Brain Injection of Cultured Neurons: A Potential Cure for Epilepsy

Researchers at Neurona Therapeutics, a biotech company based in San Francisco, have achieved a remarkable reduction of over 90 percent in seizures experienced by two epilepsy patients through a brain injection. The patients were administered NRTX-1001, an experimental cell therapy developed by the company. Prior to the treatment, the patients were suffering from an average of 32 and 14 seizures per month, respectively.

Conventional anti-epileptic medications had proven ineffective, leaving highly invasive surgical options as their last resort. Instead, they opted to participate in the clinical trial and receive the innovative NRTX-1001 cell therapy. Cory Nicholas, the CEO of Neurona Therapeutics, commended the patients for their bravery in choosing this experimental treatment, as it offered a potential alternative to lobectomy or ablation surgery, which carried the risk of irreversible cognitive deficits.

In the past year, there has been a significant improvement in the condition of the patients, suggesting that their decision to undergo the cell therapy was a beneficial one. This breakthrough treatment offers hope not only to these individuals but also to millions of others whose lives are severely impacted by epilepsy.

Here is how the brain injection work

The brain injection operates based on addressing the underlying cause of epilepsy, which involves an imbalance between excitatory and inhibitory neuron activity. Neuronal communication and information exchange in the human brain rely on electrical signals known as action potentials.

Excitatory neurons play a role in amplifying action potentials, facilitating the rapid transmission of information between neurons. Conversely, inhibitory neurons function to hinder the passage of signals, reducing action potential and preventing excessive brain hyperactivity.

When there is an imbalance between excitatory and inhibitory neurons in the brain, it can lead to various brain disorders, including epilepsy, characterized by seizures and memory loss. To address this imbalance, the researchers at Neurona Therapeutics developed a solution called NRTX-1001.

This brain injection contains a concentrated dose of inhibitory neurons that have been grown in the laboratory using human embryonic stem cells. By introducing these inhibitory neurons, the overall action potential in the brain is reduced, ultimately preventing seizures.

Remarkable results have been observed within a year of administering NRTX-1001 to the patients. The first patient, who previously experienced an average of 32 seizures per month, witnessed a staggering 95 percent reduction in monthly seizures. Similarly, the second patient, who had been experiencing 14 seizures per month, saw a reduction of over 90 percent.

“While our clinical investigation is still ongoing with additional patients, it is highly encouraging to see the initial two patients achieve seizure relief without any noticeable cognitive decline thus far. This success provides strong support for the therapeutic potential of NRTX-1001,” commented Nicholas, expressing his satisfaction with the outcomes.

NRTX-1001 promises a risk-free epilepsy cure 

NRTX-1001 offers a potential solution for epilepsy that is free from risks. According to a research paper from 2010, approximately 30 percent of patients do not respond to anti-epileptic medications. Moreover, a recent study published in 2021 indicates that around 30 to 50 percent of patients experience a relapse of epilepsy once they discontinue their medication.

Currently, surgical interventions are the primary option for achieving long-term relief for such patients. However, these brain surgeries carry their own set of risks. Patients may encounter complications such as bleeding, allergic reactions, or tissue damage. In certain cases, the surgery can result in temporary vision loss, memory impairment, or speech difficulties.

In contrast, NRTX-1001 presents a less invasive and non-destructive treatment approach for epilepsy. The hope is that future trials of this cell therapy will also yield positive results, eventually providing a safe and effective cure for epilepsy.

For further valuable information on epilepsy, it is recommended to consult the lists of epilepsy patient resources provided by reputable organizations such as the CDC (Centers for Disease Control and Prevention) and the National Association of Epilepsy Centers (NAES).

The findings of the NRTX-1001 trial are unveiled at the 2023 Annual Meeting of the International Society for Stem Cell Research (ISSCR).

Read the original article on Interesting Engineering .

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