Peak Alpha Frequency as an EEG Biomarker for Angelman Syndrome
Michael Sidorov, PhD
Exciting scientific progress over the past ten years has led to clinical trials to treat Angelman syndrome at its root cause. Three current trials by different groups all use the same mechanism: antisense oligonucleotides that are designed to unsilence the dormant paternal UBE3A gene in the brain. As these trials begin, other promising treatment approaches are also in development. What all clinical trials have in common is the need to reliably measure clinically meaningful improvement as a result of treatment. If individuals with AS improve but this improvement cannot be quantified, a clinical trial will be deemed a failure and can set the community back years. Measurable biomarkers are needed to quantify improvement in clinical trials. Electroencephalography (EEG) meets many of the criteria of a good biomarker: (1) it is safe and non-invasive, (2) differences in AS are measurable and reliable, and (3) the degree of differences in AS is linked to the severity of AS symptoms. Current EEG biomarkers work well in children with AS, but their value wanes with age. With this proposal, we will develop new EEG biomarkers specifically designed for clinical trials in older children, adolescents, and adults with AS. Development of new biomarkers will ensure that improvement can be accurately measured during clinical trials in individuals of all ages.