Neurona's Interneuron Cell Therapy Looks Promising in Drug-Resistant Mesial Temporal Lobe Epilepsy


The trial is recruiting 5 more participants for the highest dose cohort following a positive DSMB review.

NRTX-1001 interneuron cell therapy was well-tolerated and showed favorable effects on seizure control in 5 participants with drug-resistant mesial temporal lobe epilepsy treated in a first-in-human phase 1/2 clinical trial (NCT05135091).1

These data, current as of October 20, 2023, in the first 5 participants in the trial, were presented at the Annual Meeting of the American Epilepsy Society (AES), held December 1-5, 2023, in Orlando, Florida.

“The DSMB’s decision to allow the enrollment of five more subjects in our ongoing open-label Phase I/II clinical trial is a testament to NRTX-1001 being well-tolerated in the first cohort,” Cory R. Nicholas, PhD, chief executive officer, Neurona, said in a statement.2 “The early data from the first cohort in the trial have been very encouraging, with substantial, durable seizure reduction in the first subjects with drug-resistant MTLE. We look forward to advancing the development of NRTX-1001 cell therapy with our top-dose cohort of five subjects now enrolling into early 2024.”

The participants received 1 dose of NRTX-1001 along with 1 year of immunosuppression.The first 2 participants treated have continued to report over a 95% reduction in average monthly seizures 1 year after treatment. The first participant went from 32 seizures per month without focal impaired awareness seizures since the first month post-treatment and the second went from 14 to 0 focal impaired awareness seizures in the past 6 months. The first participant has tapered off immunosuppression and the second is currently tapering off, and these 2 have had no antibodies to the NRTX-1001 cells. They have also shown some improvements and no deterioration in modality-specific cognitive tests.1

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

Two of the 3 newer participants have demonstrated average total seizure reductions from baseline (64% and 75%) and the other participant, with 1 month of follow-up, has continued to experience variable seizure counts consistent to their disease history, but longer follow-up may elucidate the results.1

Investigators have observed mild to moderate adverse events (AEs) typical with immunosuppression. There have been no serious AEs related to the cell therapy, delivery, or immunosuppression. One participant, with a prior history, had an unrelated serious AE of a focal to bilateral tonic-clonic cluster of seizures (status epilepticus) and is stable. Based on these positive data from the first cohort, the data safety monitoring board (DSMB) recommended dosing to commence in the second, highest dose cohort, and 5 additional participants are being recruited.1

“Neurona’s regenerative cell therapy approach has the potential to provide a single-administration, non-destructive alternative for the treatment of drug-resistant MTLE,” David Blum, MD, chief medical officer, Neurona, added.2 “Currently, people with MTLE who are not responsive to anti-seizure medications have limited options, such as an invasive surgery that removes or destroys the affected brain tissue. FDA approval of novel tissue- and memory-sparing approaches could be transformative for such patients.”

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.
2. 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.
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