Translational Cancer Medicine
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Wednesday, September 19
» Opening Plenary Session
2:00-3:00 Main Conference Registration
3:00-3:10 Welcoming Remarks from Conference Director
Julia Boguslavsky, Executive Director, Conferences, Cambridge Healthtech Institute
3:10-3:15 Chairperson's Opening Remarks
3:15-3:45 Rules, Tools and Data Pools: The Critical Path's Recipe for Speeding Drug Development
Carolyn Compton, M.D., Ph.D., President & CEO, Critical Path Institute
Both regulatory agencies and the medical products industry recognize the need for new biomarkers and methods to speed the development and delivery of effective, safe medicines to patients. However, developing consensus on the utility of new biomarkers and establishing a process for putting them into routine practice in medical product development and regulatory decision making is a complex undertaking. In 2009 and 2010, respectively, the European Medicines Agency and the FDA released guidance that describes a voluntary pathway for "qualification" of novel drug development tools or methods. Determining the relative advantages/disadvantages and evidentiary standards required for regulatory "qualification," applying a novel biomarker on a specific drug development program, or submitting an application for a diagnostic device are challenging tasks. The Critical Path Institute (C-Path) acts as a trusted third party to lead six pre-competitive global consortia that, with the cooperation of the FDA, develop and qualify molecular and imaging biomarkers as well as patient-reported outcomes instruments for specific contexts of use in medical product development. Several C-Path consortia use Clinical Data Interchange Standards Consortium disease area data standards in constructing clinical trial databases from which disease progression models and virtual clinical trial simulations are developed. Specific examples from these consortia will be utilized to illustrate the power of pre-competitive collaboration in the generation and qualification of drug development tools.
3:45-4:15 Technology Transformation at FDA: Driving Efficiency and Unleashing Innovation
Eric D. Perakslis, Ph.D., Chief Information Officer and Chief Scientist, Informatics, U.S. Food and Drug Administration
The U.S. Food and Drug Administration has recently taken significant steps to modernize and improve its information technology and informatics capabilities. The resulting infrastructure, architecture, innovation pathways and data sharing initiatives are intended to ease regulatory burden on innovators while maintaining the highest quality standards. In this talk, Eric Perakslis, Ph.D., the Chief Information Officer and Chief Scientist for Informatics at FDA, will discuss these initiatives in detail and will provide success examples, recent progress and future directions.
4:15-4:45 Turning an Active Compound into a Personalized Medicine: Do Biomarkers Help or Hinder?
Geert Kolvenbag, M.D., Ph.D., Global Product Vice President, AstraZeneca
The co-development of drug and biomarker has several inherent risk and challenges. In addition the expectations and demands of the oncology community have increased over the last years. These challenges will be illustrated by a case study of a very active compound destined for a fast development program as a personalized medicine, but running into scientific, diagnostic and development challenges. This experience creates questions for development of drugs and diagnostics today and in the future.
4:45-5:15 Strategies to De-Risk Drug Development Utilizing Biomarkers in Early Clinical Trials
Scott Kennedy, Ph.D., Global Head, Biomarker Development, Novartis Institutes for BioMedical Research, Inc.
Other than recent examples in oncology, the emerging field of personalized medicine has yet to live up to its promise of enabling more rapid, efficient drug development and providing customized therapies to patients. This presentation will exemplify and discuss how leveraging biomarkers which inform a biological understanding of targeted and disease pathways can increase the success of early clinical development. These biologically relevant markers can also enable the identification and treatment of parallel disease populations and serve as stratification or response markers for later stage development. This presentation will also discuss several of the challenges that this paradigm presents.
5:15-5:45 Implementing a Personalized Medicine Strategy: Is There Light at the End of the Tunnel?
Jeremy Barton, M.D., Vice President, Head of Oncology Clinical Research, Pfizer
Personalized medicine, the practice of tailoring treatment to individual characteristics of the patient, has emerged as a major force in oncology in the last decade with the expectation of significant improvement in safety and efficacy of therapeutics. The ultimate success of this paradigm is dependent on a variety of factors. The choice of drug target, selection of patients by companion diagnostic, regulatory authority acceptance of novel trial designs which best serve this strategy, improved health care information technology, insurance coverage and reimbursement are just a few of the important variables in the equation. This presentation will provide an overview of the current situation and discuss factors which will impact future progress in the field.
