Thursday, January 30, 2014
The National Center for Advancing Translational Sciences (NCATS)
was founded at the NIH a little over two years
ago, in the wake of increasing concern over the slow pace of converting scientific
discoveries into new therapies. "BioCentury This Week"
television sat down with NCATS director Christopher Austin to discuss how NCATS
is delivering on its promise to overcome the roadblocks and accelerate the
NCATS's mission is to re-engineer the way basic research is translated into
medicine. Supporters say NCATS will solve problems academics and industry can't
tackle on their own, but skeptics inside academia and industry and even at NIH
say it diverts funds from NIH's core research mission and is taking on tasks
that should be left to drug companies.
Christopher Austin: Right. Thanks to the Genome Project, among other things,
there are many more targets than we can possibly deal with. For example, there
are about 6,000 rare diseases, and we now know, from the Genome Project and
other advances, the genetic basis of about 4,000 of them. That's up from 50
about 15 or 20 years ago.
And can you give examples of outcomes or of how NCATS can deliver real results?
The NCATS RNAi program is focused on the general principles that underlie using
RNAi as a target validation tool. We started by using genomewide RNAi to knock
out every gene one by one and find every gene involved in disease or a cellular
process-and thereby identify targets.
Another area NCATS is working on is improving the process of getting drugs
and new therapies to people who need them-not simply getting them approved, but
actually getting them disseminated. Many biotech and pharma companies and
academics don't seem to focus much on that. What are you doing there?
This is a very important problem, as part of NCATS's mission is to improve
health. Getting a drug approved can be very important, but we haven't actually
improved health there.
So again-why is this something that NIH needs to do? A drug company has a
fantastic incentive to get new drugs used as widely as possible as quickly as
We should do it because NCATS is focused on areas where we think we can do
things better. As an example, it's well known that most patients who were
prescribed a medicine either never fill the prescription or they only take it
one month and then they stop.
Can you give us some other examples of the way that you've tried to use your
funding to meet your goal of transforming medicine-and at $10 million a year
that funding is a lot less than the half-a-billion dollars originally
We are using the money well; for example, for the Tissue Chip program-a body on
a chip to do toxicology-which, if successful, will transform how we identify
the safety and efficacy of novel therapeutics.
Going back to rare diseases, tell us about the TRND project, which is trying to
find new therapies for really rare diseases. What are you working on there, and
again why is it something that the private sector, which has invested a lot in
rare diseases, wouldn't do on its own?
There are many rare diseases that are not sufficiently de-risked for a company
to make a business case to adopt them. And the purpose of TRND is to be the
starting point of proto-drug development up to a point where a company is
willing to adopt them. So it's really an adapter or a chaperone for those
Thank you very much.
Fishburn, C.S. SciBX 7(4); doi:10.1038/scibx.2014.106 Published online Jan.
1. Haas, M.J. SciBX
2. Hasson, S.A. et al.
Nature 504, 291-295 (2013)
AND INSTITUTIONS MENTIONED
AesRx LLC, Newton, Mass.
Defense Advanced Research Projects Agency, Arlington, Va.
Food and Drug Administration, Silver Spring, Md.
Life Technologies Corp. (NASDAQ:LIFE), Carlsbad, Calif.
National Center for Advancing Translational Sciences,
National Institute of Neurological Disorders and Stroke, Bethesda, Md.
National Institutes of Health, Bethesda, Md.
NIH Center for Regenerative Medicine, Bethesda, Md.