Enabling Anyone to Translate Clinically Relevant Ideas to Therapies (original) (raw)

Abstract

How do we inspire new ideas that could lead to potential treatments for rare or neglected diseases, and allow for serendipity that could help to catalyze them? How many potentially good ideas are lost because they are never tested? What if those ideas could have lead to new therapeutic approaches and major healthcare advances? If a clinician or anyone for that matter, has a new idea they want to test to develop a molecule or therapeutic that they could translate to the clinic, how would they do it without a laboratory or funding? These are not idle theoretical questions but addressing them could have potentially huge economic implications for nations. If we fail to capture the diversity of ideas and test them we may also lose out on the next blockbuster treatments. Many of those involved in the process of ideation may be discouraged and simply not know where to go. We try to address these questions and describe how there are options to raising funding, how even small scale investments can foster preclinical or clinical translation, and how there are several approaches to outsourcing the experiments, whether to collaborators or commercial enterprises. While these are not new or far from complete solutions, they are first steps that can be taken by virtually anyone while we work on other solutions to build a more concrete structure for the “idea—hypothesis testing—proof of concept—translation—breakthrough pathway”.

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ACKNOWLEDGMENTS AND DISCLOSURES

SE owns stock in Scientist (Formerly Assay Depot). SE is the CEO of Collaborations Pharmaceuticals, Inc. and Phoenix Nest, Inc.

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Authors and Affiliations

  1. Collaborations Pharmaceuticals, Inc., 5616 Hilltop Needmore Road, Fuquay-Varina, Noth Carolina, 27526, USA
    Sean Ekins
  2. Phoenix Nest, Inc., P.O. BOX 150057, Brooklyn, New York, 11215, USA
    Sean Ekins
  3. Department of Neurology, Los Angeles Biomedical Research Institute, Torrance, California, 90502, USA
    Natalie Diaz, Paul Mathews & Aaron McMurtray
  4. Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, California, 90095, USA
    Natalie Diaz, Paul Mathews & Aaron McMurtray
  5. Department of Neurology, Harbor-UCLA Medical Center, Torrance, California, 90509, USA
    Natalie Diaz & Aaron McMurtray
  6. Department of Psychiatry, Los Angeles Biomedical Research Institute, Torrance, California, 90502, USA
    Julia Chung
  7. Department of Psychiatry, Harbor-UCLA Medical Center, Torrance, California, 90509, USA
    Julia Chung
  8. Department of Psychiatry, David Geffen School of Medicine, University of California, Los Angeles, California, 90095, USA
    Julia Chung

Authors

  1. Sean Ekins
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  2. Natalie Diaz
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  3. Julia Chung
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  4. Paul Mathews
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  5. Aaron McMurtray
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Correspondence toSean Ekins.

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Funding

Joseph Stahlberg Foundation grant to Dr. Aaron McMurtray.

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Ekins, S., Diaz, N., Chung, J. et al. Enabling Anyone to Translate Clinically Relevant Ideas to Therapies.Pharm Res 34, 1–6 (2017). https://doi.org/10.1007/s11095-016-2039-5

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