Success Breeds Success: The Anticonvulsant Drug Development ProgramJul 4, 2014
Female Voice: Examining the latest research and telling you about the latest breakthroughs. The Science and Research Show is on The Scope.
Julie: The anticonvulsant drug development program at the University of Utah has been instrumental in bringing an amazing 16 epilepsy drugs to the clinic. My guest, Dr. Steve White, is director of the program. Dr. White, I understand there are some impressive statistics associated with your program.
Dr. Steve White: Yes, Julie. Since the inception of this program in 1975 there have been approximately 31,000 investigational new drugs that have been through the doors here at the University of Utah. Of those 31,000 about 16 new drugs have come to market since 1993. The Utah program has actually been responsible for the early identification of anti-seizure activity for six of those compounds.
Julie: What do you think it is about the program that has made it so successful?
Dr. Steve White: One of the important aspects of this program is it really is a partnership between the National Institutes of Health, the academic and the pharmaceutical sponsor, and the University of Utah. The way the program works is that it is a free service to the sponsor. Anyone worldwide who thinks they may have a drug that could be useful for the treatment of epilepsy can submit their compound to the National Institutes of Neurological Disorders and Stroke, or the N.I.N.D.S., and then that compound is actually sent to Utah. We do the initial testing in a series of animal seizure and epilepsy models which over the years have proven really predictive efficacy in the patient population with epilepsy. The other important aspect of it is that success breeds more success. As new drugs come to market that show promise more sponsors come to the table, and because this is a service provided free to the sponsor there's really no reason for someone not to submit their compound.
Julie: Yeah. So a sponsor could be a researcher or, say, a company?
Dr. Steve White: Absolutely. The sponsor could be an academic chemist next door, or it could be someone in Europe or China or India, and indeed we see compounds coming from all over the world.
Julie: I imagine you test them on many types of epilepsy, because there are many causes of the disease.
Dr. Steve White: Indeed. One of the things that makes epilepsy so challenging to treat and to understand is because there are so many causes. The common causes that we think about of acquired epilepsy would be traumatic brain injury, stroke in the elderly patient, C.N.S. or brain infection in the younger patient. In addition to that, you have many of the genetic causes of epilepsy that are being identified today, and with every passing month or year more and more genetic mutations are being identified that can lead to epilepsy. By profiling a drug that a sponsor might submit to the program in these various models of epilepsy that are intended to model the different human epilepsies or at least that particular class, we provide the sponsor with sufficient information for them to move forward and be thinking about a clinical development program.
Julie: How long might it take for a compound to come to you and you either to say okay this isn't going to do anything or to ultimately get it perhaps to market in the best cases?
Dr. Steve White: If a compound came in, and we had enough compound, and we could move it all the way through, and it was showing efficacy or effectiveness at each of the various steps, it could move all the way through our program in about a year very effectively. Then, it would go onto the toxicology studies, and then it would go into the clinical testing. That overall program is estimated to take about eight to ten years. It's a long process from beginning to end.
Julie: What different directions do you envision the program taking?
Dr. Steve White: Over the years we've learned that epilepsy is more than about a patient's seizure control. One of the things that we have been doing recently in the last two years with the support of the N.I.H. is developing models of learning and memory and testing drugs in animals to see whether the most promising drugs actually do affect cognitive processes. It's not only about the drugs, but it's also trying to understand the effect of the epilepsy on learning and memory and affect. We're constantly developing those models to try and study that particular aspect of epilepsy.
Julie: Looking to the future, how might your program be changing?
Dr. Steve White: Take for example the patient with traumatic brain injury. We know that about 20% of those patients are high risk for developing epilepsy within the next 24 to 36 months. Imagine a day if we had biomarkers which could predict which of that 20% patient population would develop epilepsy, and imagine our ability to identify a neuro- protective drug that the patient could be treated with for a short period of time during the remodeling of the brain that leads to epilepsy to prevent that. This whole area falls under the idea of disease modification, and this is something that our program is certainly very interested in and the entire epilepsy research community is involved in.
Announcer: Interesting, informative, and all in the name of better health. This is The Scope Health Sciences Radio.