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NCATS Launches Drug Repurposing Project with Big Pharma, Investigators

NEW YORK (GenomeWeb News) – Launching what will be the maiden initiative for the months-old National Center for Advancing Translational Sciences, National Institutes of Health Director Francis Collins today unveiled a program that will support researchers using biomarker-based studies and other approaches to repurpose compounds owned by three of the world's largest drug developers.

At a press conference today, Collins joined executives from Pfizer, AstraZeneca, and Eli Lilly to lay out a plan that will enable researchers to use NIH funding to study new uses for more than 20 drugs that have already been safety-tested in humans but which have been set aside by the three drugmakers for one reason or another or were not approved.

The projects to re-assess these compounds will likely lean heavily on biomarkers that can be used to find new ways of measuring their applicability and usefulness.

"In this pilot phase, the program will match US researchers with dozens of these companies' compounds to explore how they might be put to new uses in treating patients. And NIH is happy to serve as the matchmaker," Collins said at the press conference.

"Tens of millions of dollars and years of work have already been invested in each compound. They have already cleared safety testing in humans, but did not prove effective for the specific disease or condition for which they were developed, or they were not pursued for business reasons," he explained.

"The goal is simple: to see whether we can teach old drugs new tricks," US Department of Health and Human Services Secretary Kathleen Sebelius said. "And we are taking an innovative approach, crowd-sourcing these compounds to our brightest minds and most inventive companies.

"Out of thousands of compounds that enter the drug development pipeline only a very few will ultimately make it to approval," she said. "The drug AZT, for instance, was first tested against cancer, and failed. Only later, it was discovered to be a very effective treatment for HIV, the first medicine we found to work against the virus."

Sebelius also said these compounds offer a "big advantage" over many that are still in the lab, because they "have already cleared many of the clinical and regulatory hurdles" and have "cleared the toxicity hurdle."

The project offers a glimpse at the vision Collins had when he swiftly created NCATS last year, and pushed against opposition for it to be funded in 2012.

To kick off the Discovering New Therapeutic Uses for Existing Molecules program, NCATS plans to release a new funding opportunity sometime this month that will provide up to $20 million in 2013 through two types of cooperative agreements to fuel the new projects.

Applications for the program will be peer-reviewed, and they should propose milestone-driven pre-clinical or clinical trial-based projects that could be used to guide "go/no go" decisions on these compounds.

The one-year long pre-clinical studies will use the selected agents to provide sufficient evidence for the new therapeutics use. Those studies could involve biomarkers that link the agent's target or pathway to a disease pathology or to a change in the disease state in patients, or in vitro, in vivo, or ex vivo assays or models of human disease that are critical for making go/no go decisions.

The clinical trials studies will fund two-year projects that are biomarker-driven and can establish the relationship between the magnitude and duration of target modulation and clinical efficacy. NCATS plans to encourage projects involving pharmacogenomics, patient-stratification strategies based on molecular disease markers, or studies involving applicable biomarkers.

The data from these projects should provide evidence that the drug candidate is safe, modulates the target mechanism, and has efficacy in the disease population. That kind of knowledge would help de-risk further development of the selected drug candidates.

Collins said the whole initiative hinges on the use of "innovative" template agreements between the drugmakers and investigators that will enable all parties to streamline the legal and administrative issues involved in sharing and using proprietary compounds and data.

He said these standard template agreements will "significantly reduce the amount of time it takes for each of the partners to negotiate the terms" of the collaboration. "Too often, it has taken longer to negotiate the terms of the agreement than to do the research," he said.

The agreements will enable the pharmaceutical companies to retain ownership of their compounds, while the research partners will own any intellectual property they develop and will be able to publish their results.

Collins summed up the program as "a win-win-win. It is a win for investigators, who get access to valuable compounds for new uses. It is a win for companies, whose compounds have a chance for new applications and new life. And, most importantly, it is a win for patients, whose hopes for discoveries of new treatments are significantly advanced by this partnership."

Collins added that he hopes that the "pioneering spirit" shown by Pfizer, Astrazeneca, and Eli Lilly will encourage other companies "to join us in this important effort."

"That invitation is wide open," he said.

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