Safer Epilepsy Treatments for Women with Lab-Grown Organoids

Image credit: Giovanni Pietrogrande, The University of Queensland (used with permission)

A promising breakthrough from Queensland researchers could provide women with epilepsy safer access to a widely used anti-seizure medication.

The anticonvulsant drug, Sodium Valproate – also known as valproic acid – is commonly prescribed for epilepsy and some mental health disorders, but its use during pregnancy is deemed harmful due to associations with spinal cord defects and other complications in newborns.

Using lab-grown organoids, a team led by the University of Queensland’s Australian Institute for Bioengineering and Nanotechnology (AIBN) has discovered a drug that may eliminate the harmful side effects.

Researchers initially aimed to uncover why valproate leads to spinal cord malformations in foetuses by using organoids – tiny human spinal cords grown in a dish – that closely resemble a foetal spinal cord during early gestation.

“When these mini spinal cords were exposed to valproate, the team discovered that the drug changes the cells that normally form the spinal cord, leading to malformations,” explained the AIBN’s Dr Giovanni Pietrogrande in a story with the University of Queensland.

The AIBN team subsequently treated the organoids with the clinically-approved drug Rapamycin, discovering that it prevented the adverse effects of valproate – pointing to a potential method for unlocking safe access to an otherwise effective treatment for women with epilepsy.

“Organoids are a tool that allow us to develop new treatments, and also to uncover new ways to improve the safety and efficacy of existing ones,” said study co-researcher, the AIBN’s Professor Ernst Wolvetang.

“We hope this research is another step towards fostering regulatory change in the Therapeutic Goods Administration (TGA) to establish organoids as a powerful tool for drug screening and discovery.”

Outcomes of the study were published last month in Molecular Psychiatry.

The research was undertaken in partnership with the AIBN, the University of Queensland’s School of Chemical Engineering, The University of Melbourne and Vita-Salute San Raffaele University in Milan.

For further information, check out this story from the University of Queensland or refer to the research paper in Molecular Psychiatry.