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Abstract Details

First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Treatment of Focal Epilepsy - Emerging Clinical Trial Results
Epilepsy/Clinical Neurophysiology (EEG)
S19 - Epilepsy Clinical Trials and Long-term Studies (3:42 PM-3:54 PM)
002
NRTX-1001 is an investigational GABAergic interneuron product derived from human pluripotent stem cells. In pre-clinical testing, hippocampal transplantation of NRTX-1001 improved spontaneous seizure phenotypes in a mouse kainic acid model of focal epilepsy with hippocampal sclerosis (Bershteyn et.al., Cell Stem Cell 30:1-20, 2023). Between June, 2022 and August, 2023, 5 subjects were enrolled in an ongoing open-label study of NRTX-1001 transplantation.    
To present the early results of a first-in-human clinical study of allogeneic GABAergic interneuron transplantation for unilateral drug-resistant temporal lobe epilepsy (TLE; NCT05135091)
Study subjects are adults with drug-resistant, unilateral TLE with mesial temporal sclerosis. NRTX-1001 cells were surgically transplanted into the head and body of the diseased hippocampus using intra-operative MRI. Immunosuppression was initiated 1 week prior to NRTX-1001 transplantation and will be tapered after 1 year. Study endpoints are safety (primary endpoint) and seizure frequency (secondary) at 1 year post-implant. Other endpoints include assessments of EEG, neuroimaging, cognitive performance, and visual fields.
As of October 2023, subjects are 15, 11, 2, 1, and 1 months post-treatment (MPT) with NRTX-1001. There have been no serious adverse events. Subject #1 (15 MPT) has experienced >90% seizure reduction and has been free of awareness-impaired seizures since 1 MPT. Subject #2 (11 MPT) has also experienced >90% seizure reduction, with >50% reduction in awareness-impaired seizures. Both subjects have exhibited quantitative improvements in select measures of memory function.  Updated results for all subjects will be presented at the meeting.
NRTX-1001 transplantation was safely performed in 5 patients with drug-resistant TLE. Preliminary results at approximately 1 year (2 subjects) suggest NRTX-1001 transplantation may improve seizure control and memory dysfunction. In contrast to current surgical treatments (e.g., resection and laser ablation), NRTX-1001 could provide a non-destructive and functionally restorative approach to treating focal epilepsy.  
Authors/Disclosures
Cory Nicholas (Neurona Therapeutics)
PRESENTER
Dr. Nicholas has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Nicholas has stock in Neurona Therapeutics. Dr. Nicholas has received intellectual property interests from a discovery or technology relating to health care.
No disclosure on file
Robert Beach (Upstate Medical University) The institution of Dr. Beach has received research support from Neurona.
No disclosure on file
No disclosure on file
Matthew Luedke (Duke University Hospital Dept. of Neurology) Dr. Luedke has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bright Health. Dr. Luedke has received personal compensation in the range of $0-$499 for serving as an Expert Witness for Guidepoint. The institution of Dr. Luedke has received research support from US NAVY. The institution of Dr. Luedke has received research support from Neurona. The institution of Dr. Luedke has received research support from Xenon.
Rebecca O'Dwyer (Rush University Medical Center) Dr. O'Dwyer has nothing to disclose.
No disclosure on file
Jerry Shih (UC San Diego Health) Dr. Shih has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurelis. Dr. Shih has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Neurelis. Dr. Shih has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for SK Life. Dr. Shih has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sunovion. The institution of Dr. Shih has received research support from Neurona. The institution of Dr. Shih has received research support from NCGS, Inc. Dr. Shih has a non-compensated relationship as a Test Writing Committee Member with ABPN that is relevant to AAN interests or activities.
No disclosure on file
David Spencer Dr. Spencer has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for NeuroPace Inc. Dr. Spencer has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for AAN. Dr. Spencer has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Various. Dr. Spencer has received publishing royalties from a publication relating to health care. Dr. Spencer has received personal compensation in the range of $500-$4,999 for serving as a Faculty in Teaching Course supported by unrestricted educational grants with Creative Education Concepts.
No disclosure on file
No disclosure on file
David Blum Dr. Blum has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Blum has stock in Neurona Therapeutics.
Alessandro Bulfone (Neurona Therapeutics) Dr. Bulfone has received personal compensation for serving as an employee of Neurona Therapeutics Inc.
No disclosure on file
Holly Finefrock (Neurona Therapeutics) Ms. Finefrock has received personal compensation for serving as an employee of Neurona Therapeutics.
No disclosure on file
John Hixson (Neurona Therapeutics) Dr. Hixson has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Hixson has received personal compensation for serving as an employee of Nile Ai. Dr. Hixson has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for NextSense Inc.. Dr. Hixson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Seer Medical. Dr. Hixson has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Expert Institute.
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file