A new therapeutic approach, supported by CIRM, that blocks brain signals that can cause epilepsy has been approved by the US Food and Drug Administration (FDA) for clinical trials.

More on epilepsy

Nearly 3.5 million Americans suffer from some form of epilepsy. It can affect people in different ways, from stiff muscles or staring spells to violent shaking and loss of consciousness. Its impact on people’s lives extends far beyond the condition itself.

People who suffer from epilepsy experience a higher frequency of depression and other mood disorders, social isolation, challenges in school and with living independently, higher unemployment, limitations on driving, and higher risk of early death.

Medications can help control the seizures in some people, but around one-third of patients don’t respond to those drugs. The alternative is surgery, which is invasive and can cause damage to delicate brain tissue.

Neurona Therapeutics developed an approach called NRTX-1001. This approach turns stem cells into interneurons, a type of nerve cell in the brain. These cells secrete chemical messengers called GABA, an inhibitory neurotransmitter. GABA inhibitory neurotransmitters help rebalance misfiring electrical signals in the brain and, hopefully, eliminate or reduce seizures.

Nerona and Cory Nicholas

Cory Nicholas, PhD, co-founder and CEO of Neuron’s Therapeutics, said FDA approval for a clinical trial is a key milestone.

“Neurona’s accomplishments are a testament to longstanding support from CIRM,” Nicholas said. “CIRM has supported the NRTX-1001 program from bench to bedside, dating back to early research in the Neurona founders’ laboratories at the University of California, San Francisco. We are thankful to CIRM for their support of this important work that has the potential to provide seizure-freedom for patients who currently have limited treatment options.”

In a news release, Dr. Nicholas said the timing was perfect. “This milestone is especially rewarding and timely given that November is Epilepsy Awareness Month. NRTX-1001 is a new type of inhibitory cell therapy that is targeted to the focal seizure onset region in the brain and, in a single treatment, has the potential to significantly improve the lives of people living with focal epilepsy.”

In animal models, NRTX-1001 produced seizure freedom in more than two-thirds of the treated group, compared with just 5 percent in the untreated group. It also resulted in reduced tissue damage in the seizure-affected area of the brain.

The clinical trial will initially target people affected by mesial temporal lobe epilepsy (MTLE), where seizures often begin in a structure called the hippocampus. MTLE is the most common type of focal epilepsy.

CIRM has invested almost $6.67 million across three stages of this project. The investment spans early discovery through this latest late-stage preclinical work, highlighting our commitment to advancing the most promising science from bench to bedside.