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Short Courses | Day 1 | Day 2 | Day 3 | Download Brochure Wednesday, February 3 7:00 AM Registration and Morning Coffee 8:10 When Drug Research is Personal
Mr. Crowley’s emotion-packed presentation will focus on his personal struggle to find a cure for Pompe disease, a rare and fatal illness that is caused by a defective or missing enzyme. Pompe disease affects fewer than 10,000 people world-wide, including Mr. Crowley’s two small children. Mr. Crowley, a Harvard educated businessman, created and built a pharmaceutical company devoted expressly to finding a cure for the disease. He will detail his journey through the labyrinth of scientific and business fronts, which lead up to a first-round clinical trial. 8:55 Technology, Aging, and the Brain
New neuroimaging and other technologies are teaching us about how the brain ages and what we can do about it. Although memory declines as we age, medical and nonpharmacological strategies may protect brain health and improve memory performance. At the same time, innovation in digital technology is not only changing the way we live and communicate, it appears to be altering how our brains function. As a consequence of this high-tech stimulation, we are witnessing the beginning of a new form of the generation gap – a brain gap dividing younger digital natives, immersed in the technology early in life, from older digital immigrants, who adapt to the new technology more reluctantly. This lecture will describe this current pivotal point in brain evolution and how we can harness the new technology and lifestyle choices to improve memory and brain function so we can live better and longer. 9:40 Grand Opening Refreshment Break in the Exhibit Hall
TRENDS IN TRANSLATIONAL MEDICINE 11:00 Chairperson’s Remarks Christina M. Coughlin, M.D., Ph.D., Medical Director, Oncology Clinical Research and Development, Wyeth Research 11:10 Translational Approach to Studying Stroke
To improve success of clinical trials and speed drug development, departments of Translational Medicine in pharma have formed to figure out the which molecular, biochemical and physiological biomarkers can best substitute for the absence of cinical outcome studies. My presentation will illustrate how we've applied a translational approach to develop better therapies for stroke. Specifically we've focused on reducing attrition rate of compounds/biologicals by optimizing 1. Target Validation; 2. Compound -Target interaction; 3. innovative Pharmacokinetic-Pharmacodynamic and proof of Mechanism of Action (MoA); 4. disease biomarkers; 5. Patient selections for clinical trials based on evidence for likelihood to respond to treatment. 11:40 Contribution of Translational Approaches to Recent Advances in Immuno-therapeutics, Immuno-rejection and Beyond
The complexity underlying a pathological process does not necessarily require complex solutions. The biology determining allograft or cancer rejection, autoimmunity or tissue damage during pathogen infections is complex; however, common patterns are emerging that lead to a common final outcome: tissue destruction with resolution of the pathogenic process (cancer, infection) or tissue damage and organ failure (allograft rejection, autoimmunity). Human observations based on transcriptional profiling converge into an “immunological constant of rejection” that signals such occurrences. This constant includes the coordinate activation of interferon stimulated genes (ISGs) and immune effector functions (IEFs). Understanding this final effector pathway may suggest novel strategies for the induction or inhibition of tissue-specific destruction with therapeutic intent in cancer and other immune pathologies. This presentation will discuss how vaccines may play a role in tissue-specific destruction and use this as a model to demonstrate how to understand the dynamics of therapeutics by studying target tissues in real time. 12:10 PM Panel: Practical Translational Medicine Moderator: Vivek Kadambi, Ph.D., Senior Director, Millenium Pharmaceuticals
Panelists: Giora Z. Feuerstein, M.D., Assistant Vice President and Head, Discovery Translational Medicine, Wyeth Research Labs Lise Kjems, M.D., Ph.D., Executive Director, Global Program DiagnosticDirector, Molecular Diagnostics Novartis Francesco Marincola, M.D., Chief, Infectious Disease and Immunogenetics Section, NIH; Editor-in-Chief, Journal of Translational Medicine William B. Mattes, PhD, DABT, PharmPoint Consulting, Former Executive Director, Predictive Safety Testing Consortium Sponsored by 
12:40 Luncheon Presentation I ZDSD Rat: A Model for Diabetes, Metabolic Syndrome, and Obesity without Leptin or Leptin Receptor Mutations Richard G. Peterson, Ph.D.; Executive VP Research & Development, PreClinOmics, Inc.
1:10 Luncheon Presentation II (Sponsorship Opportunity Available) 1:45 Dessert in the Exhibit Hall
WORKING BACKWARDS IN CANCER: 2:15 Chairperson’s Remarks Michael Liebman, Ph.D., Managing Director, Strategic Medicine, Inc. 2:20 Using Drug-Induced Feedback Loops to Identify Indications and Combination Partners
James W. Watters, Ph.D., Associate Director, Molecular Profiling Oncology, Merck Treatment with molecular targeted agents can result in compensatory feedback regulation as cells respond to inhibition of signaling pathways. We will present clinical evidence that treatment with a small molecule inhibitor of gamma secretase results in pathway modulation and compensatory feedback, and describe pre-clinical experiments designed to leverage this concept for drug response prediction. 3:00 Moving Research Closer to the Bedside, in Vitro and in Vivo Analyses with Primary Tumors Fred Poordad, M.D., Chief of Hepatology, Liver Disease and Transplant Center, Cedars-Sinai Medical Center; Xin Wei Wang, Ph.D., Senior Investigator, Head, Liver Carcinogenesis Section, Laboratory of Human Carcinogenesis, National Cancer Institute, NIH; Michael R. Briggs, Ph.D., Senior Director, Biology, Vertex Pharmaceuticals, Inc. The incidence of Primary Liver Cancer is increasing in the west and constitutes a tremendous burden on world health as the third leading cause of cancer deaths worldwide. The 5 year survival rate is a dismal 11 %, due in large part to late diagnosis and limited treatment options. The etiology of this devastating disease as well as current and proposed new therapies will be discussed. Steps to better diagnose and stratify patients for targeted therapy will be considered as a new and exciting phase of cancer research. Finally, a move toward more relevant research will be presented as an hypothesis that will be tested in the coming years as more new and current therapies are compared and contrasted to current best practice.
4:35 Reception in the Exhibit Hall (Sponsorship Available)l 5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall Moderator: Jingsong Wang, M.D., Director of Immunology, Discovery Medicine & Clinical Pharmacology, Bristol-Myers Squibb, Co.
2. Biomarkers in Translational Medicine Co-Moderators:
3. New Funding Opportunities for Biotechs Moderator:
4. New Animal Models in Translational Medicine
Co-Moderators: 6:20 Close of Day
Short Courses | Day 1 | Day 2 | Day 3 | Download Brochure |
John F. Crowley, Founder, Novazyme Pharmaceuticals, Inc.
Gary W. Small, M.D., Parlow-Solomon Professor on Aging, Professor of Psychiatry
& Biobehavioral Sciences, Director, UCLA Center on Aging, Director, Memory &
Aging Research Center, Director, Geriatric Psychiatry Division, Semel Institute
for Neuroscience & Human Behavior, David Geffen School of Medicine at UCLA
Giora Z. Feuerstein, M.D., Assistant VP, Discovery
Translational Medicine, Wyeth Research Labs 
Francesco Marincola, M.D., Chief, Infectious Disease and
Immunogenetics Section, NIH; Editor in Chief, Journal of Translational Medicine
Donald Bergstrom, Ph.D., Director, Experimental Medicine
Oncology, Merck 
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