Neuroene Therapeutics awarded $1.5 million to develop anti-seizure compound for epilepsy

Neuroene Therapeutics awarded $1.5 million to develop anti-seizure compound for epilepsy

With funding from NIH Phase II SBIR grant, Neuroene Therapeutics will take the next steps toward bringing their vitamin K analogues for drug-resistant epilepsy to clinical trial

Neuroene Therapeutics, a start-up company founded by mitochondrial biologist Sherine S. L. Chan, Ph.D. and medicinal chemist C. James Chou, Ph.D. of the Medical University of South Carolina, has received a $1.5 million NIH Phase II Small Business Innovation Research grant to optimize vitamin K analogues that could improve seizure control in patients with drug-resistant epilepsy. Richard Himes, Ph.D., a chemist at the College of Charleston, serves as the company’s Chief Scientific Officer.

Photo: Dr. Chan and Dr. Chou are the founders of Neuroene Therapeutics, an MUSC start-up company that was awarded a 1.5M SBIR Phase II grant to develop a novel anti-seizure compound for drug-resistant epilepsy.

Of the 3.4 million Americans estimated to have epilepsy, one third do not receive adequate seizure control with current medications, either because the drugs do not work for them or because they cannot tolerate the drug’s side effects.

The SBIR grant will enable Neuroene Therapeutics to test the efficacy and safety of its lead compounds, which are analogues of a naturally occurring form of vitamin K that is essential for mitochondrial and neuronal health.

“Mitochondria are the powerhouses of the cell, and the brain needs a lot of energy for its function. A particular form of vitamin K protects the integrity of the mitochondria and helps them produce enough energy for brain cells,” explained Chan.

The form of vitamin K needed by the brain is not the same as the vitamin K we get from foods in our diet. The vitamin K we eat must first be processed by intestinal bacteria before transport to the brain, and then within neurons must be converted into the specific form of Vitamin K that is needed for mitochondrial and neuronal health.

Because the compound developed by Neuroene Therapeutics mimics this specific form of Vitamin K that the neuron needs (not the ingested form) and because it travels directly to the brain, it bypasses the need for transport systems.

“Unlike other vitamin K analogues, which require additional processing before they are in a usable form, our compounds are a direct substitute for the active form and go directly to the brain where they are needed,” said Chou.

Early testing of these vitamin K analogues by the MUSC investigators with pilot funding from the South Carolina Clinical and Translational Research Institute, a Clinical and Translational Science Awards hub funded by the National Institutes of Health, showed significantly reduced seizure activity with little toxicity in a zebrafish model. Testing in mouse seizure models at the National Institute of Neurological Disorders and Stroke Anticonvulsant Screening Program confirmed those findings.

With assistance from the MUSC Foundation for Research Development, Chan and Chou established Neuroene Therapeutics in 2015 and received a patent on their lead compounds earlier this year.

The current SBIR award will enable additional testing of the compounds’ efficacy and safety at the University of Utah’s Anticonvulsant Drug Development Program, directed by Karen Wilcox, Ph.D., which has robust rodent models of drug-resistant epilepsy. By the end of the two years of SBIR funding, Neuroene Therapeutics will have identified the lead compound to take forward into clinical trial.

Although Neuroene Therapeutics is focused currently on developing its lead compound for drug-resistant epilepsy, Chan and Chou are also studying whether vitamin K analogues could improve outcomes in other difficult-to-treat neurological diseases. They already have some promising preclinical data in Parkinson’s disease and mitochondrial DNA depletion syndrome. In addition, they speculate that the compounds could also be relevant to Alzheimer’s disease.

Apolipoprotein E4, one of the strongest genetic risk markers for late-onset Alzheimer’s disease, has a role to play in vitamin K transport. It is possible, then, that mitochondrial dysfunction due to insufficient transport of vitamin K could be implicated in Alzheimer’s and, if so, these brain-penetrating vitamin K analogues could bypass the transport process, thus improving mitochondrial health and disease outcome.


