The report discovered that a peptide found in spider venom could stimulate a crucial protein in mice with Dravet syndrome.
The secret to curing a deadly form of childhood epilepsy could come from an unlikely source: tarantulas.
A new study published in the Proceedings of the National Academy of Sciences discovered that a peptide found in spider venom, called Hm1a, could be used as a targeted therapy for Dravet syndrome, a genetic form of epilepsy.
Dravet syndrome is a rare form of epilepsy that causes frequent and prolonged seizures in children. According to the Dravet Foundation, the syndrome typically manifests in the first year of a child’s life and lasts until the end of their life. Children with Dravet syndrome typically suffer from developmental delays as a result of their frequent seizures and have a shortened life span.
The syndrome is a result of a genetic mutation in the brain that affects sodium channels, specifically the NaV1.1 protein. The exact number of people affected by Dravet syndrome is unknown, but it is estimated that 1 in 20,000 to 1 in 40,000 people are affected.
According to IFLScience, professor Steven Petrou of the Florey Institute of Neuroscience and Mental Health, one of the study’s authors, got the idea to conduct this study when he was talking to professor Glenn King of the University of Queensland, an expert on the composition of venoms.
Children with Dravet syndrome produce only half the NaV1.1 protein, so Petrou was looking for a molecule that could stimulate the NaV1.1 protein. King told him the molecule he was looking for could be found in the venom of a West African tarantula.
They used the Hm1a venom peptide to selectively activate the NaV1.1 in the brains of mice with Dravet syndrome. The Hm1a was found to hyper-stimulate the protein that is affected in those with Dravet syndrome.
“After applying the compound from the spider venom to nerve cells from the brains of Dravet mice we saw their activity immediately return to normal,” Petrou said in a statement. “Infusions into the brains of the Dravel mice not only restored normal brain function within minutes but over three days, we noted a dramatic reduction in seizures in the mice and increased survival. Every single untreated mouse died.”
While the study made progress in therapy for this form of epilepsy, there is still a significant way to translate this from mice to humans.
The report states that one of the biggest hurdles is that the Hm1a peptide is unable to pass through a human’s blood-brain barrier and so the delivery of the peptide would need to be invasively injected into the child’s spine.
Improvements on the tarantula’s peptide will need to be made before researchers can see if the venom could be used in curing Dravet syndrome in humans, but their breakthrough provides a bright spot of hope for the thousands of children suffering from the disease.