A pill called erdafitinib was tested in cancers with FGFR gene changes, including brain tumors
Original title: Erdafitinib in Patients With Advanced Solid Tumors With FGFR Alterations (RAGNAR): Results of Tumor-Specific Analyses and Secondary Cohorts.
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 tested in people in a mid-stage trial, but it is not approved or standard care for brain tumors.
The short version
A pill helped shrink tumors in a small number of people with glioma: A tumor that starts in the glial cells, the support cells of the brain and spinal cord. Gliomas are graded 1 to 4 by how fast they tend to grow. See the glossary whose cancer had a specific gene change.
What was studied. This study tested a pill called erdafitinib in adults and children age 6 and older with advanced cancer that had changes in a gene called FGFR. Everyone in the study had already tried other treatments and run out of standard options. The study included people with many cancer types, including high-grade glioma: A tumor that starts in the glial cells, the support cells of the brain and spinal cord. Gliomas are graded 1 to 4 by how fast they tend to grow. See the glossary and low-grade glioma.
What they found. In high-grade glioma: A tumor that starts in the glial cells, the support cells of the brain and spinal cord. Gliomas are graded 1 to 4 by how fast they tend to grow. See the glossary, tumors shrank in 3 of 30 people. In low-grade glioma, tumors shrank in 2 of 7 people. In the group of 11 children, one child's tumor shrank and seven children had disease that stayed stable for a while. Side effects matched what doctors already know about this drug. In children, side effects included a growth plate injury in the bone and a broken limb.
What this means, and what it doesn't
What it could mean: For people with glioma: A tumor that starts in the glial cells, the support cells of the brain and spinal cord. Gliomas are graded 1 to 4 by how fast they tend to grow. See the glossary who have an FGFR gene change and have run out of options, this drug is worth asking a doctor about. It may be available through a trial. Testing your tumor for FGFR changes is the first step, because the drug was only used in people who had that change.
What it doesn't mean: This does not mean there is a new approved treatment for brain tumors. This drug is approved for a bladder-type cancer, not for glioma: A tumor that starts in the glial cells, the support cells of the brain and spinal cord. Gliomas are graded 1 to 4 by how fast they tend to grow. See the glossary. This was a mid-stage study in a small number of people, and most people's tumors did not shrink. It is not a cure, and it is not standard care your doctor can simply prescribe for a brain tumor today.
Source: PubMed, July 15, 2026 · Read the original
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