Grant Funds Epilepsy Stem Cell Therapy Trial

(Gordon Johnson, Pixabay)

27 May 2022. A developer of stem cell therapies for neurological disorders is receiving an $8 million grant to advance a treatment for a common type of epilepsy. California Institute for Regenerative Medicine is awarding the funds to Neurona Therapeutics in South San Francisco to support a clinical trial of its experimental therapy for mesial temporal lobe epilepsy.

Epilepsy is a neurological disorder where nerve cell activity in the brain is disturbed, causing seizures with symptoms ranging from blank stares to tingling sensations to loss of consciousness. Mesial temporal lobe epilepsy, or MTLE, is a variation of the disorder, marked by focal onset seizures, where individuals are awake and aware of seizures, or their awareness is impaired. According to the Epilepsy Foundation, temporal lobe epilepsy is the most common form of the disorder with focal seizures.

MTLE occurs in the medial or internal structures of the brain’s temporal lobe, the part of the brain largely responsible for consciousness and long-term memory, as well as playing a role in processing visual images and sound. About eight in 10 temporal lobe seizures are from MTLE. While some people with MTLE respond to anti-seizure drugs, as many as one-third of those with the condition do not respond to medication, leaving surgery to remove the responsible region of the brain the only treatment option.

Neurona Therapeutics is creating treatments for neurological disorders with allogeneic or off-the-shelf stem cells. The company derives its cell therapies from embryonic pluripotent stem cells, induced to transform into functioning brain cells, both neurons and glia cells that support neurons. Neurona says its cell lines represent the medial ganglionic eminence, a class of precursor cells that develop into cerebral cortex tissue. The company says its approach replaces damaged neurons and glia cells causing neurological disease with its regenerative cell treatments, and cites preclinical studies by the company’s scientific founders and colleagues as evidence.

Repair hyperexcitable neural networks

Neurona’s lead product, code-named NRTX-1001, is designed as a one-time treatment for drug-resistant focal epilepsy, such as MTLE. The company says NRTX-1001 is derived from medial ganglionic eminence cells transforming into neurons that limit the neurotransmitter gamma-aminobutyric acid associated with seizures in epilepsy. Neurona says NRTX-1001 is injected into the brain, targets the region responsible for seizures, and non-destructively replaces damaged or dysfunctional cells. The company says its preclinical studies show NRTX-1001 suppresses seizures without signs of dose-limiting toxicity.

“Our regenerative cell therapy approach has the potential to repair the hyperexcitable neural networks that underlie focal epilepsy,” says Neurona’s chief medical officer David Blum in a company statement. “About a third of individuals with epilepsy have drug-resistant seizures. Removal or ablation of the affected temporal lobe can be an option, but many people with epilepsy are not eligible or interested in a tissue-destructive procedure.”

Based on this evidence, says Neurona, Food and Drug Administration cleared the start of a early- and mid-stage clinical trial assessing NRTX-1001 against a placebo in people experiencing MTLE seizures. The study is enrolling 40 participants, with the first 10 participants testing various doses of the therapy. In the next stage of the trial, 30 participants are randomly assigned two-to-one to receive NRTX-1001 or a placebo. Participants in the study are tracked for a year, looking mainly for signs of adverse effects, but also changes in the frequency of seizures.

CIRM, a California state agency supporting stem cell research, announced yesterday an award to Neurona of $8 million for the clinical trial. The agency notes in a statement that “current therapies for drug-resistant epilepsy are only partially effective and have serious drawbacks.” CIRM adds that with NRTX-1001 “cells are injected into the brain in the area affected by the seizures where they release a neurotransmitter or chemical messenger that will block the signals in the brain causing the epileptic seizures.”

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