Veterinarian and neurologist Sonja Bröer, together with her colleagues, has investigated the potential of regenerative cell treatments with the intention of either curing epilepsy or making its symptoms much less severe. Before joining the faculty at Berlin’s Freie Universität, Bröer oversaw the preclinical research team of San Francisco’s Neurona Therapeutics, Inc.

The company is working on a cell therapy (NRTX-1001) for seizures that don’t respond to drugs treatments. They have now released the results of preliminary tests in the journal Cell Stem Cell.

It’s estimated that epilepsy affects 50 million individuals globally. Disturbingly, a significant fraction, nearly a third, find that their seizures remain unresponsive to traditional drug therapies.

One of the pivotal neurotransmitters, gamma-aminobutyric acid (GABA), plays a role in curtailing this hyperactivity. Nonetheless, in epilepsy sufferers, the specific neurons producing GABA can deteriorate, leading to an imbalance between excitatory and inhibitory signals. This imbalance is considered a primary driver for epileptic episodes.

The ground-breaking research presented in Cell Stem Cell by Dr. Bröer and her team details a promising approach. They’ve pioneered the transplantation of inhibitory neurons, which release GABA, aiming to reestablish the brain’s equilibrium and thereby curtail epileptic activities. Such cellular therapeutic approaches hold immense promise as an alternative for those grappling with drug-resistant seizures.

Further emphasizing its potential, NRTX-1001, formulated by Neurona Therapeutics, is based on human embryonic stem cells, which are then transformed into inhibitory neurons. When tested on a mouse model mimicking chronic epilepsy, the results were astounding. Most subjects displayed profound seizure suppression, with a significant proportion experiencing complete cessation of seizures.

Beyond mere integration, these introduced neurons spread widely in the brain and seamlessly merged with the existing neural network. Not only did they exhibit long-term survival, but they also minimized epilepsy’s detrimental effects, such as brain scarring – an affliction also observed in human patients.

This therapeutic approach’s impact was found to be dose-responsive and was devoid of any detrimental side effects. Encouragingly, this propels the current phase 1/2 clinical trial (NCT05135091) focusing on drug-resistant epilepsy.

If it garners approval, it will position itself as a pioneering cell therapy for epilepsy on a global scale, potentially revolutionizing the lives of countless individuals facing treatment-resistant seizures.


Source:, Puja Molpariya