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SNPtech With PGx in its Sights, Is NIH the Newest Drug Developer?


Before the advent of laser-guided missiles, battle ships were equipped with cannons that weighed 2,000 tons and required dozens of sailors to muscle them toward their target. The National Human Genome Research Institute, as it transitions from sequencing genes to understanding how they work, moves in much the same lumbering way — though without the sailors.

These days, the NIH, and especially NHGRI, is starting to turn its focus toward functional genomics, and has formally begun to “closely examine” how its resources can be applied to pharmacogenomics, according to an NHGRI official.

“The guns here have been focused very squarely on the sequence, and now they’re moving to apply that same kind of energy … to the translational problems,” says Christopher Austin, who advises Francis Collins on translational research issues. “The question now facing NIH, and especially NHGRI, is, ‘When we have [those data], what will we do with them?’”

One of the boldest new steps the NIH plans to take — which may also carry the greatest promise for pharmacogenomics — is the decision to extend its reach down the drug-development pipeline. The NIH “is taking a very serious look at how it can contribute to drug development,” says Austin, a former Merck official.

Indeed, the agency has never been invested in therapeutic development. Traditionally, the NIH has focused on basic research, and has left the translational and therapeutic-development aspects — those areas of drug discovery and development closely linked to pharmacogenomics — to the private sector.

“I think that has served everybody well,” Austin says. “However, I think a number of things have changed, which has led the private and public sectors to rethink how this relationship has worked.”

These days, the NIH funds drug-discovery research in the earliest stages of target identification. Now, with gobs of pharmacogenomics and chemical genomics data and technologies at its disposal, the agency wants to expand its role to fund assay development and high-throughput screening. Collectively, this stretch of the drug-development pipeline covers at least three years, and ushers compounds into lead optimization and toxicology studies.

For the first time, Austin says, the NIH is “beginning to think, in a broad, trans-NIH way, of a way to get itself interested in those applications that will get it closer to patients.” Drug makers “may have more of a partner in NIH than [they’ve] had before.”

“There is no way that the private sector can do all of the target validation studies,” he says, adding that the agency will collaborate with individuals in the private sector, both from biopharma and from the tool space. “The NIH must help here. The question is, ‘What should NIH do that will be enabling to the private sector … while at the same time focusing on areas that the private sector is not interested in, such as orphan diseases,” he says. The NIH believes this goal is at least three years out, he adds.

Private-sector labs met the new NIH goals with optimism. “I think it is a natural extension from the Human Genome Project to move forward, in their attempt to bolster emphasis in this area [of pharmacogenomics],” says Mark McCamish, chief medical officer of Perlegen Sciences. “It pushes the science and the potential applications” of pharmacogenomics technologies.

McCamish suggests that the serendipitous timing of NIH’s goals and the FDA’s newly released draft guidance on pharmacogenomics bodes well for the discipline. “To get those two working together would be exciting,” he says.

“The FDA really is intrigued about the science of pharmacogenomics, and wants to understand it better and actually help apply this technology in their evaluation” of drug candidates, McCamish notes. “The NIH, on the other hand, is looking to create some technologies, and to foster the use of these technologies. The NIH may in fact help us to be able to scientifically develop and refine these technologies.”

“This is an open window, and we need to make things happen,” he says.

An expanded version of this story appeared in the December 4, 2003, edition of SNPTech Pharmacogenomics Reporter.

Kirell Lakhman is editor of SNPTech Pharmacogenomics Reporter, a weekly newsletter from GenomeWeb at www. He can be reached at [email protected]


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