A drug called trametinib helped many children with low-grade glioma
Original title: Real-life efficacy and tolerance of trametinib in BRAF-rearranged low-grade gliomas: A multicenter study.
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.
Doctors looked back at records of children already given this drug, not a planned trial.
The short version
In a look-back study of 46 children, this drug kept most tumors in check.
What was studied. Doctors looked back at 46 children and teens with low-grade glioma: A tumor that starts in the glial cells. Those are the support cells of the brain and spinal cord. Gliomas are given a grade from 1 to 4. The grade says how the tumor is expected to act. See the glossary. Each tumor had a change in 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. All of them took a drug called trametinib at three centers in France between 2015 and 2023.
What they found. Scans showed the tumor was under control in 78% of patients, or 36 of 46. Tumors got smaller in 65%, and stayed the same size in 13%. 72% of patients got better. Side effects were common. Skin problems happened in 69% and nail infections in 64%. One third of patients had a serious side effect. Of the 35 who stopped the drug, 37% were still off treatment later.
What this means, and what it doesn't
What it could mean: This drug may help hold these tumors steady. It seemed to work even in children who had already tried many other treatments. But side effects are common, and some are serious. A care team would need to watch the skin, nails, eyes, and blood tests closely.
What it doesn't mean: This is not a cure, and it is not proof. The doctors looked back at old records instead of running a planned trial. No one was picked at random, and there was no comparison group. That makes the results much less certain. Many patients also stopped the drug. Talk with your own care team about what fits your child.
Source: PubMed, October 2, 2026 · Read the original
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