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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)
ES1 - Emerging Science 1 (11:51 AM-11:57 AM)
007

Implantation of human cortical-type GABAergic interneurons in the hippocampus of mice with kainate-induced mesiotemporal sclerosis can control focal seizures (Priest et al., 2021, AES poster 1.091), with over two-thirds of the cell-treated animals becoming seizure-free for the duration of the 9-month study without producing lethargy, memory deficits, or other dose-limiting toxicities. The interneuron cell therapy also reduced hippocampal damage and increased animal survival. Interneuron cell therapy offers a novel approach to the potential treatment of human focal epilepsy.

Investigate whether implantation of human GABAergic interneurons (NRTX-1001) can lead to seizure control in drug-resistant mesial temporal lobe epilepsy (MTLE).

This is a first-in-human Phase I/II clinical trial (NCT05135091). Subjects have unilateral MTLE with hippocampal sclerosis and focal seizures refractory to drug treatment. Testing includes EEG, imaging, tests of memory, mood, and assessment of visual fields. Subjects receive immunosuppression beginning 1 week prior to surgery tapering after 1 year. Cells are implanted via stereotactic injection along the long axis of the hippocampus with intra-operative MRI imaging.

Two subjects have been enrolled and had cell implantation. Data are reported as of 31Dec2022. There have been no serious adverse effects. Subject #1 is 6 months out from dosing. Baseline seizure frequency was 32/month; post-surgery, there has been a >90% seizure reduction and the subject has been free of focal awareness-impaired seizures since month 1. Subject #2 is 2 months out from dosing and has moved from a baseline seizure frequency of 14/month to one reported seizure since surgery.

This first-in-human study of NRTX-1001 GABAergic interneurons for focal epilepsy is underway, and preliminary results are encouraging.

Authors/Disclosures
Robert Beach, MD, PhD, FAAN (Upstate Medical University)
PRESENTER
The institution of Dr. Beach has received research support from Neurona.
David C. Spencer, MD, FAAN 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.
Harish Babu, MD,PhD Dr. Babu has nothing to disclose.
Kim Burchiel Kim Burchiel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurona Therapeutics. Kim Burchiel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ClearPoint Neuro. Kim Burchiel has received intellectual property interests from a discovery or technology relating to health care. Kim Burchiel has received publishing royalties from a publication relating to health care. Kim Burchiel has received personal compensation in the range of $500-$4,999 for serving as a Consultant with Harvard University.
Andrew Adler, PhD Dr. Adler has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Adler has stock in Neurona Therapeutics.
Gautam Banik, PhD (Neurona Therapeutics) Dr. Banik has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Banik has stock in Neurona Therapeutics.
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) Miss 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.
Ji-Hye Jung, PhD (Neurona Therapeutics) Dr. Jung has received personal compensation for serving as an employee of Neurona Therapeutics.
Rose D. Larios, PhD (Neurona Therapeutics) Dr. Larios has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Larios has stock in Neurona Therapeutics.
Seonok Lee, PhD (Neurona Therapeutics) Dr. Lee has received personal compensation for serving as an employee of Neurona Therapeutics.
Sheri Madrid (Neurona Therapeutics, Inc) Mrs. Madrid has received personal compensation for serving as an employee of Neurona Therapeutics, Inc. Mrs. Madrid has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Neurona Therapeutics, Inc.
Cory Nicholas (Neurona Therapeutics) Cory Nicholas has received personal compensation for serving as an employee of Neurona Therapeutics. Cory Nicholas has received stock or an ownership interest from Neurona Therapeutics. Cory Nicholas has received intellectual property interests from a discovery or technology relating to health care.
Catherine Priest (Neurona Therapeutics, Inc) Catherine Priest has received personal compensation for serving as an employee of Neurona Therapeutics. Catherine Priest has received stock or an ownership interest from Neurona Therapeutics.
Sergei Shevchuk, PhD (Neurona Therapeutics) Dr. Shevchuk has received personal compensation for serving as an employee of Neurona Therapeutics.