The Synapse Secret: How One Molecule Normalized Brain Activity in Fragile X Patients
In this episode of Power to the Patients, Brandon Li sits down with Dr. Craig Erickson, Research Director and Research Endowed Professor at Cincinnati Children’s, to explore how synaptic restoration is revolutionizing treatment for Fragile X, autism, ALS, Alzheimer's, and schizophrenia. From his decades of clinical experience and pioneering work at Spinogenix, Dr. Erickson shares the science behind restoring brain connectivity, the bold early results that surprised even experts, and why this approach could transform outcomes for patients with devastating neurological disorders.
What if the most effective way to treat some of the most devastating brain disorders isn’t by targeting symptoms in isolation, but by repairing the brain’s most fundamental unit of communication itself?
In this episode of Power to the Patients, host Brandon Li sits down with Dr. Craig Erickson, Research Director and Research Endowed Professor at Cincinnati Children’s and a leading authority on neurodevelopmental disorders, to explore how synaptic dysfunction serves as a unifying biological thread across conditions as diverse as Fragile X Syndrome, autism, schizophrenia, ALS, and Alzheimer’s disease.
Dr. Erickson shares how decades of failed large-scale neuroscience trials led him to rethink the drug development playbook - shifting from subjective clinical scales to objective, quantitative biomarkers like EEG and computer-based cognitive testing. Through his work with Spinogenix, he explains how early-phase trials can now detect true brain target engagement in small patient populations, dramatically reducing placebo effects, timelines, and patient burden.
The conversation dives deep into breakthrough Fragile X data, where a single dose of a synapse-targeting therapy normalized pathological brain activity - an effect stronger than anything previously observed in early trials. Dr. Erickson also unpacks why restoring synaptic connectivity can unlock functional gains that truly matter to patients and families, from reduced anxiety to greater independence and improved quality of life.
Whether you're a patient, caregiver, clinician, or investor seeking to understand the future of brain-targeted medicine, this conversation reveals how precision neuroscience is delivering hope where traditional approaches have failed.
What You'll Learn:
- Why synaptic dysfunction is a common biological endpoint across neurodevelopmental, neurodegenerative, and psychiatric disorders, despite vastly different underlying causes
- How objective biomarkers like EEG and computer-based testing can derisk early-phase clinical trials and outperform subjective symptom scales
- Why a single-dose normalization of gamma band activity in Fragile X represents one of the strongest early target engagement signals ever observed
- How patient stratification and biological enrichment dramatically improve signal detection in heterogeneous conditions like Fragile X and autism
- The difference between using biomarkers for trial derisking and inclusion criteria versus FDA-approvable endpoints and why that distinction matters
- How Spinogenix’s platform uniquely restores synaptic spine morphology, not just neurotransmitter signaling, enabling neurons to regain mature connections
- Why smaller, faster, highly quantitative trials may be the future of neuroscience drug development
- What patients, caregivers, and clinicians should watch for next as synapse-restoring therapies move into chronic dosing and later-stage studies
About the Guest:
Dr. Craig Erickson is a Research Director and Research Endowed Professor at Cincinnati Children’s. For more than 20 years, he has led translational research in neurodevelopmental disorders, focusing on early detection of brain target engagement and patient-specific treatment response in human drug trials. Dr. Erickson holds multiple investigator-initiated INDs, is an inventor on several therapeutic patents, and has received extensive support from the NIH, CDC, U.S. Department of Defense, foundations, and global biotechnology partners.
Episode Resources: