Gene therapy in Seattle offers hope for children with epilepsy

Breakthrough epilepsy gene therapy happening in Seattle

The team at the Allen Institute in Seattle, along with researchers at Seattle Children's Research Institute are bringing hope to families facing the devastation of Dravet Syndrome.

SEATTLE - A groundbreaking gene therapy being developed in Seattle could one day save the lives of children suffering from a debilitating form of epilepsy. The research, taking place at the Allen Institute for Brain Science, offers new hope to families fighting against Dravet Syndrome, a severe form of epilepsy that devastates childhood development.

Dravet Syndrome, usually starting in the first year of life, is a severe form of epilepsy that can severely hinder a child’s development or even cause death, often while children are sleeping.

John Mich, Senior Scientist at the Allen Institute, explains, "It is severe, long-term, and debilitating. In fact, it is so severe that up to 10-20% of children actually won't survive."

What we know:

Current treatments for Dravet Syndrome primarily involve oral medications, but these come with serious side effects and can affect the entire brain. "This is a key limitation for the way that drug development occurs in neurology today. The new technologies like the ones we are developing here really have a great potential impact to move the needle," says Mich.

A breakthrough: Gene therapy shows promise

A new gene therapy tested in mice has shown remarkable results: long-term recovery without any side effects. This gene therapy involves an injection directly into the brain—an invasive procedure, but one that’s only done once and targets the affected area.

Boaz Levi, Associate Investigator and Project Lead describes the research as the "most exciting project" of his career, adding, "I could not have anticipated a better outcome where we could completely rescue this disease."

Next steps for gene therapy

This years-long research project, recently published in Science Translational Medicine, represents a significant scientific success. Now, the team at the Allen Institute is preparing to take this gene therapy to the next phase, with the goal of eventually launching a clinical trial.

"It is a very long road. We are trying to interact with the families, patient advocacy groups which will help us design the best path forward because we really believe this is something that should be a therapy and get to the patient community," says Levi.

A ripple effect of innovation

The breakthrough happening in Seattle could soon have a positive ripple effect, not just in local communities, but across the world. Researchers and families alike are optimistic about the potential of this gene therapy to change lives on a global scale. This story of science and innovation is just one step closer to offering a life-saving cure for children battling Dravet Syndrome.

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