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Wednesday, February 3
7:00 AM Registration and Morning Coffee
Plenary Keynote Session
8:10 When Drug Research is Personal
John F. Crowley, Founder, Novazyme Pharmaceuticals, Inc.
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
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
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
11:00 Chairperson’s Remarks
Michael Liebman, Ph.D., Managing Director, Strategic Medicine, Inc.
11:10 Personalizing Medicine: It’s a System-Based Challenge
Franklyn G. Prendergast, M.D., Ph.D., Professor, Pharmacology, Biochemistry & Molecular Biology, Director, Center for Personalized Medicine, Mayo Clinic
11:40 Genomic Strategies for Personalized Cancer Treatment
Joseph R. Nevins, Ph.D., Barbara Levine Professor, Duke University
We have made use of expression profiling to develop signatures of oncogenic pathway deregulation that can then be used to profile the state of these pathways within populations of tumors. In addition, the pathway signatures also link the patterns of pathway activation with therapeutics since we have shown that predicting the activation of a pathway also predicts sensitivity to drugs that target the pathway. We have extended this concept to develop more refined signatures that can dissect the complexities of many of the known signaling pathways, providing a more precise capacity to probe the activity or deregulation of the pathway and linking to a broader array of therapeutics.
12:10 PM Panel: Impact of Personalized Medicine on Oncology Drugs and Treatment
Additional Panelist: Mike Boswood, President, CEO, Thomson Reuters
12:40 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own
1:45 Dessert in the Exhibit Hall
2:15 Chairperson's Remarks
Tracey Colpitts, Ph.D., Manager, Abbott Molecular
2:20 Relating Biomarkers to Efficacy: The Efficacy Curve
Tracey Colpitts, Ph.D., Manager, Abbott Molecular
A method of predicting response in a subgroup defined by a biomarker will be discussed and demonstrated using data from therapeutic trials involving EGFR inhibitors in lung, colon, and breast cancer. Biomarkers that aid in selecting subgroups of patients of response were analyzed and compared. Striking similarities between the different cancers, therapies, and subgroups reveals a relationship between biomarkers and efficacy, which is visualized in the efficacy curve.
2:50 Network and Pathway Analysis of a Novel 3D Breast Carcinoma Model by Both Digital Gene Expression (DGE) and Whole Genome Array Analyses
Ray Mattingly, Ph.D., Associate Professor, Pharmacology, Wayne State University
We have developed a tractable, in vitro model of ductal carcinoma in situ (DCIS) based on 3D overlay culture in reconstituted basement membrane (rBM). We have applied and cross-validated whole genome microarray (Affymetrix) and digital gene expression (DGE) analyses (Illumina/Solexa) to explore the networks and pathways that underlie DCIS. DGE analysis revealed a broad range of products that are transcribed outside of standard (NCBI 36.3) genes models. These transcripts suggest truncations and changes in anti-sense driven regulatory pathways in DCIS.
3:20 Population Based in vivo Biomarker Discovery Using Engineered Human Tumors
Min Wu, Ph.D., Principal Scientist, Translational Research, AVEO Pharmaceuticals, Inc.
Human tumor populations exhibit significant inter-tumor variation, where each tumor harbors a unique set of genetic alterations that impact prognosis and response to treatment. Unfortunately, this variation results in low response rates in the clinic and creates significant challenges for drugs to meet regulatory endpoints. Cancer cell line based xenografts have traditionally been the preclinical model of choice to assess the efficacy of clinical compounds, however, such models exhibit inherent artifacts due to long term in vitro culture, and are unable to adequately capture natural variation seen in human tumor populations. To address this challenge, we have created a population based tumor model system based on Human-in-Mouse tissue transgenic human tumors that feature naturally occurring tumor variation akin to that observed in human tumor populations. Each tumor of the population has been comprehensively characterized at the RNA and DNA level, and the population has been adapted to conduct quantitative efficacy studies of anti-cancer agents and combinations, enabling correlations between response and the genetic context of the tumors. This platform enables us to identify and validate biomarkers of therapeutic response in an in vivo human tumor system.
3:50 Sponsored Presentation (Sponsorship Opportunity Available)
4:50 Reception in the Exhibit Hall (Sponsorship Available)
5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall
What is the Forecast for Epigenetics and microRNA?
Moderator: Enal Razvi, Ph.D., System Biosciences SBI
Status of the microRNA and epigenetics markets
The research market for microRNA and epigenetics: growth and evolution
Diagnostics and therapeutics development based on microRNA and epigenetic signatures
Current challenges and opportunities in these spaces
Challenges to Whole Genome Sequencing
Moderator: Pauline Ng, Ph.D., Assistant Professor, Genomic Medicine, J Craig Venter Institute
- Challenges to whole-genome sequencing
- Identifying de novo and re-current mutations in cancer
- Addressing tumor heterogeneity
- How can we move from characterizing gene variation to utilizing the whole genome
- Sequencing tumors rather than tumor cell lines
- The Complex genomic structure of tumor cells: de novo assembly or strategy to detect structural variants
Are there Cancers of Unknown Primary Tumors?
Moderator: Dalia Cohen, Ph.D., Chief Scientific Officer, Rosetta Genomics, Inc.
- Debate over cancers of unknown primary tumors (CUP)
- Methods to detect CUPs
- Consequences of detection of primary
Gene Signatures in Cancer Diagnostics
Co-Moderators: Gary Geiss, Ph.D., Principal Scientist, NanoString Technologies and David Kern, MBA, Director, MyRaQa
- Developing a gene signature
- Validation of gene signatures
- Regulatory considerations for gene signature diagnostics
Systems Chemical Biology-A New Paradigm
Moderator: Ally Perlina, Senior Application Scientist, GeneGo Inc.
Utilizing tools for drug repositioning
Understanding side effects
Understanding the mechanisms of action for drugs
- Networkable compounds
6:20 Close of Day