Workshops

There are four pre conference workshops available on Tuesday 20th September. Attendees can book attendance on up to three workshops. 

Workshop B2 (8.00am - 11.00am)

Workshop B1 (11.30am - 2.30pm)

Workshop C (3.00pm - 6.00pm)

Workshop B2  (8.00am - 11.00am)

 Strategic Considerations in the use of In Vitro Models

 In designing a pre-clinical development program, companies are faced with universe of choices when considering implementing in vitrotoxicity screens, be they well-established, novel, or available through contract laboratories. This workshop will focus on the first and most important step in implementing such screens, that of a critical analysis of the true needs of the company’s development program, its decision-making paradigms, and the critical demands of the business strategy. One size does not fit all, and the most successful in vitroscreening program is customized to a particular organization and development program.

Considerations explored will include:

• Where does the information from such a screening progam impact the business strategy (eg, progression of a candidate successfully through Phase I or all the way through Phase III, providing value to a licensing prospect)?
• Where in the development program might the in vitro screen be positioned (e.g. HTS, lead optimization)?
• How many compounds are realistically being screened?
• What toxicity or efficacy issues are common (and/or tolerated) in the therapeutic area?
• What key success factors must an in vitro screen meet to make it worth implementing?
• How might animal info be combined with in vitro assay to help validate the test?
• Models will be useful for predicting some in vivo endpoints but not others - How can one connect in vitro endpoints with these endpoints that you are ultimately interested in?

 Workshop leader: William B Mattes, Senior Director, COPD Biomarkers Qualification Consortium at COPD Foundation and Principal,PharmPoint Consulting

 William B. Mattes received his BA from the University of Pennsylvania and PhD in biological chemistry from the University of Michigan, Ann Arbor.  He did his postdoctoral training at the Johns Hopkins University, and was a staff fellow at the National Cancer Institute, the National Institutes of Health (NCI/NIH).

Dr. Mattes is the founder and principal of PharmPoint Consulting, providing expertise in innovative drug development, biomarker implementation, mechanistic, genetic, molecular and systems toxicology, and collaboration leadership. Prior to establishing PharmPoint Consulting, Dr. Mattes was Director of Toxicology at the Critical Path Institute where he developed and directed the Predictive Safety Testing Consortium (PSTC).

Dr. Mattes was also senior scientific director of Toxicogenomics at Gene Logic, associate director of toxicogenomics and group leader of genetic toxicology at Pharmacia Corp, group leader of experimental toxicology and metabolism at Ciba Pharmaceuticals and group leader of molecular and cellular toxicology at Ciba-Geigy. He is a full member of the Society of Toxicology (SOT).

Workshop B1  (11.30am - 2.30pm)

Setting Up In Vitro Models Of Disease: iPS Cells And Beyond

In vitro disease models can offer unprecedented mechanistic insights into disease development and progression as well as invaluable platforms for drug screening. Human embryonic stem (ES) cells and induced pluripotent stem (iPS) cells, which can be generated by epigenetic reprogramming of somatic cells, provide an invaluable tool for such an approach as they retain the ability to be differentiated into all somatic cell types. iPS cells in particular offer the ability to create somatic cell types ‘tailored’ to a patients genetic makeup and even perhaps epigenetic predisposition to disease. More recently, direct conversion of somatic cells such as patient fibroblasts into a cell type of interest such as a neuron, has emerged as an alternative approach.

During this workshop we will examine several examples of in vitro disease modeling, in a series of 20min talks. Type I diabetes, Amyotrophic Lateral Sclerosis, Parkinson’s disease, Gaucher disease and Spinal Muscular Atrophy as being investigated using pluripotent stem cells as well as somatic cells, utilizing reprogramming and transdifferentiation. Participants will be able to consider a variety of approaches and experiments all geared up towards in vitro disease modeling and drug screening.

