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Surmounting the Insurmountable: Obstacles in Drug Discovery and Development - Real World Case Studies

April 24, 2018
By Tamara Escajadillo

This symposium was sponsored by the BIG IDEAS Initiative, which was introduced in 2014 as a unique opportunity for members to propose a project that would directly benefit ASPET’s membership. This specific symposium was approved in 2016 and addresses real-world case studies in drug discovery and development, by providing a forum for pharmacological experts to recount their own experiences dealing with insurmountable problems that arouse during their research. Through this platform, speakers were able to first explain what the critical problem was, then describe how the problem was solved successfully (if possible!), what the impact of this was and what they learned, in an effort to help subsequent investigators know how to address these same problems should they arise in their own research.

The list of speakers included four researchers from different pharmaceutical companies, and the session began with Margaret Bradbury PhD from Prana Biotech, who spoke about the challenges and solutions in the approval of Austedo, the first deuterium substituted small molecule therapy for potential long term chronic use, which is of significant clinical importance for the treatment of Huntington’s disease. The next speaker was Frank Lee PhD, from Angex Pharmaceuticals Inc. who spoke of the drug Dasatinib for the treatment of chronic myelogenous leukemia, where their challenge was to find a way to override imatinib resistance, which ultimately led to the development of this novel ABL kinase inhibitor. The other two talks included Sandeep Dutta PhD from Amgen, who spoke of the problems faced in modeling and simulation studies that resulted in the development of Evolucumab, and Francis Wolenski PhD, from Takeda Pharmaceuticals International Co, who spoke of how Fasiglifam (TAK-875) alters bile acid homeostasis in rats and dogs and its potential involvement in drug induced liver injury.

The focus of this symposium was especially novel in that not only did it help elucidate the process involved in successfully discovering and developing new drugs, but it allowed speakers to shine a light on what were possibly untold or lost stories that contained hidden “treasures” or discoveries that might not have fit into any other platform, ultimately enriching our knowledge of scientific discovery by highlighting new technologies and new strategies involved in drug discovery.

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