PROGRAM:
The Salon is designed to foster discussion of the broad potential for stem cells in the discovery of new compounds and therapeutics for the treatment of human disease, and how advances made with stem cell-based drug screens in the laboratory can be translated for use in the clinic.  Our guests from academia and industry will share their personal experiences as well as their perspectives on the latest technology and translational aspects of drug discovery. The program will conclude with informal roundtables discussions with each of our guests. It is our ambition to deliver both the scientific perspective and the vision behind the use of stem cells in discovery of next-generation drugs and therapeutics.  

FACULTY HOSTS:
Lee Rubin, PhD (Harvard)
Leonard Zon, MD (CHB)

DISCUSSANTS:
iPS Cell-based Disease Models:  A New Evolution in Drug Discovery
Michael C. Venuti, PhD
CEO and President
iPierian, Inc.
iPSC (induced pluripotent stem cells) reprogrammed from fibroblasts are capable of being differentiated into many cell types involved in the basic pathologies of complex diseases. Patient-derived cells retain genetic and epigenetic information during the reprogramming and differentiation processes that allows disease-specific phenotypes to manifest in the differentiated cultures.  At iPierian, we have industrialized this process from iPSC generation through differentiation, disease model establishment, and into drug discovery.  Our current areas of focus are the neurodegenerative diseases SMA, ALS, AD & PD, and Type 2 Diabetes.

The Integration of Stem Cell Derived Cells into Safety and Toxicity Testing
Sandra Engle, PhD
Sr. Principal Scientist, Genetically Modified Models Center
Pfizer, Inc.
Safety and toxicology issues of drug candidates contribute to approximately 30% of drug attrition rates. In vitro models which better predict human physiology are absolutely critical. The properties of human adult stem cell and pluripotent stem cells make them an attractive cell source for developing these in vitro models. This talk will examine ways in which stem cell derived cells may be used to address safety and toxicity in drug development.

Human iPS-Derived Cardiomyocytes for Early Safety Screening
Joshua E. Babiarz, PhD
Principal Scientist, Non-Clinical Safety Unit
Hoffman-La Roche
Cardiac liabilities are a significant cause of pharmaceutical attrition, however preclincial methods for predicting drug-induced cardiac toxicities are insufficient, relying on transformed cell lines or model species that do not always predict the drug responses seen in humans.  Our work has focused on using human pluripotent stem cell-derived cardiomyocytes as a model system for predicting drug-induced cardiac toxicities. Using high resolution impedance measurements, we have developed high throughput assays for predicting drug-induced cardiotoxicity and cardiac arrhythmias.  These studies highlight the vast potential for utilizing human PSC-derived tissues as a novel platform for early safety screening.

Improved Assays for Drug Discovery
Daniel Curtis, PhD
Developmental and Molecular Pathways
Novartis institutes for BioMedical Research
The ability to reprogram differentiated adult patient cells to the pluripotent iPSC state opens up innumerable possibilities for medicine and for drug discovery.  Foremost is the ability to design in vitro assays using specific cell types of interest derived from defined human genetic backgrounds for screening and target validation.  In some contexts, purified single cell types are desired, while in others complex assays with mixed tissues are needed to create the desired biological context.  When relevant assays can be built, in vivo screening in zebrafish provides a context to identify regenerative factors in the intact organism.

Beer, wine and light refreshments will be provided for a post-discussion roundtable session!

ORGANIZERS:
Elmar Nurmemmedov, PhD (CHB)
Pamela Wenzel, PhD (CHB)
Wei Zhang, PhD (HSCI)