A brain injection filled with lab-grown neurons promises to cure epilepsy

Phase-1 trials of a brain injection achieve over 90 percent reduction in seizures in two patients.
Rupendra Brahambhatt
An illustration of neurons
An illustration of neurons

Mohammed Haneefa Nizamudeen/iStock 

Researchers have achieved over 90 percent reduction in seizures experienced by two epilepsy patients using a brain injection. 

Each patient received a dose of NRTX-1001, an experimental cell therapy treatment developed by researchers at San Francisco-based biotech company Neurona Therapeutics.

Before the therapy, the patients had 32 and 14 seizures on average per month, respectively. No anti-epileptic medicines worked for them; highly invasive surgical treatment was their last hope. 

“Both patients entered the clinical trial with significant seizure activity, impaired cognition, and suboptimal quality of life. They were candidates for lobectomy or ablation surgery to remove the epileptic seizure-generating temporal lobe, albeit with an associated risk of causing further, irreversible cognitive deficits. Instead, they courageously chose to be the first to receive NRTX-1001 cell therapy,” said Cory Nicholas, CEO of Neurona Therapeutics.  

Their condition has drastically improved in the last year, and it seems like they made the right choice so far. The cell therapy has also raised hopes for millions of other patients whose lives are severely affected by epilepsy.

Here is how the brain injection work

Epilepsy develops due to an imbalance between excitatory and inhibitory neuron activity. The communication and exchange of information between neurons in the human brain occurs via electrical signals called action potential.

Excitatory neurons increase the action potential and promote a rapid flow of information from one neuron to the other. Inhibitory neurons, on the other hand, prevent signals from passing between neurons, reduce action potential, and avoid any hyperactivity in the brain. 

An imbalance between excitatory and inhibitory neurons can result in seizures, memory loss, and other brain disorders, including epilepsy. To restore this balance, the researchers developed NRTX-1001.

This brain injection contains a high-concentration dose of lab-grown inhibitory neurons developed from human embryonic stem cells. It brings down the overall action potential and eventually prevents seizures.

Within one year of NRTX-1001 treatment, the first patient who used to have 32 seizures a month experienced a 95 percent decline in monthly seizures. Whereas for the second patient (14 seizures per month), the reduction was over 90 percent. 

“Although our clinical investigation is ongoing in additional patients, it is gratifying to witness the first two patients achieving seizure relief without additional cognitive impairment to date, which supports the therapeutic potential of NRTX-1001,” said Nicholas.

NRTX-1001 promises a risk-free epilepsy cure 

A 2010 research paper suggests anti-epileptic medicines are ineffective in about 30 percent of patients. Another study published in 2021 reveals that about 30 to 50 percent of patients experience epilepsy relapse once they stop taking their medicines.  

Currently, all such patients rely on surgical treatments for permanent relief. However, brain surgeries come with their own risk. Patients might experience problems ranging from bleeding, allergic reactions to tissue damage. In some cases, the surgery may cause temporary loss of vision, memory, or speech in patients.

NRTX-1001, on the other side, promises a less invasive and non-destructive epilepsy treatment. Hopefully, future trials of this cell therapy will also be successful, and finally, the world will have a safe and sound epilepsy cure

For more valuable information on epilepsy, you can check the lists of epilepsy patient resources provided by the CDC and the National Association of Epilepsy Centers (NAES).