About Neuroene Therapeutics

Neuroene Therapeutics is a startup biotechnology company developing novel Vitamin K-based therapeutics for neurological disorders such as epilepsy. The company originated from collaborative research between Medical University of South Carolina investigators C. James Chou, Ph.D., and Sherine Chan, Ph.D., who cofounded and continue to lead Neuroene Therapeutics. Visit us at

About MUSC

Founded in 1824 in Charleston, The Medical University of South Carolina is the oldest medical school in the South. Today, MUSC continues the tradition of excellence in education, research, and patient care. MUSC educates and trains more than 3,000 students and residents, and has nearly 13,000 employees, including approximately 1,500 faculty members. As the largest non-federal employer in Charleston, the university and its affiliates have collective annual budgets in excess of $2.2 billion. MUSC operates a 700-bed medical center, which includes a nationally recognized Children’s Hospital, the Ashley River Tower (cardiovascular, digestive disease, and surgical oncology), Hollings Cancer Center (a National Cancer Institute-designated center) Level I Trauma Center, and Institute of Psychiatry. For more information on academic programs or clinical services, visit For more information on hospital patient services, visit

About the South Carolina Clinical and Translational Research Institute

The South Carolina Clinical and Translational Research (SCTR) Institute is the catalyst for changing the culture of biomedical research, facilitating sharing of resources and expertise, and streamlining research-related processes to bring about large-scale, change in the clinical and translational research efforts in South Carolina. Our vision is to improve health outcomes and quality of life for the population through discoveries translated into evidence-based practice.

About MUSC Foundation for Research Development

FRD has served as MUSC’s technology transfer office since 1998. During that period, FRD has filed patent applications on more than 400 technologies, resulting in over 150 U.S issued patents. Additionally, FRD has executed more than 150 licenses and spun out more than 50 startup companies. MUSC startups have had products approved by the FDA and acquired by publicly traded corporations while attracting substantial investment dollars into South Carolina. Innovations from MUSC, including medical devices, therapies and software, are positively impacting health care worldwide. Please visit us online at


OPINION COLUMN: Big Pharma, Special Interests Set Medication Legislation Adrift

OPINION COLUMN: Big Pharma, Special Interests Set Medication Legislation Adrift

Via: The Berkshire Eagle Columnists

By Brian Sullivan, Special to The Eagle

My politics are to the right. They are so far to the right that there are days I can’t believe I’m left-handed. But I don’t vote the party line, I don’t think that’s right, uh, correct. I’m not possessed of a great political mind, so at the end of the day I’m hoping that common sense prevails, and that the best interest of all involved is served.

That’s why the political tale that is Jackie Quetti’s both baffles and infuriates me. It’s why the state of politics leaves citizens bamboozled, when well-meaning politicos can’t take their individual good intentions and coalesce that energy into progressive and positive government. Quetti’s story is a good example of state politics adrift at sea.

Quetti, 21, will host her fifth Western Mass. Walk for Epilepsy on Sunday at Farnam’s Crossing on Route 8 in Cheshire. The combination fundraiser and awareness event (three-mile walk at 10 a.m., registration at 9) was sparked in large part in March 2009, when Quetti suffered a near-tragic grand mal  Tonic-Clonic seizure at home on an otherwise routine Saturday morning.

Having rallied against smaller or milder seizures since an early age, this “breakthrough seizure” dealt the 2010 Pittsfield High graduate and Elms College sophomore a new hand, one that would change her life dramatically. The events of that morning were caused in all probability by a switch in epilepsy medicine from brand name Lamictol to generic brand Lamotrogene.

The formulation of the new medication was different enough to bring on the more aggressive type of seizure. It was an episode that Quetti had never experienced before and one that shook the foundation of the entire family. The switch in medication, which arrived through the mail, was done without notifying either Quetti’s primary care physician or the neurologist in Boston who also treats her case.

We’re not talking about the common cold. Epilepsy is serious, and the medicines used to treat it, if changed even slightly, can have very negative impacts. In the reconfiguration of a new pill, a little bit more of this and a little bit less of that can be possibly fatal. That Saturday morning changed a young girl’s immediate and long range future. A chance to go away to college was delayed until this year, when the family finally felt comfortable enough to let her attend Elms in Chicopee.

Massachusetts State House

Massachusetts State House

A bill is currently floating in the Statehouse that, if/when passed, will change the level of communication and allow primary care doctors and the patient to be notified in advance of any formula changes in medicine.

