By Steven O. Mukoro
Research scientists have taken a vital step aimed at making it possible for women with epilepsy to have safer access to an ordinary but extremely effective anti- seizure medicine. Sodium Valproate or valproic acid is extensively approved for epilepsy and certain mental health ailments, but has always been seen as unsafe for use during pregnancy owing to links to spinal cord defects in addition to other complications for new born babies. A team of organoid scientists have come up with a medication that will possibly quash the unsafe side effects.
A team of researchers from University of Queensland have made a significant step that would enable women with epilepsy gain safer access to a common and very effective anti-seizure drug.
Sodium Valproate or valproic acid is extensively used by doctors to treat epilepsy and specific mental health illnesses, but is looked at as being harmful to use in the course of pregnancy in that it has links to spinal cord defects and other complications for babies newly born.
Dr Giovanni Pietrogrande and Professor Ernst Wolvetang from UQ’s Australian Institute for Bioengineering and Nanotechnology (AIBN) led a team of organoid experts who have identified a medication that is capable of cancelling out the hazardous side effects.
“We first set out to understand why valproate causes spinal cord malformations in fetuses,” Dr Pietrogrande said.
“To do this we created organoids — human mini-spinal cords in a dish — that closely mimic the spinal cord of a fetus in the early weeks of 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.”
Professor Wolvetang said the AIBN researchers subsequently treated the organoids with the clinically approved medication Rapamycin, and discovered that it prohibited the negative effects of valproic acid.
“Therefore co-treatment with Rapamycin could be the thing that opens safe access to an extremely effective treatment for women with epilepsy,” Professor Wolvetang said.
Co-author of the research, Professor Terence O’Brien, who heads the School of Translational Medicine at Monash University and programme director as well as deputy director of research at Alfred Brain, said the discoveries could assist healthcare providers and patients explore the difficult problems about the treatment of epilepsy.
“It may also provide a pathway to enabling women to continue to take this life saving medication while having healthy children,” Professor O’Brien said.
Professor Wolvetang stated that the research highlights the possibility of new technologies such as human stem cell derived organoids to explore the molecular and cellular effects of medications.
“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,” he said.
“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.”
The research was carried out in collaboration with AIBN colleagues Dr Mohammad Shaker, Dr Julio Aguado, Dr Ibrahim Javed, Professor Tom Davis, Tahmina Tabassum and Sean Morrison, in addition to colleagues from UQ’s School of Chemical Engineering, The University of Melbourne and Vita-Salute San Raffaele University in Milan.
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