Patient recruitment for the clinical trial is currently underway at epilepsy centers throughout the US.
Neurona Therapeutics’ phase 1/2 clinical trial (NCT05135091) for NRTX-1001, a regenerative neural cell therapy derived from human pluripotent stem cells, has dosed its first patient with Mesial Temporal Lobe Epilepsy (MTLE).1
“Administration of NRTX-1001 to the first patient in our clinical trial for MTLE represents a huge milestone for Neurona and the neurology field,” Cory R. Nicholas, PhD, Neurona’s president and chief executive officer said in a statement.1 “This promising program is the result of many years of work, dedication, and innovation by the Neurona team and its collaborators as well as key funding secured by grants received from the California Institute for Regenerative Medicine. We are very excited to take this next step, bringing a first-in-class, novel regenerative treatment to people living with chronic focal seizures. I would like to thank the excellent team at SUNY Upstate Medical University who treated the first patient, as well as our other clinical sites across the country who are part of this multicenter study.”
NRTX-1001 delivers human interneurons that provide long-term secretion of gamma-aminobutyric acid (GABA), an inhibitory neurotransmitter, to repair neural networks. In the 2-stage clinical trial, participants will receive a single stereotactic intracerebral administration of the interneurons and afterwards take an immunosuppressive regimen for 1 year. Evidence of neural cell viability and local inflammation will be assessed using MRI scans for 2 years following the transplant.
The trial is open to male and female patients aged 18 to 55 who have focal seizures clinically defined as TLE and have not achieved seizure control after adequate trials of at least 2 different anti-seizure drugs (ASD) at appropriate doses. Patients must additionally have been on stable doses of approved ASDs for at least 1 month. Patients who have epilepsy due to other and/or progressive neurologic disease, have significant other medical conditions which would impair safe participation, have a primary or secondary immunodeficiency, have had a suicide attempt in the past year, or are currently pregnant or breastfeeding will be excluded from the study.
The first stage of the study is an open-label, single-arm sequential dose escalation and will enroll up to 10 participants with MTLE. The second stage will enroll up to 20 participants in an experimental arm and up to 10 participants in a sham comparator arm. The primary end point will measure the frequency of serious or severe adverse events over 1 year after administration and will compare participants from both stages who received NRTX-1001 to those who received the sham treatment. Secondary end points include the change in seizure frequency and the difference in the 75% responder rate between participants who received NRTX-1001 in stage 2 and those who received the sham treatment.
“Neurona’s regenerative cell therapy approach has the potential to provide a single-administration, non-destructive alternative for the treatment of drug-resistant focal epilepsy,” Harish Babu, MD, PhD, assistant professor of neurosurgery, SUNY Upstate Medical University and the surgeon who administered the first dose of NRTX-1001, added to the statement.1 “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.”
Patient recruitment for the clinical trial is currently underway at epilepsy centers in the United States and the trial is estimated to be completed in May 2026.