Imagine living with constant, unpredictable seizures that medications just can't control. That’s the reality for many people with epilepsy. But a new kind of treatment using stem cells might be about to change lives.
In a small but exciting trial in the U.S., two epilepsy patients received a one-time brain injection of specially grown brain cells. The results? Their seizures dropped dramatically — by over 90% — and the effects lasted for a whole year.
A New Kind of Treatment
The therapy is being developed by a company called Neurona Therapeutics in San Francisco. It works by injecting lab-grown brain cells directly into the area of the brain where seizures start. These cells are called "inhibitory neurons," and their job is to calm down the overactive brain signals that cause seizures.
In people with epilepsy, there's often an imbalance between brain cells that excite and those that quiet things down. Too much excitement leads to seizures. This new treatment tries to fix that by adding more calming cells to the mix.
Meet the Patients
The first patient in the trial had been dealing with epilepsy for seven years and was having around 32 seizures every month. The second had been living with seizures for nine years and experienced about 14 seizures monthly. Both had tried medications without success and were even considering brain surgery before joining the trial.
Instead, they chose to try this new stem cell therapy, called NRTX-1001.
And it paid off:
- The first patient saw their seizures go down by more than 95%. By the seventh month after the injection, they stopped having seizures altogether.
- The second patient had more than a 90% reduction in seizures.
- Even better, both patients showed no signs of memory loss or mental decline — a major concern with epilepsy treatments that affect the brain.
Why This Matters
Roughly one in three people with epilepsy don’t get enough relief from standard treatments like medication. Surgery is an option, but it comes with serious risks, like permanent memory or speech problems. That’s why a non-invasive, long-lasting solution like this could be a game changer.
The therapy is still in the early stages and more testing is needed, but experts are optimistic. Lab studies in animals have already shown that these injected cells can blend in with the brain’s natural circuits and keep seizures from coming back.
What the Experts Are Saying
Cory Nicholas, CEO of Neurona Therapeutics, called the early results “gratifying,” and hopes they’ll continue as more patients join the trial.
Peter Oliver, a professor at the University of Oxford who wasn’t involved in the research, said the results “suggest that delivering these inhibitory neurons can significantly reduce seizures.” He also pointed out that this could be a solid alternative for people who don’t respond to drugs or want to avoid brain surgery.
Maxine Smeaton, head of Epilepsy Research UK, said the findings are exciting — but also a reminder of how underfunded epilepsy research is. “Despite being one of the most common neurological conditions, only a tiny fraction of government health research money goes into epilepsy,” she said.
What’s Next?
The trial is continuing with more patients, and researchers are watching closely to see if the benefits last long-term and across a wider group of people.
For now, this small study gives real hope to those living with severe epilepsy — and opens the door to a future where seizures might no longer control their lives.
Source: https://clinicaltrials.gov/search?term=NCT05135091&viewType=Table
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