A medication used to treat epilepsy could reduce the risk of developing Alzheimer’s, scientists have discovered.
The brain disease affects up to 800,000 people in Spain and is becoming one of the major health challenges on the planet.
It is caused by the build-up of toxic proteins in the brain that slowly destroy communication between neurons, and there is still no cure.
But a new experimental study from Northwestern University suggests that a widely used epilepsy drug could help stop the disease before it takes hold – and may even restore some neuronal function in affected brain areas.
The research, published in Science Translational Medicine, tested the anticonvulsant drug levetiracetam in animal models, human neurons and brain tissue from people at high risk of Alzheimer’s, including individuals with Down syndrome.
The results showed that in laboratory conditions, the drug appeared to prevent the formation of the toxic proteins that trigger Alzheimer’s.
How it works
At the centre of the disease is a protein called APP. Inside neurons, APP can follow one of two biological ‘routes’. One pathway is harmless. The other produces a toxic fragment known as amyloid-beta 42 – the molecule that kickstarts plaque formation in the brain.
In younger brains, cells largely keep APP on the safe path. But as we age, that control weakens. In people who develop Alzheimer’s, too much APP is diverted down the harmful route, leading to amyloid plaques, tau tangles, neuron death, inflammation and ultimately dementia.
The study suggests levetiracetam may act as an early ‘brake’, preventing neurons from producing amyloid-beta 42 in the first place. Rather than clearing existing plaques, it appears to alter how APP is processed inside cells – stopping the cascade before it begins.
Jeffrey Savas, professor of behavioural neurology at Northwestern’s Feinberg School of Medicine and senior author of the study, described the findings as pointing to a possible ‘new pharmacological target’.
A drug already in use in Spain
One of the major advantages, researchers say, is that levetiracetam is not experimental as it has been prescribed worldwide for epilepsy for more than 25 years and has an established safety profile. In Europe, including Spain, it has been in use since the early 2000s and is widely available in generic form.
Scientists also analysed large medical databases to examine Alzheimer’s rates among epilepsy patients taking levetiracetam. While some still developed the disease, diagnoses tended to occur later and progression appeared slower compared to those who had never taken the drug.
Researchers stress that this does not prove causation, but it does suggest a possible protective effect that warrants further targeted clinical trials.
Prevention, not cure
For now, the potential benefit appears primarily preventive. According to the research team, high-risk individuals might need to begin treatment decades before symptoms appear – possibly 20 years earlier – to meaningfully reduce risk.
That raises significant questions about screening, long-term medication use and who would qualify for preventive treatment.
The team is now working on developing an improved version of the drug. Standard levetiracetam breaks down relatively quickly in the body and was designed to control seizures, not specifically target Alzheimer’s pathways. Researchers hope to create a longer-lasting, more selective compound that could enhance its preventive potential.
Experts caution that more studies – particularly large human trials – are needed, but that the findings represent one of the more promising preventive avenues in Alzheimer’s research.
