a rare tumor got much better on a drug matched to the tumor's gene change.
What was studied. Doctors report on one baby, 4 weeks old, with a rare tumor inside the spinal cord in the neck. The tumor was growing fast and hurting the baby's nerves. Gene tests on the tumor found a change called an RBPMS::NTRK3 fusion.
What they found. Lab tests showed the tumor was a type called infantile fibrosarcoma. Doctors gave the baby a drug called larotrectinib, which targets this kind of gene change. Within 1 month, the baby was clearly better, and scans showed the tumor had improved too.
What this means, and what it doesn't
What it could mean: Gene testing on a rare tumor like this can matter. It may point to a drug made for that exact gene change. This kind of tumor in the spinal cord has not been treated this way often, so the report adds useful knowledge for doctors.
What it doesn't mean: This is one case report, not a trial. One baby doing well does not prove the drug works for others. The report does not say how long the good result lasted. It is not a cure, and it does not change care for people whose tumors lack this gene change.
Source: PubMed, October 1, 2026 · Read the original
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