Turning Scientific Discoveries Into Treatments

Question: What is some of the most exciting research the NIH is currently working on?

Francis Collins:
We are experiencing right now a remarkable deluge of discovery in terms of the causes of disease, much of it coming out of genomics, the ability to pinpoint at the molecular level what pathway has gone awry in causing a particular medical condition.  And that in itself is exciting because it’s new information, but what you really want to do is to take that and push that forward into clinical benefit.  

Some of that could be in prevention by identifying people at highest risk and trying to be sure they are having the right preventive strategy.  But people are still gonna get sick, and so you want to come up with also, better treatments than what we have now.  How to do that has largely been left to the private sector in the past, basically private sector has made their whole business out of identifying possible ways of coming up with a therapeutic, which is often a small molecule an organic compound that would have just the right properties to improve the situation.  

In the past, those were derived rather empirically, just trying thing to see what worked and not always knowing why it worked.  More recently because we do have a better handle on what’s going on inside a cell and how a disease affects that, there are rational strategies for screening a very large library of chemical shapes trying to find the one that’s got the right properties.  This is a high throughput screening approach.  

NIH has gotten much more involved in that in the last six or seven years and many academic investigators who are really unfamiliar with these steps towards therapy have gotten pretty excited about being able to take their basic discovery and move it in the direction of a therapeutic.  But it’s a long path, it’s one thing to have a compound that works in a petri dish, that looks like it might potentially have the right properties to treat a disease.  The idea of actually giving that to a patient means you’ve got a lot more work to do in terms of testing its toxicity in an animal, its ability to be metabolized and absorbed.  All of those which are long, expensive processes.  And in that degree, that’s called ‘The Valley of Death’ and that’s where a lot of projects die.  

NIH is now pushing very hard to provide bridges across that “Valley of Death” for carefully chosen project so they don’t stop at that point.  Not that we’re competing with the private sector.  We wouldn’t undertake projects of that sort that the private sector is already going after, but for diseases that are relatively less common; the economic incentive is just not there to push these things closer to the clinic.  

So through new programs, particular one called The Cure is Acceleration Network, we are investing in that process and making it possible for academic investigators to go much further down that pipeline towards a therapeutic, and we have clinical centers scattered all over the country; about 60 of them, plus the largest of them right here at NIH with 240 research beds that are set up to do those initial clinical trials to see if the drugs work.  We built a much stronger relationship with the FDA than has ever been in place before to try to be sure there’s a synergism there between the development of these compounds and their oversight.  And we’re optimistic.  This is gonna change the paradigm in partnership with the private sector to get more effective treatments out through this pipeline and approved by the FDA and into the hands of the public even for conditions that are not that common.  

We can’t wait for the next blockbuster, there aren’t going to be very many blockbusters.  Diseases are actually being broken apart into subsets by molecular understanding, which means that the block buster model is getting less and less viable.  But if we want to see program in therapeutics, NIH has to play a larger role.  And we embrace that.  And that for me is one of my highest priorities while I am the director.


Recorded September 13, 2010
Interviewed by David Hirschman

Image courtesy of Shutterstock

NIH has to play a larger role in working with the private sector to get more effective treatments through the development pipeline, approved by the FDA, and into the hands of the public.

China’s artificial sun reaches fusion temperature: 100 million degrees

In a breakthrough for nuclear fusion research, scientists at China's Experimental Advanced Superconducting Tokamak (EAST) reactor have produced temperatures necessary for nuclear fusion on Earth.

Credit: EAST Team
Surprising Science
  • The EAST reactor was able to heat hydrogen to temperatures exceeding 100 million degrees Celsius.
  • Nuclear fusion could someday provide the planet with a virtually limitless supply of clean energy.
  • Still, scientists have many other obstacles to pass before fusion technology becomes a viable energy source.
Keep reading Show less

Project 100,000: The Vietnam War's cruel and deadly experiment

Military recruits are supposed to be assessed to see whether they're fit for service. What happens when they're not?

Flickr user Tommy Truong79
Politics & Current Affairs
  • During the Vietnam War, Robert McNamara began a program called Project 100,000.
  • The program brought over 300,000 men to Vietnam who failed to meet minimum criteria for military service, both physically and mentally.
  • Project 100,000 recruits were killed in disproportionate numbers and fared worse after their military service than their civilian peers, making the program one of the biggest—and possibly cruelest—mistakes of the Vietnam War.
Keep reading Show less

Here's how diverse the 116th Congress is set to become

The 116th Congress is set to break records in term of diversity among its lawmakers, though those changes are coming almost entirely from Democrats.

(Photo: MANDEL NGAN/AFP/Getty Images)
Politics & Current Affairs
  • Women and nonwhite candidates made record gains in the 2018 midterms.
  • In total, almost half of the newly elected Congressional representatives are not white men.
  • Those changes come almost entirely from Democrats; Republican members-elect are all white men except for one woman.
Keep reading Show less