Neurona Therapeutics has achieved a significant milestone in epilepsy treatment by dosing the first patient in a Phase 1/2 clinical trial of NRTX-1001, a novel regenerative neural cell therapy for drug-resistant mesial temporal lobe epilepsy (MTLE). The groundbreaking treatment represents the first-in-human study of this regenerative approach, addressing a critical unmet medical need for the estimated 3 million Americans with epilepsy, of whom 25% to 35% continue to experience seizures despite dozens of approved medications.
Revolutionary Cell Therapy Approach
NRTX-1001 is a regenerative neural cell therapy derived from human pluripotent stem cells that have been differentiated into stable interneurons. These specialized cells secrete gamma-aminobutyric acid (GABA), a key inhibitory neurotransmitter designed to repair hyper-excitable neural networks associated with MTLE, the most common form of focal epilepsy in adults. The therapy is delivered as a single dose directly into the brain, where the human interneurons are intended to integrate and provide long-term GABAergic inhibition.
"Administration of NRTX-1001 to the first patient in our clinical trial for MTLE represents a huge milestone for Neurona and the neurology field," said Cory R. Nicholas, PhD, Neurona's President and Chief Executive Officer. "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."
Dramatic Early Results
The initial results from the first patient treated at SUNY Upstate Medical University have exceeded expectations. According to Robert Beach, MD, PhD, Professor of Neurology and principal investigator of the trial at Upstate, "Our first patient went from roughly 30 seizures a month down to one or two a month." Dr. Beach, who directs the Comprehensive Epilepsy Program, described the improvement as "very remarkable, very unusual to see such a dramatic improvement."
The first dose was administered by Harish Babu, MD, PhD, Assistant Professor of Neurosurgery at SUNY Upstate Medical University, who noted that "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."
Addressing Current Treatment Limitations
Current treatment options for patients with drug-resistant MTLE are limited and often involve significant risks. As Dr. Beach explains, "Epilepsy is one of the most common neurologic diseases, and about half of patients with epilepsy cannot get full control with medication." The standard treatment for uncontrolled temporal lobe epilepsy is a temporal lobectomy, which involves removing the hippocampus and part of the anterior temporal lobe.
While surgical intervention can reduce seizures by approximately 70-80%, it carries substantial risks. "If surgery is done on the dominant hemisphere, there's usually some change in language function," Dr. Beach noted. "Some patients have seizures coming from both sides, and if you perform the surgery on both sides the patient loses all their short-term memory."
Clinical Trial Design and Support
The multicenter Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. The first stage 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.
The trial is supported by an $8 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355), and patient recruitment is underway at epilepsy centers across the United States. The study can be found on clinicaltrials.gov under identifier NCT05135091.
Scientific Recognition and Future Prospects
The promising initial results have garnered significant attention in the medical community. The paper detailing the initial results was featured at the annual meeting of the American Academy of Neurology, the largest meeting of neurologists. "It was clearly recognized that this is a novel and important study," said Dr. Beach.
Looking ahead, researchers are optimistic about the potential for even greater efficacy. "The next question is whether we can actually get seizure freedom," Dr. Beach explains. "I think with the second step, which is a higher dose, that could possibly happen."
The treatment's impact extends beyond seizure reduction, with research coordinator Lena Deb observing significant quality of life improvements: "Meeting them over a year ago and seeing how they were and their quality of life, and then to see where they're at now; they're a lot happier. They're more social, they're out doing things that they couldn't do previously."
