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Good science takes time. That’s an essential guiding principle for researchers, but a frustrating reality for patients who need help now.

CIRM-funded epilepsy research

That point was clear when the CIRM Board voted to invest nearly $8 million to test a new approach for drug‑resistant epilepsy. The therapy shows promise, but it took time and effort to get here.

Epilepsy affects more than three million people in the U.S. More than one‑third have seizures that do not respond to medication. Their only options are surgery or laser ablation to remove the seizure‑causing brain region—procedures that can permanently affect memory, mood, and vision. Many patients understandably avoid them.

Specialized neurons

Neurona Therapeutics has developed a new approach called NRTX‑1001. The therapy consists of specialized neurons derived from embryonic stem cells. Researchers inject these cells into the seizure‑affected area of the brain, where they release a neurotransmitter that blocks the signals driving seizures. Preclinical studies suggest a single dose may suppress seizures long‑term.

Neurona CEO Cory Nicholas, PhD, says the therapy will be tested in people with drug‑resistant temporal lobe epilepsy, the most common form.

“To our knowledge, NRTX‑1001 is the first human cell therapy to enter clinical trials for epilepsy. It could offer a less invasive, non‑tissue‑destructive alternative for people with chronic focal seizures.

“Advocates and clinicians say a regenerative cell therapy like this could become a first option—possibly eliminating the need for surgical procedures. And for patients who cannot undergo surgery, it could be the only path to potential seizure‑freedom.”

From university researcher to company founder

Nicholas emphasizes that the work took decades. “This effort began in the early 2000s in the laboratories of John Rubenstein, Arturo Alvarez‑Buylla, and Arnold Kriegstein at UC San Francisco. They were among the first to uncover how inhibitory interneurons develop in the forebrain before birth, and they pioneered using these cells as therapy in preclinical models.”

Over time, the research team grew and eventually formed Neurona Therapeutics. CIRM backed their progress with several awards.

“CIRM provided the funding and expertise needed to translate our discoveries toward the clinic, using human embryonic stem cell technology to generate a sustainable supply of interneurons. Truly, CIRM has been the essential catalyst in accelerating this research from bench to bedside.”

For Nicholas, the work is deeply personal and profoundly meaningful.

“It’s emotional to reflect on this first‑in‑human trial. I think about our amazing team—Neurona employees, CIRM staff, clinicians, professors, trainees, collaborators, and investors—who have worked tirelessly to advance this mission. I am humbled by the responsibility to the brave patients in this study. We remain committed to patient safety and cautiously optimistic that NRTX‑1001 will improve life for people with chronic focal epilepsy.

“We are grateful to Californians for supporting CIRM’s vision, and proud to be part of this effort that places our state at the forefront of regenerative medicine.”