NRTX-1001 Reduces Mesial Temporal Lobe Epilepsy Seizures in 2 Patients


The first participant has been followed-up for 3 months so far after treatment while the second is only 1 week post-treatment.

The neural cell therapy NRTX-1001 was well-tolerated and reduced seizures in the first 2 patients with drug-resistant unilateral mesial temporal lobe epilepsy (MTLE) treated in a first-in-human phase 1/2 study (NCT05135091).1

“The early clinical results with NRTX-1001 in epilepsy are very encouraging, and we look forward to enrolling additional patients in the study,” Cory R. Nicholas, PhD, president and chief executive officer, Neurona Therapeutics, said in a statement.1 “NRTX-1001 is designed to be an off-the-shelf, one-time administration therapy with the potential to durably eliminate seizures and provide a new regenerative cell therapeutic approach in patients for whom anti-seizure medication has failed. It has the potential to be disease-modifying without the tissue-destructive procedural risks associated with lobectomy. Further, there are many who are not currently eligible for lobectomy surgery who may be eligible for NRTX-1001 in the future. We are sincerely grateful to everyone involved in the development of NRTX-1001, including the first participants in this pioneering study, their families, and the respective clinical site teams.”

The patients had up to a 9-year history of seizures and, in the 6 months prior to NRTX-1001 treatment, had averages of 32 and 14 seizures a month while on antiepileptic medications. Both patients received the single dose of NRTX-1001 without any treatment-related serious adverse events (AEs). One patient has had 4 seizures in the first 3 months after treatment and the other has not had any seizures as of the first week after treatment. These data were presented at the ISSCR Boston International Symposium: Translating Pluripotent Stem Cell Discoveries to the Clinic: Preclinical, Manufacturing, and Regulatory Strategies for Success on November 19, 2022, by Nicholas.

READ MORE: Upstaza Approved for AADC Deficiency in UK

NRTX-1001 is designed to repair neural networks by delivering human interneurons that provide long-term secretion of the gamma-aminobutyric acid (GABA) inhibitory neurotransmitter. Participants in the study receive a single stereotactic intracerebral administration of the interneuron cells and remain on a immunosuppressive regimen for 1 year. Evidence of neural cell viability and local inflammation will be assessed using MRI scans for 2 years following the transplant.The single-arm, dose-escalation first stage of the study will enroll up to 10 participants with MTLE and the second will enroll up to 20 to be treated and 10 for a sham comparator arm.

The study is primarily assessing the incidence and severity of AEs over 1 year after NRTX-1001 administration compared to sham treatment. Secondary end points include the change in seizure frequency and the difference in the 75% responder rate between participants who received NRTX-1001 in stage 2 and those who received the sham treatment. The first patient was dosed in the trial in July 2022.2

“Neurona’s regenerative cell therapy approach has the potential to provide a single-administration, non-destructive alternative for the treatment of drug-resistant focal epilepsy,” Harish Babu, MD, PhD, assistant professor of neurosurgery, SUNY Upstate Medical University and the surgeon who administered the first dose of NRTX-1001, said in an earlier statement.2 “Currently, people with mesial temporal lobe epilepsy who are not responsive to anti-seizure medications have few options, such as an invasive surgery that removes or destroys the affected brain tissue.”

1. Neurona Therapeutics presents encouraging clinical data from first-ever regenerative human cell therapy trial in adults with drug-resistant focal epilepsy and shows supporting manufacturing, delivery, and preclinical data. News release. Neurona Therapeutics. November 21, 2022.
2. Neurona Therapeutics announces initial subject dosed in first clinical trial of regenerative human cell therapy, NRTX-1001, in adults with drug-resistant focal epilepsy. News release. Neurona Therapeutics. June 29, 2022. 
Recent Videos
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Ignacio Mata, PhD, an associate professor of neurology at the Cleveland Clinic Lerner Institute
Subhash Tripathi, PhD, on Developing Safe, Specific Engineered Treg Cell Therapy
Ignacio Mata, PhD, an associate professor of neurology at the Cleveland Clinic Lerner Institute
Sharif Tabebordbar, PhD, on Improving In Vivo Gene Editing for DMD
Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine
© 2024 MJH Life Sciences

All rights reserved.