Epilepsy has challenged people for as long as we have recorded history. A 4,000‑year‑old Akkadian tablet from Mesopotamia describes a person with stiff muscles, wide eyes, frothing at the mouth, and no awareness—an early description of a seizure.
Early treatments
Despite this long history, effective treatments took centuries to emerge. In the mid‑19th century, physicians began using bromides, but the drugs caused severe side effects, including depression, fatigue, and even coma.
Fast forward 150 years, and we may be entering a new era. This week, Neurona Therapeutics announced it has dosed the first patient in its first‑in‑human clinical trial for mesial temporal lobe epilepsy, the most common form of focal epilepsy in adults. The trial targets people whose seizures do not respond to medication.
Cell therapy
Neurona’s therapy, NRTX‑1001, uses specialized neurons derived from embryonic stem cells. Researchers inject these cells into the seizure‑affected brain region, where they release a neurotransmitter that blocks signals that trigger seizures. Preclinical data suggest a single dose may provide long‑lasting seizure suppression.
A new approach is urgently needed. Current treatments for drug‑resistant epilepsy are only partly effective, and one option—removing the affected brain region—can cause irreversible problems with memory, mood, and vision.
CIRM has a vested interest in this therapy’s success. The agency has invested more than $14 million across four awards to move this work from basic research into clinical testing.
The first dose
In a news release, two clinicians who administered the first dose called the milestone an important step forward.
Harish Babu, M.D., Ph.D., assistant professor of neurosurgery at SUNY Upstate Medical University, said, “Neurona’s regenerative therapy could offer a single‑dose, non‑destructive option for drug‑resistant focal epilepsy. Today, patients with mesial temporal lobe epilepsy who don’t respond to medication have very few choices, often facing invasive surgery that removes or destroys brain tissue.”
Robert Beach, M.D., Ph.D., professor of neurology at SUNY Upstate, added, “NRTX‑1001 is designed to add cells that can repair damaged circuits and reduce seizure activity.”
The unmet medical need is enormous. Around three million Americans have epilepsy, and 25–35 percent continue to experience seizures despite dozens of approved drugs.
If this therapy succeeds, that 4,000‑year‑old tablet may become a medical footnote—rather than a reminder of how far we still have to go.
