HD Insights™

HD INSIGHTS Walter Koroshetz, MD VITAL SIGNS NAME: Walter Koroshetz, MD CURRENT POSITION: Deputy Director of the National Institute of Neurological Disorders and Stroke PREVIOUS POSITIONS: Vice Chair of the Neurology Service at Massachusetts General Hospital; ! ! EDUCATION: MD, The University of Chicago HOBBY: Long-distance bicycle trips. Most recent ride was 50 miles from Arlington, Virginia to Purcellville, Virginia and took 4.5 hours. INSIGHTS: Turning to the NIH funding outlook, it has been a very tough time for the NIH in terms of its budget. In 2011, NIH spent $65 million on research on Huntington disease. Is that enough? KOROSHETZ: No, but unfortunately, we could say that about every disease. No disease is getting too much, as far as I can see. There are now unique opportunities in neuroscience to make advances in lots of different diseases, and I think the pace at which that happens depends on what resources you have available. We still get quite a bit of money, so the taxpayers' investment of $31 billion to the NIH is something I think the country can be proud of. INSIGHTS: Given limited resources, what are your top priorities for Huntington disease? KOROSHETZ: My thoughts are that we have an opportunity with a penetrant dominant genetic disorder to really try and understand how to block the pathology before it happens. Huntington disease is a nice example of how basic science, disease science, and human studies can come together and hopefully come up with a new treatment that slows down the disease. And the great thing about it is, if we do find something that slows down the disease in patients, we have the opportunity to identify people before they have the disease, and treat even before the disease becomes manifest. From the NIH point of view, Huntington is a very attractive disease. INSIGHTS: The NIH is trying to facilitate clinical research and clinical trials in neurology with a program called NeuroNEXT. Can you tell us what NeuroNEXT is? KOROSHETZ: NeuroNEXT is a network of sites throughout the country that have come together to do very innovative biomarker studies and phase two clinical trials in neurological disorders. And there is a really good group of committed principal investigators at the 25 sites. Dr. Merit Cudkowicz, who has been active in the HSG, is the principal investigator of the coordinating center; Dr. Karen Marder is on the steering committee as the principal investigator from Columbia. We have a lot of hope that this group will be able to partner with industry and academicians and be the first to test innovative and exciting treatments in humans. INSIGHTS: What does it mean for investigators? KOROSHETZ: For investigators working on therapies, this is a potential avenue by which therapies can get to patients, to test, and see whether they really hit proof-of-principle or target engagement. For industry investigators, there's a potential mechanism that they could use to test whether their drugs are actually engaging their anticipated targets. For disease organizations who are involved in funding Huntington research in the early stages, if there's a really promising drug that meets a good score with peer review, this network could be testing those drugs. INSIGHTS: How can industry work with NeuroNEXT? KOROSHETZ: We have mechanisms by which the NIH can enter a cooperative research agreement with industry to try and protect their intellectual property as one of their drugs comes into the network for testing. We would be looking for co-funding, in some instances, to get this done. But if we have an efficient network that can really quickly do high-quality studies in patients, then I think that would be of benefit to industry as well. INSIGHTS: You say co-funding. Does that mean the NIH would help companies investigate their own drugs? KOROSHETZ: Absolutely. If they think they're going to make a difference for patients, we're definitely interested in partnering with industry to do that. INSIGHTS: A pharmaceutical company might think that working with the NIH means a lot of bureaucracy in a long time horizon that usually isn't compatible with the pharmaceutical industry's priorities. How will NeuroNEXT address that? KOROSHETZ: At NeuroNEXT, we put together mechanisms by which companies come in with an idea, and get a turnaround time that's very quick with regards to whether there's interest on the part of the institute and the network to move forward with that proposal. I think for a large number of industry applications, we can really shorten the turnaround time, and still try to compete in terms of going as fast as industry trials could. INSIGHTS: Thank you Dr. Koroshetz for talking with us today. 2 Copyright © Huntington Study Group 2012. All rights reserved. HD Insights, Vol. 2 Director of Stroke and Neurointensive care at Massachusetts General Hospital; Professor of Neurology at Harvard Medical School

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