Enabling Anyone to Translate Clinically Relevant Ideas to Therapies

• Published in: Pharmaceutical research 2017, Vol.34 (1), p.1-6
• Main Authors: Ekins, Sean, Diaz, Natalie, Chung, Julia, Mathews, Paul, McMurtray, Aaron
• Format: Article
• Language: English
• Subjects: Animals; Biochemistry; Biomedical and Life Sciences; Biomedical Engineering and Bioengineering; Biomedicine; Cooperative Behavior; Drug Discovery; Drug Industry - methods; Drug therapy; Humans; Laboratories; Medical Law; Neglected Diseases - drug therapy; Neglected Diseases - therapy; Perspective; Pharmacology; Pharmacology/Toxicology; Pharmacy; Therapeutics - methods
• ISSN: 0724-8741; 1573-904X
• DOI: 10.1007/s11095-016-2039-5

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”.