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NCATS' Austin Seeks to Tackle Big Bottlenecks in Biomedical Science

NEW YORK (GenomeWeb News) – What has surprised Chris Austin most as he has begun settling into his new job as director of the National Center for Advancing Translational Sciences has been the volume of eager responses the new center has received from people across the biomedical science spectrum.

He has spent much of his time answering emails and calls from "high level people in every arena of the research ecosystem" who want to know what programs NCATS has in the works and how they may be able to get involved.

Leaders from research centers, biotech and pharma companies, patients groups, foundations, and academia have been seeking Austin's ear to talk about what will be required to "mine tangible therapeutic human health value" out of the vast amount of basic research data, including the "mother lode" of information generated by the National Institutes of Health, Austin told GenomeWeb Daily News in a recent interview.

"There is no more important question" facing biomedicine, Austin said, than how to turn basic science data into deliverable drugs, diagnostics, and devices. "Everybody is worried about that problem — and that is precisely the problem that NCATS was formed to solve."

NCATS launched at the end of 2011 after a nearly year-long restructuring and persuasion effort by leadership at NIH, headed by NCATS' champion NIH Director Francis Collins. Its creation involved the dissolution of one long-standing NIH center (the National Center for Research Resources) and garnered a range of criticism. NCATS' skeptics objected that the process to create the center was too rushed, that it was an effort to compete with drug companies, and that NCRR's popular programs would get lost or ignored after being shuffled around.

When NCATS launched with a $577 million budget for its first year — with most of that coming from former NCRR programs — the center arranged itself in three main sections, with one division focused on clinical innovation, another on preclinical innovation, and an Office of the Director to house other remaining programs and pursue new ones.

Although advancing genomic medicine is not a specific focus of the NCATS mission, genome-based science will help fuel much of its activities, and it could generate new personalized medical advances as well.

The selection of Austin, with a strong background in applying genomics to discover and validate therapeutics, to run the new center suggests that genome-based research will run throughout many NCATS initiatives.

Austin, who has served as director of the NCATS Division of Preclinical Innovation since its launch, previously was a senior advisor to the director at the National Human Genome Research Institute, where he started the Molecular Libraries Roadmap Initiative and the Knockout Mouse Project. He also has previously served as a researcher focused on genome-based drug target discovery at Merck, and was director of the NIH Chemical Genomics Center and of the Therapeutics for Rare and Neglected Disease program.

With his history stretching across both institutes, Austin sees NCATS and NHGRI as addressing the same problem: how to move basic science data about diseases and health past the problematic bottlenecks that dam up the flow of innovation.

"If you think about it from the standpoint of the genome, they (NHGRI) are starting out with the genome and pushing forward. Whereas, NCATS is starting from the patient or disease side and pulling from the other direction … but it is very much the same problem," Austin told GWDN.

"One of the many translational bottlenecks," Austin said, noting that this is a point that NHGRI Director Eric Green makes frequently in his public talks, "is how do you go from the deluge of genetic data with weak associations and small effect sizes to targets that you would actually want to [work] on.

"That's a typical NCATS problem in the sense that NCATS was founded to address the many problems in the process of translation, applying to any disease," Austin explained.

He said that the center will address issues related to the clinical application of genetic and genomic data, with an emphasis on moving this knowledge into clinical trials and using it to prescribe drugs, and using stratified medicine to help create new trials that are more effective.

Austin expects that NCATS will play a role in an NIH effort to identify and validate drug targets that was launched last year and seeks to develop strategic tools for a harmonized approach for target validation, to identify targets more swiftly and efficiently, and to provide a pre-competitive collaborative environment in which validation efforts can occur.

NCATS also is running a genome-wide RNAi initiative, which currently is only available to intramural investigators because of funding constraints, he said.

"We are creating [an RNAi] database that will be available to everybody, and my hope is that as NCATS grows we will be able to get the funding to make this open to everybody," he explained.

Efforts like this and others at NCATS will pursue the larger, multifaceted problems facing genomic medicine, the ones that nag the biomedical research community, said Austin.

"The issue here is the issue that I came to NIH 10 years ago to [pursue], which is, "How do you identify and derive therapeutic potential out of the genome?"

He said that the pursuit of usable, applicable knowledge in the genome is a bit like searching for oil. Early successes such as Mendelian disorder discoveries suggested that mining the genome would be like pumping away at a vast sea of oil near the surface.

"But as those targets have gotten exhausted, we have had to use more and more complicated methods to extract the remaining value out of the genome, much like people are doing now in oil shale, such as fracking," Austin said.

New Kind of Center

As a new institute on a new kind of mission, what will be the measures of NCATS' success? Most other NIH centers and institutes have a prime objective — to cure cancer at the National Cancer Institute, to fix eye diseases at the National Eye Institute, etc. But NCATS is addressing a different type of problem altogether, and Austin said that there will not be one type of measuring stick for analyzing its success.

"We're going to have a portfolio of metrics, some of which are going to be long term and some are going to be short term, and they're going to be across the translational spectrum," he said. "NCATS is going to work in every area, from genome-based target discovery or target validation, through assay development, screening, chemical informatics, bioinformatics, medicinal chemistry, preclinical development, clinical pharmacology, clinical trials, and regulatory science … a tremendously broad palette across the translational spectrum."

Success at the center certainly will not be based on how many new drugs are approved each year, Austin said.

"If you have that as your goal then NCATS would have the same goal as thousands of other organizations around the world that are trying to do that. The last thing the world needs is another organization that is 100 percent focused on getting a drug through the FDA, that's what biopharma does," he said.

It is precisely because NCATS is not focused on a single disease area that it will be able to "study the process and the science underlying translation as a discipline in itself, the process of translation as a discipline," Austin said.

With other research organizations and institutes all focused on their separate problems, no one so far has taken ownership of working out the kinks that jam up the biomedical research system as a whole, according to Austin.

"Who owns the regulatory science problem? Who owns the harmonization of IRBs problem? Who owns the clinical trial recruitment problem? Who owns the preclinical toxicology problem? Well, no disease-specific organization – that's not their day job, which is curing that disease," said Austin. "So, nobody feels or has felt up until this point that it has been job number one for them. And when you look at the ecosystem, this is what has gotten us in large part into our current pickle."

The problem is like "everybody building towns, but nobody building roads to get from one town to another," he said. "What you need is an organization that is … worried about how we make all of these pieces work together, and if you do that, that is when magical things happen. That is what happens with transportation systems, and that is the sort of thing that NCATS is going to do with in the research ecosystem."

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