Nearly 3.5 million Americans live with epilepsy. Symptoms vary widely, from stiff muscles or staring spells to violent shaking and loss of consciousness.

The condition’s impact extends far beyond seizures. People with epilepsy face higher rates of depression, social isolation, school and employment challenges, limits on driving, and increased risk of early death.

Medication helps some, but about one‑third of patients do not respond. The alternative—brain surgery—is invasive and can damage delicate tissue.

Neurona Therapeutics has developed NRTX‑1001, a therapy made from a specialized type of neuron derived from embryonic stem cells. Researchers inject these cells into the seizure‑affected brain region, where they release a neurotransmitter that blocks the signals that trigger seizures.

First patients treated

So far, the first two patients treated in the groundbreaking clinical trial—both of whom entered the study with a history of significant monthly seizures that were not controlled by anti-seizure medications—have seen encouraging signs of reduction, which suggest that a single dose of NRTX-1001 may have a long-lasting ability to suppress seizures.

The first patient had a nine‑year history of seizures and averaged 32 seizures per month despite multiple medications. After receiving a single dose of NRTX‑1001, the treatment was well tolerated with no serious adverse events. The patient reported four seizures in the first three months post‑treatment.

The second patient also had drug‑resistant seizures, averaging 14 per month before treatment. After receiving NRTX‑1001, this patient experienced no serious adverse events—and no seizures—in the first week.

“The early clinical results with NRTX‑1001 are very encouraging, and we look forward to enrolling more patients,” said Dr. Cory Nicholas, Neurona’s president and CEO. “NRTX‑1001 is designed as an off‑the‑shelf, one‑time therapy that may durably eliminate seizures for patients who do not respond to medication.”

He added, “It may be disease‑modifying without the risks of lobectomy. Many patients who cannot undergo surgery may be eligible for NRTX‑1001. We are grateful to the study participants, their families, and the clinical teams.”

CIRM has invested more than $14 million across four awards to help move this therapy from early research into the current clinical trial.