NCATS finds new tricks for old dogs

NIH launches industry-government collaboration with Big Pharma to repurpose clinical-stage drugs

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BETHESDA, Md.—The U.S. National Institutes of Health (NIH)last month announced a landmark partnership with three top pharmaceuticalcompanies that gives researchers across the United States a chance to explorethe potential of dozens of stalled clinical-stage compounds to treat a varietyof diseases and conditions.
The Discovering New Therapeutic Uses for Existing MoleculesInitiative will give American scientists access to select compounds fromAstraZeneca PLC, Eli Lilly & Co. and Pfizer Inc.
The goal of the program, as stated by U.S. Health and HumanServices Secretary Kathleen Sebelius during a press conference, "is simple: tosee whether we can teach an old drug new tricks."
For the NIH—and its newest initiative, the National Centerfor Advancing Translational Sciences (NCATS)—the collaborative program willboth spur therapeutic development and bridge the gap between industry andacademia/government.
"We're really hoping this collaboration will be verybeneficial to all of the NIH's institutes," says Linda Brady, director of theNational Institute of Mental Health's Division of Neuroscience and Basic BehavioralScience. "This pathway for how to negotiate and streamline interactions betweenthe public and private sectors will be beneficial to the entire researchcommunity and enable researchers and Big Pharma to learn more about clinicaltarget validation in humans."
The program also gives the pharmas an opportunity to explorenew uses for clinical-stage molecular compounds that did not prove effectivefor their initial use—but the companies are quick to note it's not merely adrug-repurposing program.
"This is not about a narrow sense of repurposing a molecule,but how to foster a collaborative spirit—getting scientists and industrytogether to talk about science, which tends to promote science," says SteveFelstead, head of clinical research for Pfizer's PharmaTherapeutics division."This gives us the opportunity to initiate research contacts across the UnitedStates. What we hope we will find are collaborators who we have not worked withbefore coming to us and forming new relationships with our principalinvestigators."
"As is often the case for anyone involved in innovation,we'll develop something and have a specific intent in mind, which is stronglylinked to areas in which we have strong therapeutic and R&D expertise,"adds Jamie Dananberg, vice president of translational science and tailoredtherapeutics at Lilly. "But as in other industries, sometimes your initialideas don't work out, but this doesn't mean the target is not a useful one fordisease. This was a chance for us to rethink what we do with molecules thatlook like good chemical entities and targets in areas we haven't thought ofbefore. With the broad range of experience in the academic world, this opens upa new frontier of possibilities for us."
"I think this is an entirely new partnership scheme for drugdevelopment," adds Don Frail, vice president of Science and New OpportunitiesiMed at AstraZeneca. Frail's unit seeks early drug discovery and developmentopportunities outside of the company's current disease area focus as well as additionalopportunities for the company's current R&D projects using both internalresources and external partnerships.
"While it does initially focus on finding new uses for thesemolecules, how you do that really serves as a template for new drug developmentpartnerships," Frail adds.

"The combination of our strength and expertise with molecules and theexperience of investigators across the NIH population can identify the bestrationale for further testing of these compounds."
Beginning in fiscal year 2013, NCATS will provide up to $20million to fund two- to three-year research grants for preclinical validationand clinical feasibility studies. The pilot program incorporates templateagreements designed to streamline the legal and administrative process forparticipation by multiple research organizations, and additionally provides aroadmap for handling intellectual property (IP) used in or developed throughthe program. The pharmas will retain the ownership of their compounds, whileacademic research partners will own any IP they discover through the researchproject, and retain the right to publish the results of their work.
AstraZeneca, Lilly and Pfizer will make more than 20compounds and their related data available to a broad spectrum of biomedicalresearchers, who will then test their effectiveness against a variety ofdiseases and conditions. The pharmas are mum for now about the specificcompounds to be explored, as the NIH and NCATS are still vetting them. The NIHis expected to release details on the pilot phase compounds sometime thisquarter.
The companies were selected to participate in the pilotphase of the program from their participation in a workshop administered lastyear by the NCATS. Additional companies may join and contribute their compoundsfor further investigation.
"This kind of partnership takes advantage of areas whereeach of us are strongest," says Dananberg. "The pharma industry is strong inproducing actual molecules that could be drugs, and we have the initialdevelopment machine capable of doing exploration of safety, tolerability andefficacy of drug targets in specific diseases areas in which we have expertise.On the academic side, they have an amazing breadth of expertise. If you couplethose two things together, you have a creative way of exploring areas ofdiseases that most pharma companies might not pursue otherwise."
Ideally, the program will advance compounds across a broadspectrum of indications and disease areas supported by the NIH, says Brady.
"Our challenge is to bring these molecules into the rightarena to address the right problems in humans," she says. "This is importantright now because certain therapeutic areas, such as neuroscience, are provingto be challenging when trying to bring new therapeutics forward."

The mission of the National Center for AdvancingTranslational Sciences (NCATS) at the National Institutes of Health (NIH) is"to catalyze the generation of innovative methods and technologies that willenhance the development, testing and implementation of diagnostics andtherapeutics across a wide range of human diseases and conditions." NCATS wascreated in December 2011 by the Consolidated Appropriations Act of 2012, whichamended the Public Health Service (PHS) Act to include the center.
A working group of the NIH Advisory Committee to theDirector, comprised of experts from industry, private equity firms, nonprofitorganizations and academia, identified a need to streamline translationalsciences. Priority areas include research on biomarkers; predictive toxicity;target validation; regulatory science; and de-risking the therapeutic pipeline.
As with all of NIH, the budget for NCATS is based on thefiscal year appropriation provided by Congress and the president. Fiscal year2012 appropriation for NCATS is $574,713,000, plus funding provided by the NIHCommon Fund and other sources.

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