Neural Cell Therapy Reduce Seizures in MTLE


Updated data were presented at the AES annual meeting in Nashville.

Neurona Therapeutics’ regenerative cell therapy NRTX-1001 reduced seizures and was well-tolerated in participants with mesial temporal lobe epilepsy (MTLE) in an ongoing phase 1/2 clinical trial (NCT05135091). These data were presented at the annual meeting of the American Epilepsy Society, held in Nashville, Tennessee, December 2-6, 2022.1

“We are very encouraged by the early clinical data from our NRTX-1001 trial in people with drug-resistant focal epilepsy who have tolerated the treatment well and have reported reductions in seizure activity thus far,” David Blum, MD, chief medical officer,Neurona, said in a statement.1 “Currently, there are few effective therapeutic options for people with this type of epilepsy, including a lobectomy surgery to remove or ablate brain tissue, which can have tissue-destructive adverse effects on memory and vision. NRTX-1001 is a regenerative cell therapeutic with the potential to durably eliminate seizures after a single administration. We look forward to enrolling additional patients into the NRTX-1001 clinical trial and advancing this novel therapy for patients for whom anti-seizure medication has failed.”

Data from the first 2 patients treated in the trial were presented. The participants had entered the trial with a history of significant monthly seizure activity not controlled by anti-seizure medications. The first participant had a 9-year history of seizures with an average of 32 monthly seizures in the 6 months leading up to NRTX-1001 administration despite being on several antiepileptic medications. This patient’s seizures reduced to 4 in the first 3 months after treatment. The second patient had an average of 14 drug-resistant monthly seizures in the 6 months prior to treatment with 1 seizure in the first month after treatment. The updated findings follow-up on data announced earlier in November.2

WATCH NOW: Exploring Cell Therapy for the Treatment of Drug-Resistant Epilepsy

The trial is designed to evaluate the efficacy and safety of a single dose of NRTX-1001 in participants with drug-resistant MTLE in 2 phases.1 The first stage is an open-label dose-escalation phase in up to 10 participants, with 5 patients treated as a starting dose and 5 at a higher dose and the second expanding on these doses in up to 20 participants. The primary endpoint is the incidence and severity of adverse events and secondary endpoints include change in seizure frequency and responder rate.

NRTX-1001 is a regenerative neural cell therapy derived from human pluripotent stem cells. The therapy consists of interneurons that secrete gamma-aminobutyric acid to provide long-term GABAergic inhibition to repair hyper-excitable neural networks.

“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 an earlier statement.2 “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.”

1. Neurona Therapeutics presents clinical data for NRTX-1001 regenerative cell therapy for drug-resistant focal epilepsy at American Epilepsy Society Annual Meeting. News release. Neurona Therapeutics. December 5, 2022.
2. 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.
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