$225,000 funding for promising epilepsy research

The Epilepsy Research Foundation (ERF), a unique joint venture of non-profit epilepsy organizations, and the Milken Family Foundation, a private philanthropic foundation, today announced two new grants recognizing promising epilepsy research in clinical development, totaling $225,000 in funding. The grant awards were announced by the Epilepsy Therapy Project and the Epilepsy Foundation, as well as the Milken Family Foundation, during the 7th Judith Hoyer Lecture in Epilepsy at the annual meeting of the American Epilepsy Society in Boston, MA.

These grants are designated to support critical stages of advancement - through early clinical or to bridge from preclinical to clinical development - for groundbreaking therapies in epilepsy. The award committee, comprised of clinical, scientific and industry representatives, evaluates research programs demonstrating the greatest potential for near-term patient benefit and pioneered by qualified clinicians and scientists advancing new therapeutic approaches in epilepsy and the central nervous system. The two recipients announced today have been recognized for promising clinical work in advancing a novel agent, 2-deoxy-D-glucose (2DG), for control and reduction of seizure frequency, and a new therapeutic specifically designed for the treatment of newborns with hypoxic brain injuries resulting in seizure-related conditions.

"These awards recognize important advances in the treatment of epilepsy, providing new hope for patients living with uncontrolled seizures and for newborns with serious brain cell injury," said Warren Lammert, Chairman, Epilepsy Therapy Project. "Funding today for new therapies is limited, and with these awards we are able to advance important research through critical junctures so that innovators can broadly access resources necessary to bring these new treatments to patients."

"Epilepsy affects nearly three million people in the United States and 50 million people worldwide," said Eric R. Hargis, President and CEO of the Epilepsy Foundation. "Even with current treatments, approximately 30 percent of people with epilepsy live with uncontrolled seizures. Our goal in awarding these grants is to help provide them with new and better treatment options."

"We are gravely in need of novel epilepsy treatments with new underlying modes of action for all patients living with seizure conditions, and in particular to provide more effective options for young children and for women of childbearing age," said Howard R. Soule, Ph.D., Chief Science Officer for the Milken Family Foundation. "We are proud to support high-caliber research in the field of epilepsy, and truly hope to see progress in making available effective and tolerable treatments in our lifetimes."

The December 2009 Grant Recipients

Benefits of the Ketogenic Diet Lead to Discovery of New Anticonvulsant

Nathan Fountain, M.D., Director, F.E. Dreifuss Comprehensive Epilepsy Program, University of Virginia and Thomas Sutula, M.D., Ph.D., Detling Professor and Chair, University of Wisconsin-Madison and Co-founder, Chief Technical Officer of NeuroGenomeX, Inc., will be assessing the clinical activity of 2-deoxy-D-glucose (2DG) in reducing seizure frequency in subjects with a history of frequent seizures. The investigators will use the grant funding in an initial Phase II safety, tolerability and proof-of-principle clinical study of 2DG. By investigating the mechanisms of anticonvulsant action of the ketogenic diet (isocaloric replacement of carbohydrates by fats and proteins), researchers identified the inhibition of glycolysis as a novel mechanism of anticonvulsant and antiepileptic action. Administration of 2DG, a known inhibitor of glycolysis, also demonstrated acute anticonvulsant action in early evaluation, and had pronounced anticonvulsant and antiepileptic effects in preclinical studies. Importantly, the pattern of anticonvulsant efficacy of 2DG in screening models is distinctive as compared to currently available drugs. 2DG has a well-established preclinical efficacy and toxicity profile, with decades of use as an imaging tracer, as well as safety established during previous Phase I/II human clinical trials for cancer.

Demonstration of efficacy in this initial clinical proof-of-principle trial would be a substantial milestone in the development of 2DG as a therapy for seizure disorders by adding evidence of efficacy and safety to current preclinical evidence in epilepsy animal models.

New Therapeutic with Promise for Neuroprotection and Treatment in Neonatal Care

A University of Minnesota research team headed by James Cloyd, PharmD, Professor of Experimental & Clinical Pharmacology, Lawrence C. Weaver Endowed Chair-Orphan Drug Development, and Director, Center for Orphan Drug Research, received funding to support the study of an intravenous formulation of the anticonvulsant Topiramate (TPM), a drug that has demonstrated important neuroprotective properties in preclinical models. Topiramate, in its oral formulation, is currently prescribed to treat epilepsy in children and adults. In this program, researchers are pursuing the long-term goal of developing a novel, FDA-approved, intravenous topiramate for the neuroprotection and treatment of seizures in neonates with hypoxic brain injuries. An estimated 15,000 neonates experience seizures, which cause brain injury and death. Current therapy is only partially effective and is associated with serious adverse effects. The National Institutes of Neurological Disorders and Stroke has identified improved treatment of neonatal seizures as a significant unmet need.

Grant funding will be applied to conduct a safety and pharmacokinetics study of a new IV and oral topiramate in healthy volunteers.