Researchers Hope to Develop Novel Epilepsy Therapy from New Compound

Researchers Hope to Develop Novel Epilepsy Therapy from New Compound

A new compound call CERC-611 (LY3130481) that targets specific neural circuits in epilepsy could become a new drug candidate for the disease, according to a study by researchers at Eli Lilly and Company.

The study, “Forebrain-Selective AMPA-Receptor Antagonism Guided By TARP ƴ-8 As An Antiepileptic Mechanism,” was published in the journal Nature Medicine.

Epileptic seizures happen when certain neuronal proteins become over-activated, dysregulating the communication and exchange of electrical signals between neurons. One such group of proteins are the AMPA receptors, whose activation is crucial for neuronal activity but, when overactivated, contribute to the onset of epileptic seizures and neuronal damage.

Previous research has attempted to investigate whether blocking these proteins could improve seizures in epileptic patients. However, given that the proper functioning of AMPA receptors is so important for the brain, such therapies need to be considered with caution to avoid undesirable side effects such as dizziness, lack of muscle coordination, and falling.

CERC-611 acts by blocking a protein called TARP-ƴ-8, that supports the activity of AMPA receptors in certain regions of the brain such as the forebrain, an area involved in the generation of focal epileptic seizures. Because TARP-ƴ-8 is not present in all areas of the brain, blocking its activity allows the selective control of AMPA receptors without interfering with their normal activity in the rest of the brain.

“Targeting these receptors may lead to improved anti-seizure efficacy, safety and tolerability, and make a significant impact on treatment outcomes,” Michael Rogawski, professor of neurology and pharmacology at the University of California, said in a news release.

”No prior epilepsy treatment targets a subset of brain receptors involved in seizure generation in a regionally-selective fashion,” he said.

Researchers tested the effectiveness and specificity of CERC-611 in brain tissue from an epileptic patient as well as in animal models of the disease. They found that CERC-611 was able to prevent seizures without causing motor side effects.

“We believe CERC-611 has the potential to provide a true advancement in epilepsy therapy,” said Uli Hacksell, CEO of Cerecor, the company developing the potential drug.

Cerecor is planning to file an Investigational New Drug application for CERC-611 with the U.S. FDA, as well as begin a Phase 1 clinical trial in 2017.

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Joana brings more than 8 years of academic research and experience as well as Scientific writing and editing to her role as a Science and Research writer. She also served as a Postdoctoral Researcher at the Center for Neuroscience and Cell Biology in Coimbra, Portugal, where she also received her PhD in Health Science and Technologies, with a specialty in Molecular and Cellular Biology.

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