AAN 2026 | NRTX-1001 restores inhibitory signaling in temporal lobe epilepsy

Yeah, this is a trial that uses not stem cells, which is important to note, but mature interneurons that produce an inhibitory neurotransmitter, GABA, and the whole biological concept behind it is to restore the balance between excitatory and inhibitory impulses in the epileptogenic focus, which is now driven predominantly by excitatory impulses. So by having these cells transplanted into the focus, and in this study, it’s entirely limited to temporal mesial epilepsy and hippocampal sclerosis. Our trial was in bilateral hippocampal epilepsy. So we inject these cells into the hippocampus, into the hippocampal long axis, stereotactically, and then follow these patients in a very rigid protocol. So the outcomes so far have been in both unilateral and bilateral, a significant seizure reduction and with one patient actually with bilateral disease completely seizure-free since 14 months already. So all these patients will undergo a 12-month immunosuppression because these are cells from a cell bank. After that, the immune suppression then stops. There were no serious adverse events at all, and the mild adverse events were predominantly related just to the immune suppression, known side effects of that. So it’s a very, very safe treatment.

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