NRTX-1001 Cell Therapy Shows Promise for Drug-Resistant Mesial Temporal Lobe Epilepsy


The single-administration allogeneic cell therapy has shown positive safety—and signs of efficacy—in preliminary data from the first 2 patients dosed with the Neurona Therapeutics’ product.

Alessandro Bulfone, MD, head of operations at Neurona Therapeutics

Alessandro Bulfone, MD

A version of this article originally appeared on our sister site, NeurologyLive.

Data from the ongoing and first-in-human phase 1/2 clinical trial (NCT05135091) of the investigational cell therapy NRTX-1011 in patients with drug-resistant mesial temporal lobe epilepsy (MTLE) suggest thus far that the treatment is safe.1 The Neurona Therapeutics' product has also shown some signs of efficacy, according to the data.

Presented at the 2023 American Academy of Neurology (AAN) Annual Meeting, April 22-27, in Boston, Massachusetts, by Alessandro Bulfone, MD, head of operations at Neurona Therapeutics, the update included information from 2 patients who had recieved treatment to this point.

The first was a 26-year-old man with a 9-year history of seizures and an average of 32 seizures per month in the 6 months prior to screening who was treated in June 2022 (Subject 1). He also reported right hippocampal onset of seizures and had experienced treatment failure with 4 antiseizure medications.

The second was a 59-year-old woman with an 8-year history of seizures and an average of 14 seizures per month in the 6 months prior to screening who was treated in October 2022 (Subject 2). She had a right hippocampal onset of seizures, and to date had experienced treatment failure with 3 to 4 antiseizure medications.

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The cells were well tolerated by Subject 1, without any serious or severe adverse events (AEs) occurring. Notably, the patient reported a reduction of seizures of more than 90% from the baseline values, and additional improvements on measures of spatial and verbal memory. The rate of focal aware seizures per 28 days dropped to 5 or fewer per month by 1 month post intervention; likewise, the rate of focal impaired seizures per 28 days dropped to none per month by 2 months post intervention.

In the case of Subject 2, the cells were also well tolerated, and in line with what was reported with Subject 1, there have been no serious or severe AEs. Additionally, her seizures have been reduced by more than 90% from baseline—focal aware seizures per 28 days dropped to a rate of none per month by 1 month post intervention, and focal impaired seizures per 28 days dropped to a rate of none per month by 4 months post intervention.

Bulfone explained that NRTX-1001 poses a potential option for patients who do not wish to undergo these procedures, as the allogeneic cell therapy poses promise as a nondestructive option for with TLE originating in the dominant or nondominant hippocampus. “The state of the art is the lobectomy and/or laser ablation, but in both cases, there is a 50% to 60% chance that the seizures are reduced by at least 60%, and obviously the surgery is very aggressive,” he said.

He added that the company had connected with the FDA on the treatment’s exploration in patients with bilateral temporal lobe epilepsy, as well. Bulfone also pointed to the success of prior preclinical work in this area, specifically work from 2021 that suggested that the implantation of human cortical-type GABAergic interneurons in the hippocampus of mice with kainate-induced mesiotemporal sclerosis can control focal seizures.2 More than two-thirds of the cell-treated animals were seizure-free for the duration of the 9-month study without producing lethargy, memory deficits, or other dose-limiting toxicities. The interneuron cell therapy also reduced hippocampal damage and increased animal survival.

The testing process for the ongoing phase 1/2 study includes electroencephalogram; imaging; the Rey Auditory Verbal Learning Test (RAVLT); and other tests of memory, mood, and visual fields. Patients received immunosuppression treatment 1 week prior to surgery, with tapering after 1 year, with NRTX-1001 cells were implanted via stereotactic injection along the long axis of the hippocampus with intraoperative MRI imaging.

1. Babu H, Beach R, Burchiel K, et al. First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Treatment of Focal Epilepsy - Emerging Clinical Trial Results. Presented at: AAN Annual Meeting; April 22-27, 2023; Boston, MA and Virtual.
2. Neurona Therapeutics Presents Preclinical Data from Lead Cell Therapy Candidate NRTX-1001 at Neuroscience 2021. News release. Neurona. November 8, 2021. Accessed May 8, 2023.
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