Scientists from Northwestern University (USA) have found that the anticonvulsant drug levetiracetam may prevent the formation of toxic protein plaques in the brain — one of the key hallmarks of Alzheimer’s disease. The study results were published in the journal Science Translational Medicine (STM).

Researchers discovered that the most dangerous form of amyloid — the amyloid-beta 42 protein — accumulates inside synaptic vesicles of neurons involved in signal transmission between cells. This process is believed to trigger the formation of toxic aggregates that damage the brain.

Levetiracetam, approved by the U.S. Food and Drug Administration (FDA) more than 20 years ago for the treatment of epilepsy, interferes with this mechanism. The drug slows protein processing inside neurons, thereby preventing the formation of amyloid-beta 42.

The effect was tested in animal models, cultured human neurons, and brain tissue samples from individuals with Down syndrome — a group at extremely high risk of early-onset Alzheimer’s disease. In all cases, use of the drug reduced production of the toxic protein. In addition, analysis of clinical data showed that patients with Alzheimer’s disease who were taking levetiracetam experienced slower progression of cognitive decline.

The authors emphasize that the drug’s potential effectiveness is likely limited to the earliest stages of the disease — before pronounced symptoms appear. Once dementia develops, many pathological changes in the brain become largely irreversible.