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Patient-Specific Stem Cells and Cardiovascular Drug Discovery

Nicholas M. Mordwinkin, PharmD, PhD1,2,3; Andrew S. Lee, PhD1,2,3; Joseph C. Wu, MD, PhD1,2,3
[+] Author Affiliations
1Stanford Cardiovascular Institute
2Department of Medicine, Division of Cardiology
3Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, California
JAMA. 2013;310(19):2039-2040. doi:10.1001/jama.2013.282409.
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The pharmaceutical industry faces numerous challenges in the development of novel compounds. Phase 2 clinical trial success rates are at a 5-year low of 22%, and the average number of preclinical drug development programs needed to produce a single new drug has increased from 12 to 30 between 2007 and 2012 alone.1 One estimate suggests that the average new drug requires more than $1.8 billion and 12 years from the time of discovery to commercial launch.2 If drug development is to remain commercially viable in the future, the current model for drug discovery and development must undergo a fundamental shift. To reduce these high attrition rates and increased costs, drug developers need to be able to predict and identify potential efficacy and safety issues as early as possible during the drug discovery process. Doing so would enable more attention to be focused on programs with optimal chances of progressing through end-stage clinical trials rather than on costly failures.

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Patient-Specific iPSC-CMs Represent a Potential Platform for Phenotypic Drug Discovery Assays Because of Their Capacity to Recapitulate Cardiac Phenotypes

Coupled with current advances in high-throughput screening technologies, patient-specific iPSC-CMs could help accelerate the ability to investigate the molecular mechanisms of cardiovascular disorders resistant to current pharmaceutical treatments and could potentially identify novel therapeutic targets for these diseases.

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