A cache of at least 24 drugs abandoned during development by three major pharmaceutical companies will become available to scientists seeking novel therapeutic uses for them under a new, competitive awards programme, the US National Institutes of Health (NIH) and executives from the drug companies announced today.

At a press conference at the National Press Club in Washington DC, Francis Collins, the NIH director, called the pilot programme “the first signature initiative” of NIH’s new translational medicine centre — one that is timely, he said, in an era in which the “triple frustration” of long drug-development timelines, steep costs and high failure rates have increasingly jinxed the pharmaceutical industry.

The NIH, through its National Center for Advancing Translational Sciences (NCATS), will spend up to US$20 million in 2013 to finance competitively awarded projects by scientists aiming to “teach old drugs new tricks,” as Collins’s boss, Kathleen Sebelius, Secretary of Health and Human Services, put it at the press conference.  Applications for the pilot programme, Discovering New Therapeutic Uses for Existing Molecules, will be welcomed from academics, non-profit groups, biotechnology companies and others.

The companies contributing the clinic-ready drugs, and all the information that they have accrued on them, from toxicology profiles to biomarker data, are Pfizer, AstraZeneca and Eli Lilly. Donald Frail, the vice-president of AstraZeneca’s Science New Opportunities and Innovative Medicine Unit, described the “at least eight” compounds his company is contributing as “fantastically well characterized.” They come from across therapeutic areas,” he said. “Where they’re going to go to, that’s the interesting part.”

The compounds passed through preclinical testing and through phase I safety trials in humans, but were abandoned later for lack of efficacy or because the company moved out of a given therapeutic area. Rod MacKenzie, the head of pharmatherapuetics research and development at Pfizer, described the roughly 12 molecules his company will be making available as having been 5–7 years into development, with “certainly tens of millions” of dollars already invested in them.  They were drawn, he added, from a universe of dozens of clinic-ready, but abandoned, molecules at Pfizer.

The two-dozen compounds, whose particular molecular targets are already mostly known, are not intended to be tossed at myriad targets in the kind of full-bore screening that occurs much earlier in drug discovery, Collins added. “This is not a random screen. This is an intentional designed connection” between a drug and a disease.

Kathy Hudson, the acting deputy director of NCATS, who is the centre’s point person on the collaboration, said that the NCATS expects next year to award eight to ten grants, each worth up to $2 million in that first year. During that year, a successful awardee will be expected to reach certain milestones in completing preclinical work, providing evidence of the biological rationale for the drug’s new use. If these are met, that scientist will receive money for up to two additional years to undertake early phase II clinical trials establishing efficacy against the disease. The expectation is that success here will draw a commercial sponsor interested in carrying the molecule through larger, more expensive, late-stage trails.

Early details for prospective applicants are available here; more will appear in a request for applications that the NIH intends to publish later this month. The first application due date will be as soon as mid-July. Hudson said that the award competition will involve two peer-reviewed stages:  peer review of a project summary, and then peer review of a full proposal, which must include a signed agreement between the researcher and the company.

The NIH intends, said Collins, to play “matchmaker” between companies and interested scientists, in part by supplying already-developed template agreements between the companies and the scientists. Under these, the company will retain ownership of the compounds while scientists will own any intellectual property they discover through the project. The company in question will be given the right of first refusal as a licensee should a scientist develop a promising new application for a drug.  The terms of the licensing — who gets how much money — will be negotiated then, but the hope is that the user-ready templates will prevent the often-protracted delays, and sometimes complete deaths, of projects that occur when single companies negotiate deals with individual scientists.

“We wanted to set an example,” said Jan Lundberg, Eli Lilly’s executive vice-president of science and technology, “by not ending up in endless discussions about legal agreements.”

The proposed templates, on which the NIH is seeking comment from today, are available for viewing here.

Among other historical examples of failed drugs that have been resurrected to human benefit, Collins cited thalidomide’s repurposing as a drug for multiple myeloma. (It is also approved for combating leprosy.) He noted that the osteoporosis drug raloxifene has now been shown to be as effective as tamoxifen in treating invasive breast cancer.  Sebelius told the story of AZT (azidothymidine), which received US marketing approval in the 1960s as a cancer drug but failed to be effective.  In the 1980s, 25 months after it was discovered to be efficacious against HIV in the lab, the US Food and Drug Administration approved it as the first HIV drug.

“A drug rescued from obscurity 25 years ago” is the reason that HIV “will likely not be the cause of my death,” said Michael Manganiello, a patient activist who spoke at the press conference.

AstraZeneca last December launched a similar collaboration with the UK Medical Research Council and received more than 100 applications from scientists in eight weeks, Frail said.

The NIH is hopeful that the store of two-dozen molecules marks only a beginning, and that other companies will join the collaboration even as the three already participating expand the number of compounds that they make available.

The agency is looking for scientists’ feedback on the programme for 4 weeks, beginning today, in this request for information.