Neurona Therapeutics reported encouraging data from the first two patients dosed with its regenerative stem cell therapy for epilepsy.

One year after receiving a single dose of NRTX-1001, the first patient in a Phase I/II trial (NCT05135091) reported a more than 95% reduction in seizure frequency, meeting the prespecified efficacy endpoint. Meanwhile, a second patient experienced a more than 90% reduction in seizure frequency seven months after their dose.

Both patients have drug-resistant mesial temporal lobe epilepsy (MLTE), and the first patient had experienced an average of 32 seizures per month leading up to the study. Neither subject has reported a serious adverse event.

Though the data is so far limited to just two patients, it adds to the growing hype surrounding stem cell therapies in neurological disorders. Pharma companies are already testing stem cell therapies in Alzheimer’s disease and Parkinson’s disease, and BrainStorm Cell Therapeutics is seeking regulatory approval for its stem cell therapy in amyotrophic lateral sclerosis (ALS).

Neurona’s NRTX-1001 is a regenerative stem cell therapy designed to deliver neural cells that secrete the neurotransmitter gamma-aminobutyric acid (GABA), silencing seizure activity in the epileptic region of the brain. NRTX-1001 is derived from human pluripotent stem cells, which are manufactured in-house by Neurona.

Neurona’s stem cell therapy in epilepsy

San Francisco, California-based Neurona is looking to bolster strong early data by actively recruiting new patients with MLTE. The Phase I/II trial will enroll up to 10 patients in the initial dose-escalation stage and up to 40 patients in total.

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Approximately 50 million people globally experience epilepsy, and up to one-third of people do not respond to existing therapies. Though many available epilepsy treatments target seizure symptoms, researchers and patients are pushing for more holistic treatment approaches to the potentially fatal condition.

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