Studying Nerve Cell Therapy NRTX-1001 to Treat Mesial Temporal Lobe Epilepsy: Jonathan Parker, MD, PhD

WATCH TIME: 3 minutes

"Looking from the outside in and what's gone into preparing the cell type, it makes a lot of sense to me that this is a strategy that makes sense, because if we don’t have those assurances from the preclinical data... then that’s obviously concerning."

GABA (gamma-aminobutyric acid) is the primary inhibitory neurotransmitter in the central nervous system, playing a critical role in maintaining the balance between excitation and inhibition in the brain. This balance is vital for normal brain function, and disturbances in GABAergic signaling are closely linked to epilepsy. NRTX-1001, an investigational drug in development for drug-resistant unilateral mesial temporal lobe epilepsy, is made from human stem cells that have been developed into a non-dividing type of nerve cell called an interneuron, which releases GABA.

This unique and innovative therapy is currently being tested in a phase 1/2 trial (NCT05135091), dubbed NTE001, that includes 2 stages. The first stage is an open-label, dose-escalation study, with half of the cohort treated at a starting dose and the other half treated with a higher dose. Developed by Neurona, NRTX-1001 represents a novel strategy to replace lost interneurons in the hippocampus, leveraging embryotic stem cells driven toward a specific, well-characterized interneuron subtype.

During the 2024 American Epilepsy Society (AES) Annual Meeting, held December 6-10, in Los Angeles, California, NeurologyLive® sat down with Jonathan Parker, MD, PhD, an epilepsy expert who runs one of the study’s sites, to discuss more about the agent. In the discussion, Parker outlined the rigorous preclinical work that has gone into enrusring these cells remain stable, do not revert to pluripotency, and avoid unintended effects like excitatory neuronal behavior. Furthermore, Parker, an assistant professor of neurosurgery and neuroscience at Mayo Clinic Arizona, provided an overview of how the study is conducted and expressed optimism about advances in cell therapies, particularly autologous approaches that could eliminate the need for immunosuppression.

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