Dr. Walter Koroshetz became Deputy Director of the National Institute of Neurological Disorders and Stroke (NINDS) in 2007. He received his medical degree from the University of Chicago and trained in neurology at Massachusetts General Hospital (MGH). After completing his residency, he was introduced to the HD Center without Walls at MGH and Boston University. He started seeing patients with HD and became involved in the first pre-symptomatic testing for HD. Dr. Koroshetz was also involved in early HD imaging research, and helped discover the effect of HD on lactic acid levels in the brain.1
At the 2011 Huntington Disease Clinical Research Symposium in Indianapolis, Indiana, Dr. Koroshetz sat down with HD INSIGHTS to discuss his role as Deputy Director of NINDS and the NIH’s new project, NeuroNEXT.

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; Director of Stroke and Neurointensive care at Massachusetts General Hospital; Professor of Neurology at Harvard Medical School
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: What motivated your transition from academia to the NIH?
KOROSHETZ: I was at a point in my career where I had done a bunch of things, and there were a number of leadership opportunities opening up. One of them happened to be this job at the NIH, which I thought was unique. There’s only one Deputy Director of NINDS in the country, and you get an experience that is quite unique, that no one else really sees. You get to see neuroscience, neurology, policy and government from a unique vantage point.
INSIGHTS: Has it been interesting?
KOROSHETZ: Yes. It’s like being a kid in a candy shop. If you’re interested in all sorts of different neurologic issues, it’s the best place to be because you see everything. I may have meetings on three different diseases in one day. If you have ADHD for neurology, it’s a great job!
INSIGHTS: Were there any drawbacks?
KOROSHETZ: There’s clearly a big difference when you go from being in an academic institution to being in an administrative job in government. So I certainly miss a lot of the interactions I had with patients, young physicians, residents, fellows, colleagues in the trenches. There’s nobody really in the trenches with me anymore. I’m kind of there by myself.
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.
- Jenkins BG, Rosas HD, Chen YC, Makabe T, Myers R, MacDonald M, Rosen BR, Beal MF, Koroshetz WJ. 1H NMR spectroscopy studies of Huntington’s disease: correlations with CAG repeat numbers. Neurol. 1998; 50(5): 1357-65.