Researchers have made a noteworthy advancement in helping women with epilepsy gain safer access to a widely used and effective anti-seizure medication. Sodium Valproate, also known as valproic acid, is commonly prescribed for epilepsy and some mental health disorders, but it poses risks during pregnancy, including potential spinal cord defects and other complications in newborns. A team of organoid specialists has identified a medication that might counteract these harmful side effects.
Researchers at the University of Queensland have made an important breakthrough that allows women with epilepsy to safely use a frequently prescribed and effective anti-seizure medication.
Sodium Valproate, or valproic acid, is often used to treat epilepsy and various mental health issues but is known to be dangerous during pregnancy due to associations with spinal cord defects and other issues in infants.
Dr. Giovanni Pietrogrande and Professor Ernst Wolvetang from UQ’s Australian Institute for Bioengineering and Nanotechnology (AIBN) led a team of organoid researchers who found a drug that could mitigate these harmful effects.
“Our initial goal was to understand why valproate leads to spinal cord problems in developing fetuses,” Dr. Pietrogrande explained.
“To investigate this, we created organoids – miniature human spinal cords – that replicate the spinal cord of a fetus during the early stages of pregnancy.
“When these mini spinal cords were subjected to valproate, our team found that the drug alters the cells that usually develop into spinal cord tissue, resulting in malformations.”
Professor Wolvetang noted that the AIBN team subsequently treated the organoids with the clinically proven drug Rapamycin, which successfully prevented the harmful effects associated with valproic acid.
“Thus, combining treatment with Rapamycin could pave the way for safe access to this highly effective treatment for women suffering from epilepsy,” Professor Wolvetang added.
Co-author Professor Terence O’Brien, who leads the School of Translational Medicine at Monash University and serves as program director and deputy director of research at Alfred Brain, stated that the research findings may assist healthcare providers and patients in managing the complex issues related to epilepsy treatment.
“This could also lead to enabling women to continue using this life-saving medication while having healthy children,” Professor O’Brien remarked.
Professor Wolvetang emphasized that this research showcases the capabilities of innovative technologies like human stem cell-derived organoids in studying the molecular and cellular effects of medications.
“Organoids are a valuable resource that allow us to create new treatments and discover new methods to enhance the safety and effectiveness of existing ones,” he explained.
“We hope this study contributes to encouraging regulatory changes in the Therapeutic Goods Administration (TGA) to recognize organoids as a significant instrument for drug testing and discovery.”
This research was a collaborative effort with AIBN colleagues Dr. Mohammad Shaker, Dr. Julio Aguado, Dr. Ibrahim Javed, Professor Tom Davis, Tahmina Tabassum, and Sean Morrison, along with partners from UQ’s School of Chemical Engineering, The University of Melbourne, and Vita-Salute San Raffaele University in Milan.
