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Pioneering government-sponsored drug repositioning collaborations: progress and learning


A new model for translational research and drug repositioning has recently been established based on three-way partnerships between public funders, the pharmaceutical industry and academic investigators. Through two pioneering initiatives — one involving the Medical Research Council in the United Kingdom and one involving the National Center for Advancing Translational Sciences of the National Institutes of Health in the United States — new investigations of highly characterized investigational compounds have been funded and are leading to the exploration of known mechanisms in new disease areas. This model has been extended beyond these first two initiatives. Here, we discuss the progress to date and the unique requirements and challenges for this model.

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  1. Ashburn, T. T. & Thor, K. B. Drug repositioning: Identifying and developing new uses for existing drugs. Nat. Rev. Drug Discov. 3, 673–683 (2004).

    Article  CAS  Google Scholar 

  2. Cook, D. et al. Lessons learned from the fate of AstraZeneca's drug pipeline: a five-dimensional framework. Nat. Rev. Drug Discov. 13, 419–431 (2014).

    Article  CAS  Google Scholar 

  3. Mullard, A. Could pharma open its drug freezers? Nat. Rev. Drug Discov. 10, 399–400 (2011).

    Article  CAS  Google Scholar 

  4. Carroll, J. Washington U gets second look at Pfizer molecules. Fierce Biotech [online], (2010).

    Google Scholar 

  5. Collins, F. S. Mining for therapeutic gold. Nat. Rev. Drug Discov. 10, 397 (2011).

    Article  CAS  Google Scholar 

  6. LaMattina, J. The NIH is going to discover drugs... Really? Forbes [online], (2012).

    Google Scholar 

  7. Colvis, C. M. & Austin, C. P. The NIH-industry New Therapeutic Uses pilot program: demonstrating the power of crowdsourcing. Drug Repurp. Rescue Repos. 1, 15–16 (2015).

    Google Scholar 

  8. Southan, C., Williams, A. J. & Ekins, S. Challenges and recommendations for obtaining chemical structures of industry-provided repurposing candidates. Drug Discov. Today 18, 58–70 (2013).

    Article  CAS  Google Scholar 

  9. Kaufman, A. C. et al. Fyn inhibition rescues established memory and synapse loss in Alzheimer mice. Ann. Neurol. 77, 953–971 (2015).

    Article  CAS  Google Scholar 

  10. Strittmatter, S. M. Safety and tolerability of AZD0530 (Saracatinib) in Alzheimer's disease. [online], (2014).

    Google Scholar 

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The UK Medical Research Council (MRC) and the US National Institute of Health (NIH) initiatives were supported by a large range of individuals within the MRC and the NIH as well as external investigators, and investigators from AstraZeneca and Medimmune. At the risk of excluding many, in particular the authors would like to thank J. Latimer (MRC), C. Colvis and B. Dunn (NIH–NCATS), G. Wilkinson, C. Wilks, A. Longton, S. Curran, K. Hickling and the members of the New Opportunities Emerging Innovations Unit (AstraZeneca). The authors would like to acknowledge the contribution made by all those involved and the exciting ideas and proposals received from academic investigators.

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Correspondence to Donald E. Frail.

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M.N.P., C.D.W., H.J.S., M.B. and K.J.E are employees of AstraZeneca, D.E.F. is an employee of Allergan and C.W. is an employee of the UK Medical Research Council.

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List of AstraZeneca compounds offered in the MRC, NIH and NRPB initiatives (PDF 84 kb)

Supplementary information S2 (table)

Example of an AstraZeneca compound summary used in the MRC 'mechanisms of disease' call for proposals (PDF 102 kb)

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Frail, D., Brady, M., Escott, K. et al. Pioneering government-sponsored drug repositioning collaborations: progress and learning. Nat Rev Drug Discov 14, 833–841 (2015).

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