Written by Holly Alyssa MacCormick
About 3 million adults in the United States have epilepsy, including roughly 400,000 in California, according to the CDC. About one-third to one-half continue to have seizures that do not respond to available medications.
Hope for those with epilepsy
Neurona Therapeutics aims to help these patients with a stem cell–derived therapy now in clinical trials.
Cory R. Nicholas, PhD, CEO and co-founder of Neurona Therapeutics, recently shared an update on the CIRM-funded trials during a presentation to the agency’s governing board as part of the Closer to Cures speaker series.
Closer to Cures
For people whose seizures do not improve with drugs, the main treatment options include lobectomies—removing or ablating part of the brain—or implanting a brain stimulator. These approaches can reduce seizures, but they do not always work. They are invasive and can cause serious side effects, including memory loss, cognitive impairment, and personality changes.
“Seizures come in different forms, and they can strike at any time,” Nicholas said. “Some people have them daily; others may have one or two a month. Even if seizures occur only a small fraction of the time, people spend the rest of their lives worrying about when the next one will hit.”
Epilepsy deeply affects patients and their families. Unpredictable seizures often force people to give up driving, jobs, and hobbies, while family members take on caregiving roles.
“People often misunderstand epilepsy as a nuisance managed by medication, rather than the severe, life-altering condition it is for many,” Nicholas said.
Rewiring the brain
CIRM funded research at the University of California, San Francisco starting in the early 2000s and later at Neurona Therapeutics led to a series of discoveries related to maturing human embryonic stem cells into brain cells that calm surrounding neurons.
The thought was that implanting these calming cells near a seizure-inducing, overly excited part of the brain could reduce seizures. Neurona calls these cells NRTX-1001, and they are currently testing the cells in people with epilepsy whose seizures don’t respond to drugs. One trial includes people with seizures coming from just one side of the brain, and the other includes people with seizures on both sides of the brain. Preliminary results from these clinical trials show that NRTX-1001 has minimal side effects, and patients have fewer seizures.
Some of the patients treated are now seizure-free.
Although the trials are not yet complete and results are preliminary, both the FDA and the European Medicines Agency (EMA) have recognized the potential of this therapy and have granted it a special designation that paves the way for future clinical trials, including a double-blind phase 3 trial slated to start in late 2026.
For some patients participating in these clinical trials, NRTX-1001 regenerative cell therapy has been the difference between having seizures nearly every day, to being seizure-free and being able to return to work.
“Just maybe we can get a cure for epilepsy,” said one of the patients in a video Nicholas showed during his presentation.
Closer to Cures is a speaker series that features presentations from two CIRM-funded grantees at each of the California Institute for Regenerative Medicine’s governing board meetings, known as the Independent Citizens Oversight Committee (ICOC). The ICOC meetings and this speaker series are open to the public and are livestreamed via the CIRM official YouTube channel.
Related posts
• A conversation with Neurona’s CEO Cory Nicholas