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Small Business Award Funding Epilepsy Drug Research

James Chou and Sherine Chan

James Chou, foreground, and Sherine Chan (SherineChan.com)

26 August 2016. A federal grant is funding a project by a start-up enterprise in South Carolina to develop a new class of drugs to treat epilepsy. National Institute of Neurological Disorders and Stroke, part of National Institutes of Health, made the $225,000 award to Neuroene Therapeutics, a spin-off company from Medical University of South Carolina, in Charleston.

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. World Health Organization estimates some 50 million people worldwide have epilepsy, where in many cultures people with the condition face stigma and discrimination. While epilepsy can be treated in most cases, WHO says as many as 30 percent of episodes do not respond to treatment.

This large percentage of non-responders to current epilepsy treatments is driving research by MUSC faculty members Sherine Chan and James Chou. Chan studies defects in mitochondrial or energy functions of cells, including research with zebrafish models that share many genetic characteristics with humans. Chou investigates drug targets based on histone deacetylases, a class of enzymes associated with cancer and other disorders.

In a 2014 paper, Chan, Chou, and colleagues screened potential compounds against histone deacetylase targets associated with epilepsy using zebrafish as model organisms. They discovered some synthetic forms of vitamin K, a natural vitamin associated with blood clotting, reduced seizure-like activity in zebrafish. Tests with mice found a particular synthetic form of vitamin K reduced epilepsy-like seizures by improving mitochondrial functions in brain cells.

Chan and Chou founded Neuroene Therapeutics in May 2015 to commercialize their discovery. The company identified a lead compound it calls Alkyne-VK, a vitamin K analog, which they tested with zebrafish and mice. They report Alkyne-VK not only reduces seizures in mice, it penetrates mouse brains in about 15 minutes, is non-toxic, and even provides protection to nerve cells. Its drawback, however, is a short half-life in blood serum, of about 1 hour.

In the project funded by NIH, the Neuroene-MUSC team plans to synthesize 30 to 50 variations of Alkyne-VK that continue to show efficacy with epilepsy in lab cultures and animal models, as well as convey protection for nerve cells. In addition, the researchers will identify an optimal candidate that can retain its anti-seizure activity for at least 6 hours. If this first stage is successful, the company and MUSC will apply for second-stage funding to carry out preclinical and toxicology tests in preparation for an investigational new drug application with FDA.

“This class of compounds has a new molecular mechanism that makes it different from any of the current anti-seizure drugs available to patients with epilepsy,” says Chan in a university statement. ” We intend to provide a new generation of anti-seizure drugs that is clearly needed.”

The award is made under NIH’s Small Business Technology Transfer program that supports commercialization of biomedical technologies undertaken by collaborations between research institutions and small businesses.

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