Series of talks:

Induced Pluripotent Stem Cells To Model Type 1 Diabetes
Rene Maehr, PhD

Utilizing Embryonic And Patient-Specific Induced Pluripotent Stem Cells to Study ALS
Evangelos Kiskinis, PhD

Mitochondrial Dysfunction in Neurons Derived From Mutant PINK1 Induced Pluripotent Stem Cells

John Graziotto, PhD

Lysosomal Storage Disorders And Neurodegeneration: A Pathogenic Link Between Gaucher And Parkinson's Disease
Joe Mazzulli, PhD

Spinal Muscular Atrophy: From Screen To Target

Nina Makhortova, PhD

Defined-Factor Reprogramming For The Study And Treatment Of Disease
Justin Ichida, PhD

Workshop Leader

Evangelos Kiskinis, Postdoctoral Fellow, Kevin Eggan Lab, Howard Hughes Medical Institute, Harvard Stem Cell Institute, Dept of Stem Cell and Regenerative Biology, Harvard University

Evangelos Kiskinis MSc, PhD is a postdoctoral fellow in Kevin Eggan’s lab at the department of Stem Cell and Regenerative Biology at Harvard University. His work focuses on utilizing stem-cell based approaches to study neurodegenerative diseases including ALS. Evangelos received his PhD in Molecular Endocrinology from Imperial College London and has been the recipient of an EMBO postdoctoral fellowship (2008) as well as a Drukenmilller research fellow of the New York Stem Cell Foundation (2011).

Workshop C  (3pm - 6pm)

New Models in Drug Development: Human iPS Cells and their Derivatives

Human tissue cells derived from induced pluripotent stem cells (iPSCs) overcome existing models’ limitations in functionality, purity and reproducibility and offer a virtually unlimited supply of well-characterized cells for drug efficacy screening and safety testing.  Importantly, iPSC technology can be used to create any cell type from any individual or patient population, which is enabling more biologically relevant disease modeling, improved drug screening strategies and forward movement toward personalized medicine. 

This workshop will:

• Provide a current perspective on iPS cell technology
• Demonstrate a broad range of applications for iPS cell-derived cell types (e.g. cardiomyocytes, hepatocytes, neurons)
  • Present case studies demonstrating the utility of iPS cell-derived cardiomyocytes for drug development

Register Now!  The first 30 registrants will receive a free vial of CDI’s iCell® Cardiomyocytes ($1500 value).  Registrants must be present at this PIVM workshop in order to receive this offer. Some restrictions apply.

Workshop leader: Chris Parker, Chief Commercial Officer, Cellular Dynamics International

Co-Leaders:

Josh Babiarz, Principal Scientist, Hoffman-La Roche
Utilizing Stem Cell-Derived Cardiomyocytes for Early Safety Screening

Leon Collis, Senior Scientist, Immunology & Autoimmunity, Pfizer
Fluorometric and Contractility-Based Screening Platforms for Cardiotoxicity Profiling

Joe Reynolds, Senior Scientist, Merrimack Pharmaceuticals
Assessing Surrogates of Clinical Cardiotoxicity Using Stem Cell-Derived Cardiomyocytes

Chris Parker is responsible for sales, marketing and business development at Cellular Dynamics International. Mr. Parker brings more than 20 years of experience in the life science industry to CDI. As a Vice President at Affymetrix, he managed all aspects of sales and marketing for the Global Pharmaceutical Business Unit. Mr. Parker led the sales efforts that grew Affymetrix to over $350 million per year in revenue within 8 years. Prior to his position in the Global Pharmaceutical Business Unit, Mr. Parker was responsible for market development of Affymetrix's genetics business and flagship products, establishing key contacts and key applications that drove the growth of the genome scanning market and chromosomal copy number applications. Mr. Parker's professional background also includes serving on the drug discovery services team at Amersham Pharmacia and over a decade of research in molecular and cellular biology in the Department of Human Oncology at the University of Wisconsin-Madison, Comprehensive Cancer Center. While there, he worked with notable investigators, including V. Craig Jordan, to develop important cancer drugs such as Tamoxifen.