Cory R. Nicholas, PhD, on Addressing Unmet Needs in Mesial Temporal Lobe Epilepsy With Cell Therapy


The cofounder and CEO of Neurona Therapeutics discussed NRTX-1001, the company’s allogeneic regenerative neural cell therapy.

“Neurona Therapeutics has developed a first cell therapy derived from human stem cells for a regenerative alternative to treat drug-resistant epilepsies where there's a clear origin of these seizures—what's called focal-onset epilepsy, where we know where the seizures start. The idea here is that NRTX-1001 is designed to put back the cells that are damaged in that part of the brain where the seizures start.”

Approximately 30% of patients with epilepsy have disease that is multidrug-resistant. This patient population currently has limited pathways for further treatment. Options can include neurostimulator devices, which do not result in seizure freedom for most patients; lobectomy procedures, which can be effective in reducing seizures, but because of the tissue destruction involved can carry risks such as memory loss, damage to vision, and personality changes; and laser ablation procedures, which are less invasive than standard lobectomies, but still involve brain tissue destruction and may have slightly reduced efficacy by comparison.

In an attempt to help address this great unmet need, Neurona Therapeutics is developing NRTX-1001, an investigational allogeneic regenerative neural cell therapy. NRTX-1001 is currently being evaluated in a phase 1/2 clinical trial (NCT05135091) for the treatment of drug-resistant mesial temporal lobe epilepsy. Early data from this study was recently presented at the 2023 American Epilepsy Society Annual Meeting, held December 1-5, in Orlando, Florida.

Shortly after the conference, CGTLive™ spoke with Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics and an assistant professor, adjunct, at University of California, San Francisco. Nicholas discussed the currently available treatment options for drug-resistant epilepsy and the unmet needs that remain. He then spoke about NRTX-1001, which is composed of neurons that secrete inhibitory neurotransmitter gamma-aminobutyric acid (GABA), emphasizing that the therapy is meant to provide a disease-modifying treatment option without the tissue destruction associated with lobectomy and laser ablation.

1. Neurona Therapeutics presents new clinical data from first cohort in ongoing phase I/II open-label trial of NRTX-1001 cell therapy for drug-resistant mesial temporal lobe epilepsy (MTLE). News release. Neurona Therapeutics. December 1, 2023. Accessed January 11, 2024.
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