Quetti has appeared and spoken on behalf of the bill three times — yes, three times — at the Statehouse and offered her story as a selling point. Quetti has also lobbied her case in Washington, D.C.

So, what’s the problem? The lengthy stall is due, in part, to the usual wariness of special interest groups such as the pharmaceutical, medical and insurance communities, who want to make sure all T’s are crossed and I’s dotted before signing off. Taking care of “us” first trumps what’s best for young people like Quetti. At least that’s how I read the road signs.

“I’m usually right on top of that sort of thing,” said Nancy Quetti, Jackie’s mother. “I noticed the change in the name of the medicine, but didn’t think too much about it. It turned out to be a scary thing. Jackie began taking the new medicine about two weeks before that seizure, and she’s told me since that during those two weeks she felt not quite herself.”

The bill is not about just about notification of changing brand name drugs to generic, Nancy Quetti added. “It’s about generics to other generics, too.”

Les Brody is the president of the National Epilepsy Foundation-New England. He has also testified at the Statehouse in favor of the bill. But Jackie, Brody said, has been to the well three times in Boston and remains the bill’s biggest advocate and its strongest voice.

“When the bill does pass, it will have been Jackie who was very instrumental,” said Brody, whose foundation focuses on the education, service and research of the malady. “This bill is designed to protect individuals from medicine switches being made at the pharmaceutical level. It’s a safety-first bill.”

That may be so, but the clock keeps ticking, and that leaves people such as Jackie Quetti at a higher risk. The bill is designed to narrow that risk.

“It should have been a slam dunk,” said Brody, who added that Jackie Quetti’s repeated story and frequent visits to the Boston political arena have left her and state representatives on a first-name basis.

Nancy Quetti said that local state representatives and senators have supported and worked toward passing the bill.

Jackie Quetti is one-third of the Quetti triplets. Diana, a theater enthusiast, had a lead role in a play this past spring during her junior year at North Carolina State. Rachel, a former Pittsfield High basketball standout, has had her share of pressure moments on the hardwood.

Jackie said she’d gladly switch testifying at the Statehouse for either one of those high-pressure moments.

“Yes, nerve-wracking,” she said about testifying before committees in Boston.

But the young woman has accepted fate and learned to embrace her role as an advocate, and it was Brody who said that as much as he knows about the subject, he hasn’t walked a mile in Jackie Quetti’s shoes.

“When she speaks people have to listen,” Brody said. Brody added that when he lobbies for the bill he does so by emphasizing the social costs that come with no action. He said people with epilepsy are unemployed at three times the national average, in part because of their being prone to seizures.

“Think about it,” Nancy Quetti said. “When Jackie had her breakout seizure it involved an ambulance call and subsequent medical attention. That costs money at different levels. This bill isn’t about slamming generics or being negative about the medical, insurance and legal communities. There are so many good people in those areas. It’s just about trying to do the right thing for people like Jackie.”

I think common sense can play a role here. Agree?

Using an AED Used to Treat a Persistent Cough?

Using an AED Used to Treat a Persistent Cough?

Chronic Cough Responds to Epilepsy Drug, Study Finds

A drug commonly used to control epileptic seizures and pain, gabapentin, also appears to ease hard-to-treat chronic coughs, a new study says.

Chronic cough affects 11 percent to 16 percent of the population, the researchers report. The exact cause is unknown, but it might relate to a malfunction in a part of the brain that causes coughing. Gabapentin works by suppressing that “cough center,” they said.

“It is effective and well-tolerated, and may provide significant relief and respite from such a physically and psychologically disabling condition,” said lead researcher Nicole Ryan, a clinical research scientist at the University of Newcastle in Australia. “Gabapentin is a real treatment option for people with refractory chronic cough, especially for those with features of central sensitization.”

People with this kind of cough feel the need to cough when there is no cough stimulus present. It’s thought that neurons in the central nervous system fire off unnecessarily.

The report is published online Aug. 28 in The Lancet.

For the study, Ryan’s team evaluated 52 people with chronic cough that had not responded to other treatments to. All had had their cough two months or longer and were randomly assigned to take gabapentin or placebo. (more…)