Cory R. Nicholas, PhD, on Future Plans for Additional Research With Epilepsy Cell Therapy NRTX-1001


The cofounder and CEO of Neurona Therapeutics discussed the company’s goals to evaluate the cell therapy in more types of epilepsy and potentially even in Alzheimer disease.

This is the third part of an interview with Cory R. Nicholas, PhD. For the second part, click here.

“We would be very interested in the future—if this therapy proves safe for epilepsy—to offer this to people who have the epileptic-type hyperactivity of their neural circuits in this part of the brain on a background of Alzheimer disease...”

Neurona Therapeutics is currently engaged in a first-in-human phase 1/2 clinical trial (NCT05135091) assessing NRTX-1001, its investigational allogeneic regenerative neural cell therapy, in patients with drug-resistant mesial temporal lobe epilepsy (MTLE). Although, the patient population eligible for this trial may only be the beginning for this cell therapy, as the company expects that it could eventually be used to treat additional types of epilepsy, and even another neurological indication altogether: Alzheimer disease (AD).

Following the recent presentation of early positive results from the current phase 1/2 study at the Annual Meeting of the American Epilepsy Society (AES), held December 1-5, 2023, in Orlando, Florida, 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. After going over the unmet needs that NRTX-1001 is meant to address and the signs of efficacy and safety in the early data, Nicholas discussed the future plans for the cell therapy. He noted that the inclusion criteria for the current trial only covers patients with MTLE in 1 temporal lobe, but that the company expects that NRTX-1001 may be able to provide benefit to patients with other types of epilepsy such as bilateral TLE and epilepsy related to focal cortical dysplasias. As such Neurona Therapeutics plans to carry out future studies in these additional indications, pending regulatory clearance to do so.

Nicholas also pointed out that the company is still seeking more centers to act as trial sites in the current study and that the trial is still recruiting new patients. He concluded by highlighting the potential of NRTX-1001 to treat patients with AD who have an epileptic pathophysiology as well and stated that Neurona will pursue the evaluation of NRTX-1001 for this purpose in the years to come.

1. Blum D, Babu H, Beach R, et al. First-in-human study of NRTX-1001 GABAergic interneuron cell therapy for treatment of focal epilepsy - emerging clinical trial results. Presented at: AES Annual Meeting; December 1-5; Orlando, Florida.
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