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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
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)
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.    
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 R. Nicholas, PhD (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.
Harish Babu Harish Babu has nothing to disclose.
Robert Beach, MD, PhD, FAAN (Upstate Medical University) The institution of Dr. Beach has received research support from Neurona.
Sharona Ben-Haim No disclosure on file
Kim Burchiel Kim Burchiel has nothing to disclose.
Matthew W. Luedke, MD (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, MD (Rush University Medical Center) Dr. O'Dwyer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Pharma. Dr. O'Dwyer has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for SK Life Sciences.
Sepehr Sani The institution of Sepehr Sani has received research support from NIH. Sepehr Sani has received intellectual property interests from a discovery or technology relating to health care. Sepehr Sani has received publishing royalties from a publication relating to health care.
Jerry J. Shih, MD, FAAN (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 $500-$4,999 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.
Derek Southwell (Duke University) Derek Southwell has nothing to disclose.
David C. Spencer, MD, FAAN Dr. Spencer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jazz Pharmaceuiticals. 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 Expert Witness for Various. The institution of Dr. Spencer has received research support from NIH. 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 好色先生 Concepts.
Gautam Banik (Neurona Therapeutics) Gautam Banik has received personal compensation for serving as an employee of Neurona Therapeutics.
Marina Bershteyn (Neurona therapeutics) Marina Bershteyn has received personal compensation for serving as an employee of Neurona therapeutics. Marina Bershteyn has stock in Neurona therapeutics. Marina Bershteyn has received intellectual property interests from a discovery or technology relating to health care.
David E. Blum, MD Dr. Blum has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Blum has stock in Neurona Therapeutics.
Alessandro Bulfone, MD (Neurona Therapeutics) Dr. Bulfone has received personal compensation for serving as an employee of Neurona Therapeutics Inc.
Brianna Feld (Neurona Therapeutics) Brianna Feld has received personal compensation for serving as an employee of Neurona Therapeutics.
Holly Finefrock (Neurona Therapeutics) Ms. Finefrock has received personal compensation for serving as an employee of Neurona Therapeutics.
Luis Fuentealba (Neurona Therapeutics) Luis Fuentealba has nothing to disclose.
John D. Hixson, MD (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. Dr. Hixson has received personal compensation in the range of $5,000-$9,999 for serving as a Reviewer with Epilepsy Study Consortium.
Victoria Hosford (Neurona Therapeutics) No disclosure on file
Ji-Hye Jung (Neurona Therapeutics) Ji-Hye Jung has nothing to disclose.
Tia Kowal (Neurona Theraputics) Tia Kowal has received personal compensation for serving as an employee of Neurona Tharapeutics. Tia Kowal has received personal compensation in the range of $100,000-$499,999 for serving as a Scientist with Neurona Theraputics.
Sonja Kriks (Neurona Therapeutics) Sonja Kriks has received personal compensation for serving as an employee of Neurona Therapeutics. Sonja Kriks has stock in Neurona Therapeutics. Sonja Kriks has received intellectual property interests from a discovery or technology relating to health care.
Rose Larios, PhD (Neurona Therapeutics) Rose Larios has received personal compensation for serving as an employee of Neurona Therapeutics.
Seonok Lee (Neurona Therapeutics) Seonok Lee has received personal compensation for serving as an employee of Neurona Therapeutics.
Sheri Madrid (Neurona Therapeutics, Inc) No disclosure on file
Yves Maury (Neurona therapeutics) Yves Maury has received personal compensation for serving as an employee of Neurona Therapeutics. Yves Maury has stock in Neurona therapeutics.
Catherine Priest, PhD (Neurona Therapeutics, Inc) Catherine Priest has received personal compensation for serving as an employee of Neurona Therapeutics. Catherine Priest has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novo Nordisk. The institution of Catherine Priest has received research support from CIRM.
Kiril Shevchuk No disclosure on file
Sergei Shevchuk (Neurona Therapeutics) Sergei Shevchuk has received personal compensation for serving as an employee of Neurona Therapeutics.