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The NIH Director

NIH Sets Forth Vision for Reengineering Translational Science (Podcast Transcript)

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Hello I'm Dr. Francis Collins, the director of the National Institutes of Health. It's an exciting time in biomedical research. There's been a deluge of discoveries in the last few years about the molecular basis of diseases, from rare diseases to common ones to neglected diseases of the developing world. For all of those, we're using some of the new tools to uncover at a much more precise level than we've ever had before knowledge about why that disease strikes certain people. In fact, there are over 4000 diseases for which we now have information, many of those just really discovered in the last few years. The bad news is we only have therapies available for a few hundred of those and the rest are waiting there for something to be done.

This seems like a great scientific opportunity and yet it's a challenge because the pathway from discovering the molecular cause of a disease to developing a treatment and getting it tested and approved often stretches out to 10 or 15 or 20 years, and the success rate in actually succeeding at that is also dauntingly low. It seems like time to look at that issue of developing new drugs and see if there might be a better way, and that's what NIH now is proposing to do. It's to form a new entity called the National Center for Advancing Translational Sciences and bring together in this organizational structure some of the best and brightest minds to see if there's a way that we could look at that therapeutic development pipeline in a new way. Maybe like an engineer would, identifying where the bottlenecks are and trying to apply some of those really remarkable innovative skills of the scientific community to see if we could knock down some of those barriers.

Well what am I talking about? What kinds of things could be done? One of the things that we could do now with the computation capabilities that we have is instead of doing an exhaustive empirical screen to try to find the drug that looks like it might work in a disease, use what we've learned about the structures of proteins and other molecular information to do drug design on a computer, a virtual drugs design. Now efforts of that sort have been getting underway for some years, but it might be time to really push that forward in much more aggressive way.

Another idea, the way we currently decide whether a drug is safe to test in humans is the way we've been doing it for a long time, testing it animals. You could say it's tried, but I wouldn't say it's always true because actually there are times where that doesn’t give a good prediction. Maybe we could do better. Maybe we could use the new developments in tissue engineering to use human cells growing in laboratory dishes to assess whether a particular drug is going to be safe or not in a much closer representation of what would happen in a human patient, that would be quite valuable. If it could be shown to be highly predictive, it would also be faster and cheaper.

Another idea, there are thousands of drugs that have already been approved, that had already been tested in humans and found to be safe, but many of them were only tested for one particular application. As we're learning more about the molecular basis of disease, we're beginning to see overlaps in the pathways that are involved so that a drug developed for nausea might turn out to actually be effective for cancer, that's happened for instance. Or a drug developed for cancer might turn out to be effective against HIV, that's happened too. But most of those experiences have been serendipitous, kind of good luck. Hey, maybe we could take this on in a much more systematic way and see whether this kind of repurposing could be applied on a much larger scale.

And then what about the way in which we do clinical trials? The traditional way is you identify patients with a particular condition, you randomize them to either get the new treatment or the standard treatment, and then you watch to see what happens. It takes a long time, requires a lot of patience, doesn’t often take into account the differences between individuals that might be really important. New clinical trial designs that allow you to do this in a much more creative way could allow us to get answers to whether a new drug is safe and effective much faster and more cheaply and those also could be a goal of NCATS.

So, this National Center for Advancing Translational Sciences or NCATS could emerge at just the right moment to be able to catalyze some of these new ideas. Don't get me wrong. This is not turning NIH into a drug development company, no. NCATS would not compete with efforts that are already going on at NIH within some of our large institutes like the Cancer Institute and the Infectious Disease Institute. It would be complementary to those. It would not compete with what's going on in the private sector because that also is a critical partner for us and one that we'd want to work closely together with. And many individuals in the private sector are pretty excited about this new model for a partnership with NIH.

NCATS would not take away from support for basic science, which has been a mainstay of NIH scientific portfolio for decades and responsible for 130 Nobel Prizes to our grantees. That's a critical part of what we do. But I think it's fair to say that that basic science has been successful now that it's produced this wonderful set of new opportunities to take those discoveries and translate them into clinical applications that are going to benefit people. NIH has a long history of improving human health. The incidence of heart attack and stroke has dropped by more than 60% on the basis of NIH research. We want to keep extending that reach. We want to keep adding to the ability of individuals to live and healthy lives. We want to be the National Institutes of Health, but we also want to be the National Institutes of Hope. NCATS is just one more way that we hope to achieve that goal.

Thanks for listening.

Date: 7/6/2011

Sound Bite: Dr. Francis Collins

Additional Info: NIH Sets Forth Vision for Reengineering Translational Science

This page last reviewed on August 31, 2011

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