Autism Spectrum Disorder (ASD) affects approximately 1 in 36 children in the U.S., representing a rapidly growing and underserved population.

Despite advances in diagnosis, there remain no FDA-approved pharmacologic treatments targeting the core social and communication challenges of autism.

This need presents an opportunity for innovation and clinical impact through novel, evidence-based therapeutic approaches.

Autism Spectrum Disorder

Bar graph showing increasing autism rates in children in the US from 2000 to 2022.

Innovative Treatment Discovery —

Ethosuximide

Flowchart with three blue boxes and arrows connecting them. The first box states: "Serendipity: Dr. Niesen observed an anti-seizure drug, Ethosuximide, appear to reduce the severity of ASD in children." The second box states: "Patient Administration: Dr. Niesen monitored Ethosuximide's impact on ASD in treatment protocols with 30+ pediatric patients." The third box states: "Take-Aways: Dr. Niesen collected data on language acquisition, eye contact, and social engagement amongst these patients."

Ethosuximide’s Mechanism of Action

Diagram of a cell membrane showing ion transport, highlighting the movement of potassium, sodium, and calcium ions through various channels and pumps, with red X's indicating blocked channels.

Outline of a question mark with a check mark inside the lower right curve.

Illustration of a neuron showing an axon, dendrites, and myelin sheath.

Ethosuximide regulates neuronal excitability by reducing activity of voltage-dependent ion channels (T-type Ca 2+ channels, sustained Na+ channels, and Ca 2+-dependent K+ channels)

In an animal model T-type channel reduces autism spectrum disorder behavior

Ethosuximide’s Effect on EEG of Subjects with ASD

Electroencephalogram (EEG) recording with multiple brain wave channels in different colors, including blue, green, black, and red, showing brain activity patterns.

Sample EEG Recording

Theory of Autism Spectrum Disorder

A leading theory of Autism Spectrum Disorder (ASD) proposes that the condition arises from imbalanced connectivity between brain regions, disrupting how neural networks communicate and process information.

Ethosuximide may help restore this balance by selectively modulating specific neural connections.

Ethosuximide’s Effect

Using quantitative EEG analysis (spectral power, coherence, and entropy) we can detect these connectivity changes, providing objective biomarkers of therapeutic response.