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CURE Epilepsy is dedicated to funding patient-focused research to find a cure for the 65 million people with epilepsy worldwide. This month, we share the following articles and abstracts which are furthering the study of epilepsy and bringing the world closer to a cure.

This issue of Epilepsy Research News includes summaries of articles on: 

• A New Fruit Fly Model to Probe an Epileptic Brain Disorder

• Neurona Therapeutics Announces Publication in Cell Stem Cell Reporting the Development of Investigational Novel Regenerative Cell Therapy Strategy for Drug-Resistant Focal Epilepsy

• CHOP Researchers Develop First-of-Its-Kind Prediction Model for Newborn Seizures

• New Investigational Drug Offers Hope for Patients with Treatment-Resistant Epilepsy

A New Fruit Fly Model to Probe an Epileptic Brain Disorder

Developmental and epileptic encephalopathy (DEE) refers to a group of neurodevelopmental conditions characterized by developmental delay, cognitive impairment, and seizures in children. In 2016, the first case linking variants in both copies of the UBA5 gene to DEE44 was reported. Since then, twelve distinct missense variants in the UBA5 gene have been identified. Recently, Dr. Hugo J. Bellen and his team at Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital generated a new fruit fly model to assess the severity of symptoms caused by each of these variants. The severity and the type of symptoms varied widely among different variants. For instance, five variants caused progressive motor defects, three of which also caused developmental delays or seizure-like symptoms. Their systematic analysis lays the foundation for better evaluation of the variants, which is important for DEE44 patients in the future and for the development of drugs and gene therapy to treat this rare disorder. The study was published in the journal eLife.

Neurona Therapeutics Announces Publication in Cell Stem Cell Reporting the Development of Investigational Novel Regenerative Cell Therapy Strategy for Drug-Resistant Focal Epilepsy

Neurona Therapeutics recently published data on the development and characterization of human stem cell-derived, medial ganglionic eminence inhibitory interneurons for the potential treatment of focal epilepsy. In a preclinical study, administration of a single dose of the cells in a chronic model of drug-resistant mesial temporal lobe epilepsy (MTLE) resulted in long-lasting and consistent suppression of focal seizures, as well as improvements in neuropathology and an increase in survival. The study also demonstrated a potentially broad, safe, and effective dosing range in the model. The data support the development of Neurona Therapeutics’ regenerative cell therapy candidate, NRTX-1001, which is being evaluated in an ongoing Phase I/II clinical trial of a one-time dose in people with drug-resistant MTLE (NCT05135091).

CHOP Researchers Develop First-of-Its-Kind Prediction Model for Newborn Seizures

Researchers from Children’s Hospital of Philadelphia (CHOP) have developed a prediction model that determines which newborn babies are likely to experience seizures in the neonatal ICU (NICU). Neonatal seizures are a common neurologic issue in newborn babies. Approximately 30% of newborn babies with a temporary lack of oxygen to the brain (known as hypoxic-ischemic encephalopathy, or HIE) will have seizures. Most of these seizures can only be detected through EEG monitoring and not simply through clinical observation. This model could be incorporated into routine care to help the clinical team decide which babies will need EEGs and which babies can be safely managed in the NICU without monitoring through EEGs. This would allow families and providers to care for babies without intrusive and unnecessary procedures. The study team has made the model available as an online tool for the community.

New Investigational Drug Offers Hope for Patients with Treatment-Resistant Epilepsy

A recent article in JAMA Neurology demonstrates efficacy and safety results from the X-TOLE Phase 2b randomized clinical trial of XEN1101, a novel potassium channel opener, in adults with focal epilepsy. Treatment with XEN1101 was associated with a dose-dependent median percent reduction from baseline in monthly focal onset seizure frequency with a reduction of 52.8% for 25 mg, 46.4% for 20 mg, and 33.2% for 10 mg, compared with 18.2% for placebo. XEN1101 was generally well-tolerated and treatment-emergent adverse events were similar to those of commonly prescribed antiseizure medications. Unlike several treatments that must be started at low doses and slowly increased, the new drug can safely be taken at its most effective dose from the start, the authors say. XEN1101 is currently being evaluated in Phase 3 clinical trials in patients with focal onset seizures and primary generalized tonic-clonic seizures.

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