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NIH Launches New Screening Center: Aims Are Broader Than Drug Discovery


The US National Institutes of Health’s new Chemical Genomics Center, which launched last week, seems at first like a foray into public-sector drug discovery.

The center, which is to be led by Chris Austin, the NIH’s senior advisor for translational research and a former Merck official, plans to conduct high-throughput screening of over 100,000 small-molecule compounds in its first year, according to the NIH. As part of the Molecular Libraries and Imaging working group of the trans-NIH Roadmap for Medical Research established last year, it will also be the flagship in a consortium of ten chemical genomics screening centers, the first of which are slated to screen 600,000 compounds in their first three years of operation, according to the RFA for the centers.

The goal of the network, however, is not just to mimic the first segment of the pharmaceutical pipeline. Instead, the hope is to explore a wider swath of molecular territory that the NIH believes could be of more value in elucidating the biological mechanisms of disease — and lead to new targeted treatments.

“The grand vision of the whole network of centers is to find small molecules, chemical substances that can modulate the activity of all the gene products of the genome, which is a very tall order,” said Jim Inglese, who was hired last month as the center’s head of biomolecular screening.

The small-molecule library generated by the network will be stored in a publicly accessible repository, the NIH Small Molecule Repository, and the chemical genomics data that this network produces will be deposited into a publicly accessible database, PubChem, which is to be managed by the National Center for Biotechnology Information.

The compounds will come from a variety of sources — academic laboratories and commercial entities — and will include not only those that are of interest to drug companies, but also peptides, metabolites found within cells and organs, and natural products, according to Inglese. “The source of these molecules will be hopefully from everywhere you can imagine. In that way it’s broader than what you can find in a pharmaceutical compound collection, because we’re interested in a broader picture than just compounds that can make good drugs.”

Inglese characterized the compounds as “probes that help illuminate biology and targets that are potentially involved in disease so that one can go and do a screen and look for, specifically, drug molecules.”

National Human Genome Research Institute Director Francis Collins has indicated that the efforts of the network will be directed toward pharmaceutically relevant compounds. “What we are doing is simply giving academic and government researchers a chance to contribute in a much more vigorous way to the earliest stages of the drug development pipeline: the identification of useful biological targets,” Collins stated in the press release announcing the center’s launch. “This is a win-win situation for basic biology, for the drug industry, and, most importantly, for the American public,” he said.

Speaking with Pharmacogenomics Reporter, Austin provided further clarification. While some press reports have indicated that NIH is, in fact, becoming a drug developer, , Austin emphasized that the effort will not be aimed principally at developing compounds that are to be made into drugs, but, as Collins’ said, will focus on those that have potential utility in providing more information about the drug targets to come out of the genome.

“We are not going to look at any kinds of pharmacologic processes” related to the compounds, such as oral bioavailability, and toxicity. “About 95 percent of the time, and about 98 percent of the cost of drug development is after we stop.” But the information on potential drug targets generated from the network will be useful for drug development, he said. “To the extent that the scientific community knows more about targets before they go into traditional drug development, our hope is that the failure rate will be lower, as the more you know about your target, the more likely you are to succeed.”

Because of the potential for the compounds screened at the chemical genomics screening centers to be used as drugs, in fact, the NIH has proposed a deviation from its standard patent rights clause that would limit the rights of investigators at these centers to retain the title to inventions in which proprietary compounds are used. This clause, which is designed to encourage private companies to use the repository of compounds as well as the assay technology that comes out of the networks, would give the provider title or exclusive rights to the inventions. The NIH has not decided yet whether or not to use this clause, and is seeking public comments on it.

In a May 28 meeting, NIH officials met with outside consultants to discuss the IP issues surrounding the compound repository, the assays, and the network, Austin said. “I can’t say we came to any clear conclusions.” The NIH plans to release a guidance document in the next month, he said. “If I had to guess, what I think we will end up with is a situation where we will have general guidelines and then there will be exceptions, possibly, for special cases in one direction or another, because I don’t think there’s going to be a one-size-fits-all” solution.


While Austin and others hope that successful resolution of these IP issues will make the fruits of the chemical genomics screening centers more attractive to drug developers, the initiative does already provide a clear short-term opportunity to drug screening tool developers. The RFA for the Small Molecule Repository has closed ( /grants/guide/notice-files/NOT-RM-04-003.html), but the winner has not been announced, and the RFA for extramural centers does not close until August 24 and is open to both academic and commercial applicants (See The grant awards are expected to be made in March 2005, with total awards of $20 million from FY 2005 to go to at least six centers for the first pilot phase.

Additionally, the Molecular Libraries Initiative includes a technology development initiative.

“[W]e’re taking a lot of the technology and expertise that’s been developed, primarily in the pharmaceutical and biotech industry to uncover the seeds for drugs, the lead compounds, and incorporate it into these centers,” Inglese said.

Last week, the NIH announced that it had signed on Kalypsys, of San Diego, to build a high-throughput and pathway screening system for the NIH Chemical Genomics Center, in a contract that could be worth $30 million for Kalypsys. The system is slated to include robotic and liquid handling technologies that can dispense cells or proteins onto 4-by-6 inch 1,536-microwell plates, and is expected to be able to screen over a million compounds a day, as well as whole model organisms such as zebrafish embryos, nematodes, and yeast; and certain types of mammalian cells.

Kalypsys is now assembling the system at its plant in San Diego, and will test it out before disassembling it and shipping it to the NIH for testing at the site, said Inglese. This system will be the major piece of equipment for the Center.

Meanwhile, the Center has to find a site, which is to be located outside of the NIH campus, but most likely in the immediate area within some biotech space. To run the operation, the plan is to hire 45 to 50 scientists and other staff over the next three years. “My major role at the moment is to identify personnel,” said Inglese. Initial hires will likely be scientists in biomolecular scereening and informatics, and a head of the chemistry division.

According to the NIH’s release last week, the Center plans to start screening compounds by the end of the year. But Inglese emphasized that this is only a goal. “We’re still looking for space, we’re hiring people, we need to put an infrastructure together, we need the Kalypsys system to be built and assembled, but we would like to initiate a preliminary operation by the end of the year,” he said.


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