KU Leuven investigates antifungal drug as possible treatment for drug-resistant epilepsy

A research team at KU Leuven is studying whether clioquinol, a drug traditionally used to treat bacterial and fungal infections, could help the 25 million people worldwide with epilepsy who do not respond to existing medications. Early findings suggest that it may be effective.
Epilepsy affects around 80 million people worldwide. Despite the existence of over 20 types of medication, around one-third of patients remain resistant to treatment. The KU Leuven team has identified a new therapeutic target: the PHGDH enzyme, which plays a role in producing glycine and serine (amino acids that act as neurotransmitters) and helps to reduce brain inflammation.
Dual action
The researchers discovered that clioquinol increases the activity of PHGDH, which in turn lowers the brain's levels of glutamate - a neurotransmitter associated with seizures. This dual action appears to produce anti-seizure and anti-inflammatory effects.
Promising results have been observed in laboratory tests involving human cells and animal models. In a small-scale pilot study involving young adults with treatment-resistant epilepsy, the majority of participants experienced improvements in seizure frequency and overall quality of life. A clinical trial involving 20 patients is now being prepared to validate these results.
Lead researcher Karin Thevissen emphasises the unexpected path that the project has taken: "This started twenty years ago as research into how fungi resist treatment. The fact that it may now contribute to managing epilepsy, and possibly other disorders with similar mechanisms, is extraordinary.”
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