5:45-6:45 Welcome Reception in the Exhibit Hall with Poster Viewing
Thursday, September 20
7:30-8:15 am Morning Coffee or Sponsored Breakfast Presentation (Opportunity Available)
Contact Ilana Quigley at email@example.com or 781-972-5457
8:30-8:55 Bringing Biomarkers to Clinical Fruition: NCI’s Early Detection Research Network
Sudhir Srivastava, Ph.D., Chief, Cancer Biomarkers Research Group, National Cancer Institute
8:55-9:20 Linking Genome to Proteome: A New Cancer Program
Christopher Kinsinger, Ph.D., Program Manager, Office of Cancer Clinical Proteomics Research, National Cancer Institute
Genomic initiatives such as The Cancer Genome Atlas (TCGA) have characterized and sequenced the genomic alterations from several types of cancer. These efforts are providing a catalogue of alterations in the cancer genome and setting the stage for the development of more molecular interventions that attack cancer cells based on their specific genetic makeup. The Clinical Proteomic Tumor Analysis Consortium (CPTAC) will leverage its state-of-the-art, proteomic technologies to comprehensively translate cancer genomes to cancer proteomes. A unique feature of this program is the utilization of genomically characterized biospecimens (tumors, such as those from TCGA). These specimens provide a wealth of genomic data that can be used to refine protein targets and develop a clear biological hypothesis in furthering our understanding of cancer biology.
9:20-9:45 Pathway Activation Mapping as Both a Lead Discovery Engine and Companion Diagnostic Assay System for Personalized Cancer Treatment
Emanuel Petricoin III, Ph.D., Co-Director, The Center for Applied Proteomics and Molecular Medicine, George Mason University
Post-translational modifications such as phosphorylation drive and underpin nearly all deranged disease-related cell signaling processes and are not directly predictable using genomic approaches. We have invented a new type of technology, called reverse phase protein microarrays, to generate a functional activation map of known cell signaling networks for an individual patient obtained directly from a biopsy specimen. This patient-specific “circuit diagram” provides key information for individualized therapy. The identification of activated protein drug target networks can be used as patient selection and stratification—the realization of pathway biomarkers as perhaps the ultimate companion diagnostic assay.
9:45-10:45 Coffee Break in the Exhibit Hall with Poster Viewing
10:45 am-12:00 pm Please enjoy presentations in another track.
12:00-1:30 Enjoy Lunch on Your Own
1:30-1:35 Chairperson’s Opening Remarks
1:35-2:00 Early Adopters in Pharmaceutical Applications of Imaging: Pre-Clinical and Clinical Case Studies
Paul J. McCracken, Ph.D., Director of Imaging, Biomarkers and Personalized Medicine CFU, Eisai, Inc.
Novel therapeutic drugs may require imaging biomarkers that are at the leading edge of imaging technology or tracer discovery. Imaging biomarkers may be used to inform decision making at many key milestones in the discovery and development pipeline, but the value depends on many factors. Early use of imaging in the pre-clinical and translational space requires a significant understanding of the information provided by animal models and the applicability or challenges faced with clinical translation. Brief case studies will be reviewed including pharmacologic MRI (phMRI), novel PET tracer chemistry, oncology markers including biodistribution, and Alzheimer’s disease methods.
2:00-2:25 Homemade or Store Bought? Bringing Molecular Imaging to Cancer Clinical Trials
Peter Choyke, M.D., F.A.C.R., Chief, Molecular Imaging Program, National Cancer Institute, National Institutes of Health
Conceptually, Molecular Imaging (MI) is useful in patient selection and therapy monitoring. The realities of MI in a clinical trial are daunting. There is a limited menu of available MI PET/SPECT probes. Nonetheless, because of their availability, these agents are more relevant post-approval. Highly specific imaging probes require a “homemade” approach. Thus, there is a long lead time for the agent to become available. In this talk, examples of both “store-bought and homemade” MI probes are presented. The decision to use existing probes or develop them rests on the kind of information that is sought and speed with which it is needed.
