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Upstate doses first patient in national clinical trial of regenerative human cell therapy for treatment of epilepsy

Clinicians at Upstate Medical University have dosed the first patient in a national clinical trial—the first-ever regenerative human cell therapy, NRTX-1001, delivered as a single dose for the treatment of epilepsy.

The clinical trial is sponsored by Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative neural cell therapies for the treatment of neurological disorders.

“This Neurona regenerative cell therapy approach has the potential to provide a single-administration, non-destructive alternative for the treatment of drug-resistant focal epilepsy,” said Harish Babu, MD, PhD, assistant professor of neurosurgery at Upstate and the surgeon who administered the first dose of NRTX-1001.

“Currently, people with mesial temporal lobe epilepsy who are not responsive to anti-seizure medications have few options, such as an invasive surgery that removes or destroys the affected brain tissue,” Babu said.

The principal investigator of the study at Upstate is Robert Beach, MD, PhD, chief of epilepsy and professor of neurology, who likes what the future might hold for the trial.

“The objective of NRTX-1001 is to add cells that have the potential to repair the circuits that are damaged in epilepsy and thus reduce seizure activity,” Beach said. “We are delighted to work with the Neurona team to evaluate the potential of this exciting new therapeutic approach.”

Upstate’s Neurological Institute is one of several sites in the United States taking part in Neurona’s clinical trial.

The Upstate Neurological Institute brings together the departments of Neurosurgery and Neurology, creating multidisciplinary teams of experts to provide the best patient care and treatment options. The institute offer the region’s only Level-4 epilepsy program, the recently expanded NeuroICU, newly created neuro-oncology program at the Cancer Center. The institute totals 58 faculty, including both clinical and research members.

The neurology residency program has expanded to 9 positions per year, and neurosurgery residency program alternating one resident and two residents every other year, as well as the opportunities for fellowships.

The institute’s clinical research section conducts numerous clinical trials, such as the Neurona Therapeutics trial highlighted above. The institute belongs to the NIH StrokeNet and NeuroNEXT networks and has several NIH research grants as well as a DOD grant.

About Neurona’s Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE)

Neurona’s multicenter, Phase 1/2 clinical trial is designed to evaluate the safety and signs of efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. The first stage of the trial is an open-label dose-escalation study in up to 10 people with MTLE. Patients treated with a single infusion of NRTX-1001 cells will be monitored for safety, tolerability, neural cell viability, and effects on their epilepsy disease symptoms. Patient recruitment is underway at Upstate and other epilepsy centers across the United States. For more information, please visit

www.clinicaltrials.gov (NCT05135091).


Caption: Key faculty in the trial are Harish Babu, MD, PhD, assistant professor of neurosurgery at Upstate, and principal investigator of the study at Upstate Robert Beach, MD, PhD, chief of epilepsy  and professor of neurology.