Researchers at Stanford Medicine have shown that levetiracetam, an established anti-seizure medication, slows the growth of aggressive diffuse midline gliomas by severing their electrical connections to healthy neurons, offering new hope for a devastating childhood brain cancer.
Diffuse midline gliomas strike the brain and spinal cord, affecting roughly 300 to 400 U.S. children each year with a five-year survival rate hovering near 1%. For a condition historically burdened by a severe lack of treatment alternatives, the discovery marks a meaningful shift. The study was published Sept. 17 in Nature Medicine under the leadership of senior author Michelle Monje, MD, PhD, who serves as the Milan Gambhir Professor in Pediatric Neuro-Oncology at Stanford Medicine.
The findings stem from the emerging field of cancer neuroscience pioneered by Monje to examine how malignant cells hijack signals from the healthy nervous system. The research demonstrated that the medication pharmacologically severs an important connection between the tumor and the nervous system. Investigators discovered that levetiracetam operates through a distinct mechanism inside tumors compared with healthy neurons, raising the probability that it can target the malignancy without disrupting normal brain function.
How Levetiracetam Interrupts Brain Tumor Growth
Brain tumors frequently provoke seizures, leading clinicians to prescribe anti-seizure agents. Levetiracetam, widely recognized by the brand name Keppra, suppresses excess excitatory signaling between healthy neurons while carrying minimal side effects and few drug interactions.
To investigate whether the medication alters patient outcomes, Monje’s team examined medical records from 218 pediatric brain tumor patients treated across two academic medical centers. The data analysis revealed that levetiracetam linked to longer survival, on average, in patients with diffuse midline gliomas. Patients with hemispheric high-grade gliomas experienced no similar survival benefit.
Clinical Trials and the Intersection of Immunotherapy
The research team is already planning clinical trials to evaluate the drug further. Monje emphasizes that while levetiracetam alone will not cure these aggressive cancers, it serves as an effective companion therapy.
Because these repurposed neurological medications target the exact mechanisms hijacked by cancers, they offer a low implementation barrier. Researchers already understand their safety profiles.
Treating Grade 2 Gliomas in Young Adults
Adult diagnoses bring an entirely different set of clinical challenges, characterized by slow progression and complex molecular profiles. For 38-year-old Jonathan Lefebvre, a mounting series of headaches over several years culminated in an emergency room visit following an epileptic seizure. Doctors at the hospital diagnosed a grade 2 glioma, a diffuse cerebral cancer that infiltrates the brain between normal cells like root systems rather than forming a distinct mass.