2:25-2:50 Overcoming Regulatory Hurdles in Investigational Molecular Imaging
Paula M. Jacobs, Ph.D., Associate Director, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health
Molecular imaging agents have great promise for investigating biology and for assisting in the development of new therapeutic drugs. However, it can be difficult to obtain the regulatory approvals to investigate them in the clinical trial setting, particularly when pairing an investigational molecular imaging probe with an investigational therapeutic drug. This presentation will discuss the NCI’s experience with non-proprietary investigational molecular probes in both imaging only and imaging-therapy trial settings as well as a variety of regulatory strategies.
2:50-3:15 Presentation to be Announced
3:15-4:15 Refreshment Break in the Exhibit Hall with Poster Viewing
4:15-4:20 Chairperson’s Opening Remarks
4:20-4:45 Genetic Signatures and Their Use for Personalized Therapy
Paola Sebastiani, Ph.D., Professor, Biostatistics, Boston University
Genome-wide association studies have shown that the genetic basis of many diseases is complex, and this complexity has raised questions about the utility of genetic data for personalized therapy. I will describe an approach that can help dissect the genetic basis of complex traits. The method builds genetic risk profiles with large numbers of genetic variants, and uses cluster analysis to group them into genetic signatures. By examining the association between genetic signatures and sub-phenotypes of a trait, the method can identify genetic signatures linked to specific response to treatment. I will show examples from genetic traits with different heritability.
4:45-5:10 Next-Generation Sequencing in Drug Discovery
Joseph D. Szustakowski, Ph.D., Senior Group Head, Translational Medicine, Biomarker Development, and Bioinformatics, Novartis Institutes for Biomedical Research
Next-generation sequencing technologies and applications have seen rapid uptake in all facets of drug discovery. The inclusion of NGS platforms in our experimental armamentarium is enabling a host of experiments that were previously not tractable. Several NGS case studies will be reviewed with an emphasis on understanding and predicting how NGS will impact our clinical biomarker efforts.
5:10-5:35 Personalized Medicine: An Update on 1,985 Patients with Advanced Cancer at MD Anderson Cancer Center
Apostolia-Maria Tsimberidou, M.D., Ph.D., Associate Professor, Investigational Cancer Therapeutics, University of Texas, MD Anderson Cancer Center
We hypothesized that in Phase I clinical trials, use of targeted agents matched with tumor molecular aberrations would improve treatment outcomes. A targeted therapy was considered to be “matched” to a patient if at least one drug in the regimen was known to inhibit the functional activity of at least one of the patient’s mutations. In a 5-year period, 50% of patients who underwent tumor molecular profiling had a molecular aberration. Rates of response, time to treatment failure, and survival were higher in patients treated with matched Phase I therapy compared to those of patients treated with non-matched Phase I therapy. In patients treated with matched Phase I therapy, time to treatment failure was longer than on prior systemic therapy. These results continue to support use of a personalized molecular approach for patients with cancer. Complete molecular profiling to understand resistance mechanisms and new targeted agents are needed.
5:35-6:00 GliomaPredict – Translation from Transcriptomic Subtypes to Patient Bed
Aiguo Li, Ph.D., Senior Bioinformatician, NOB, National Cancer Institute, National Institutes of Health
We developed GliomaPredict, a tool that allows the fast and reliable assignment of glioma patients into one of six previously stratified subtypes based on sets of extensively validated classifiers. Our tool utilizes a principle component analysis (PCA)-based approach to generate a visual representation of the analyses, quantifies the confidence of the underlying subtype assessment and presents results as a printable PDF file. The GliomaPredict tool is implemented as a plugin application for the GenePattern framework. GliomaPredict provides a user-friendly, clinically applicable novel platform for instantly assigning transcriptomic subtype in patients with gliomas thereby aiding in clinical trial design and therapeutic decision making.
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