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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
Harry Glorikian, Managing Partner, Scientia Advisors
11:05 Molecular Diagnostics as a Value Driver of Pharma/Pharma as a Value Driver for Molecular Diagnostics
Michael C. Little, Global Head, Diagnostics Development, Novartis Molecular Diagnostics
Molecular Diagnostics is a critical success factor for the future of pharmaceuticals and an essential aspect of the move toward personalized medicine. At the same time, to progress better healthcare and patient outcomes, it is imperative that the pharma industry’s understanding of both targeted drug discovery and drug commercialization is fully leveraged to enable innovative diagnostics to be put into clinical practice and influence physician decision-making. The keynote will focus on these elements and discuss 1-2 case studies of how we at Novartis are using our discovery and development approach to work toward bringing innovative companion and stand-alone diagnostic tests to market.
11:40 Building a Successful Diagnostics Business Model in the Era of Personalized Medicine
Richard Ding, CEO, bioTheranostics, a bioMerieux Company
Personalized medicine has been generally accepted as an inevitable trend in healthcare. However, much debate is still ongoing related to a sustainable business model for diagnostics companies in this new space. This presentation will identify various challenges, risks and potential returns for diagnostic companies, explore partnership models and propose some basic framework to seize the growth opportunity of personalized medicine.
12:15 PM Personalized Medicine: It Takes a Village
Mark Stevenson, President & COO, Life Technologies Corp.
New technologies, such as next generation sequencing, can be rapidly adopted in the research labs and help breakthroughs in our understanding of disease mechanisms for personalized medicine. But the journey from research technology to diagnostic systems is challenging and slow. As our understanding of disease increases the promise of personalized medicine is coming closer but what will it take to cross the bridge from research tool to routine diagnostics in personalized medicine. The Presentation will focus on the journey Life Technologies has embarked on and the partnerships and collaborations necessary to translate the tools for the research lab into solutions personalized medicine.
12:50 Luncheon Presentation
Information Trends in Biomarker Research
Colin Williams, Ph.D., Director, Product Strategy, Thomson Reuters Healthcare and Science
In recent years, the quantity of data published on biomarker research has exploded. The challenge faced by researchers is to find vital, relevant information on the best biomarker quickly and reliably. In this discussion we will introduce BIOMARKERcenter, a comprehensive, fully-indexed biomarker information resource, and through case studies show how it aids the discovery process.
1:45 Dessert in the Exhibit Hall
2:15 Chairperson's Remarks
Tracey Colpitts, Ph.D., Business Development, Companion Products, 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 Isotopic Mass Tags for the Facilitated Development of Multiplex SRM Mass Spectrometric Assays for Protein and Peptide Biomarkers
Helen Byers, Ph.D., Principal Research Scientist, Proteome Sciences plc, UK
In order to verify those biomarkers that have most utility and value, the biomarker development pipeline requires rapid progression from candidate discovery to biomarker qualification using fit-for-purpose assays. Mass spectrometric methods, such as Selected Reaction Monitoring (SRM), are increasingly used for the quantitation of peptides and proteins. To overcome the limitations of the conventional Selected Reaction Monitoring (SRM), i.e. expense and delay connected with synthesis of isotope-doped standard peptides, difficulty to synthesize more complex standards (e.g. with post-translational modifications), Proteome Sciences has developed isotopic versions of its proprietary tandem mass tag (TMT) reagents to differently label sample and standard. The TMT-SRM method therefore allows the use of either synthetic or natural reference standards and thus provides a quick and economical way to establish assays for any peptide or protein in any given sample material. Furthermore, because isotopic TMT’s are identical in structure to isobaric TMT’s used for biomarker discovery there is minimal set up time or costs when moving from candidate discovery to assay qualification and validation. Proteome Sciences is the only company to offer this unique method as part of their PS Biomarker Services to the pharmaceutical and biotech sector and the presentation will include examples to illustrate the provision of multiplex TMT-SRM mass spectrometry assays for candidate biomarkers of Alzheimer’s disease and demonstrate the exceptional performance criteria than can be achieved.:
• Criteria for the development of biomarkers
• Description of fit-for-purpose assay systems for protein and peptide biomarkers
• Advantages of multiplex TMT-SRM over conventional SRM.
• Specifications of multiplex TMT-SRM assays
• Application of multiplex TMT-SRM assayd for the qualification of candidate biomarkes for Alzheimer’s Disease
• Description of biomarker services using TMT-SRM
4:20 Reception in the Exhibit Hall (Sponsorship Available)
5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall
Personalized Medicine: Commercial Hurdles to Adoption in an Era of Evidence Based Medicine
Moderator: Katherine Tynan Ph.D., Business Development & Strategic Consulting for Diagnostics Companies, Tynan Consulting LLC
The evidence required for each of the stakeholders: analytical performance and clinical validity for the FDA, clinical utility for physicians, and medical necessity for payers.
The strategic importance of "intended use statements" in guiding product/test development
The opportunities and challenges with reimbursement
Development and Application of Assays in the 3D Format
Moderator: Ray Mattingly, Ph.D., Associate Professor, Pharmacology, Wayne State University
Challenges in standardization and feasibility of 3D cultures
Development of 3D format for high-throughput assays
Protocols for drug screening in 3D culture format
Advanced 3D co-culture approaches to model tissues
How Innovative Technologies Are Selected, Evaluated, and Translated for Application in Diagnostics for Personalized Medicine to Enhance or Replace Conventional Diagnostics
Moderator: Kewal K. Jain, M.D., Professor, CEO, Jain PharmaBiotech
The ideal molecular diagnostics laboratory for personalized medicine
Selection, evaluation, and translation of new diagnostic technologies for personalized medicine
Role of sequencing
Future prospects of diagnostics for personalized medicine: supplementing, enhancing or replacing conventional diagnostics
Biomarkers of Efficacy
Moderator: Tracey Colpitts, Ph.D., Business Development, Companion Products, Abbott Molecular
How does mechanism of action hypotheses translate to population science?
When and how do we gather prevalence data?
What priorities should we be making?
Next Generation Sequencing in the Clinical Diagnostics Laboratory
Moderator: Karl V. Voelkerding, M.D., Associate Professor of Pathology, University of Utah and Medical Director for Advanced Technology, ARUP Laboratories
What are the first diagnostic applications that next generation sequencing will be used for?
What improvements would facilitate translation of the technology into the clinical laboratory?
How will laboratories process and interpret the large amounts of data generated?
6:20 Close of Day
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