A widely prescribed anti-seizure medicine slowed the growth of one of childhood's deadliest brain cancers in laboratory research, Stanford Medicine scientists reported.
The drug, levetiracetam, is often sold under the brand name Keppra. Medical records also showed that children with diffuse midline gliomas who received it lived longer on average than those who did not, according to Medical Xpress.
For families facing this diagnosis, the news offers a rare signal of possible progress. It also comes with a clear limit: researchers say clinical trials are needed to learn whether the drug actually helps patients.
An Existing Medicine Aimed at a New Target
Diffuse midline gliomas grow in the brainstem, thalamus, and spinal cord. They affect 300 to 400 U.S. children each year, and the five-year survival rate is about 1%. The group includes diffuse intrinsic pontine glioma, or DIPG.
Earlier work from the lab of Dr. Michelle Monje, a professor of neurology and pediatric neuro-oncology at Stanford Medicine, showed that these tumors can receive signals from healthy neurons through connections called synapses. The cancer cells use those signals to fuel their growth.
The new study, published in Nature Medicine, found that levetiracetam targets a type of these connections, known as GABAergic synapses, in diffuse midline glioma.
"This drug pharmacologically severs an important connection between cancer and the nervous system," Monje said, according to Inside Precision Medicine. "This is very exciting for a disease that has had so few treatment options."
Records from 218 Children Pointed the Same Direction
Earlier studies had suggested that brain cancer patients taking levetiracetam might live longer, but the results were inconsistent. The Stanford team reviewed medical records from 218 children with high-grade gliomas to look more closely.
The pattern depended on tumor location. Among children with diffuse midline glioma, levetiracetam use was associated with longer survival. Among children with hemispheric high-grade gliomas, which grow in other parts of the brain, the drug showed no survival benefit.
The researchers then tested the drug in mice carrying tumors grown from patient cells, known as patient-derived xenograft models. The results in those models supported what the records suggested. The team is now planning clinical trials.
MedicalDaily Evidence Check: The study combined laboratory experiments, animal models, and a retrospective review of 218 patient records, published in Nature Medicine. It found that levetiracetam slowed tumor growth in lab models and was associated with longer survival in children with diffuse midline glioma. It did not prove that the drug extends life, because the records did not come from a randomized trial, and children who received the drug may have differed in other ways. Treatment guidelines have not changed.
What Parents Should Know Before Asking About the Drug
Levetiracetam is already widely used and has a well-established safety record as a seizure medicine. That familiarity is part of what makes the finding appealing, since an approved drug could move into trials faster than a brand-new compound.
Even so, parents should not give the medication to a child, or change a dose, without guidance from the child's oncology team. Doctors will need to determine whether the right dose for tumor control differs from seizure dosing and whether the drug can be safely combined with radiation, chemotherapy, or experimental treatments. Levetiracetam can also cause side effects, including mood and behavior changes.
Some children with brain tumors already take levetiracetam to control seizures. Families in that situation can ask whether the new findings affect the care plan, but the answer will depend on each child's circumstances.
Families who want to explore clinical trials can ask their child's neuro-oncologist or search the federal ClinicalTrials.gov registry. Traveling to a major pediatric cancer center can add costs, and many centers have social workers who can help families find travel grants and lodging assistance.
Where Research on These Tumors Stands
Diffuse midline gliomas usually cannot be removed by surgery because of their location. Radiation can relieve symptoms for a time, but the disease almost always returns. Doctors typically diagnose these tumors with MRI scans, and a biopsy may be used to confirm genetic features, including the H3 K27M change found in many cases. That genetic information increasingly shapes which clinical trials a child may join.
Stanford has also tested other approaches. In an early trial of engineered immune cells called GD2-CAR T cells, published in Nature in late 2024, most of the 11 participants showed neurological improvement, and four had their tumors shrink by more than half. That therapy remains experimental, according to the Stanford Cancer Institute.
Warning signs of brain tumors in children can include new problems with balance or walking, double vision, facial weakness, trouble swallowing, persistent headaches with vomiting, and changes in behavior. Children with rapidly worsening symptoms should be evaluated urgently.
For now, the study offers families a science-based reason for cautious hope. It does not offer a new treatment yet, and the next step will be carefully designed trials that test the drug in children with these tumors.
Key Questions Answered
What did the Stanford study find? Levetiracetam slowed diffuse midline glioma growth in lab and mouse models, and children with these tumors who took the drug lived longer on average in a review of medical records.
What is diffuse midline glioma? An aggressive cancer of the brainstem, thalamus, and spinal cord that affects 300 to 400 U.S. children each year, with a five-year survival rate of about 1%.
Is levetiracetam now a treatment for this cancer? No. Researchers say clinical trials are needed to show whether it benefits patients.
Should parents ask for the drug? Parents can discuss the research with their child's oncology team but should not start or change any medication without medical guidance.
Where can families find clinical trials? Through their child's neuro-oncologist or the federal ClinicalTrials.gov registry.
