Tuesday, April 29
5:00-6:00 pm Conference Pre-Registration
6:00-9:00 Dinner Course*
Exosomes and Microvesicles as Cancer Biomarkers
(*Separate registration required)
Wednesday, April 30
7:30-8:30 am Conference Registration and Morning Coffee
8:30-8:40 Welcome Remarks from Conference Director
Julia Boguslavsky, Executive Director, Conferences, Cambridge Healthtech Institute
8:40-8:45 Chairperson’s Opening Remarks
8:45-9:10 Transforming a Research Assay to a Companion Diagnostic
Ron Mazumder, Ph.D., MBA, Global Head, R&D and Operations, Janssen Diagnostics, Janssen Pharmaceutical Companies of Johnson & Johnson
Developing a predictive biomarker on a diagnostic platform often involves converting an assay which uses research-grade reagents and a research platform onto a diagnostic instrument which can be commercialized globally with GMP-manufactured and validated reagents. Furthermore, design verification under Quality Systems Regulations, clinical validation and clinical reproducibility must be completed during the drug development process. I will highlight considerations from case studies to explore each of these topics.
9:10-9:35 Tracking Value Creation across Diverse Biomarker Studies and Platforms in Pharmaceutical Development
Michael Burczynski, Ph.D., Executive Director, Biomarker Technologies, Discovery Medicine and Clinical Pharmacology, Bristol-Myers Squibb
An ever-growing list of biomarkers (and diagnostic assays) is frequently analyzed across many stages of drug development. One of the more vexing challenges facing pharmaceutical companies today is determining how to ensure that biomarkers are both judiciously implemented, yet also maximally utilized, to answer questions in translational research. For various reasons (cost, limited resources, shortened cycle times) it is no longer possible or acceptable to simply run multiple biomarker assays in clinical studies for exploratory purposes or to inform future therapeutic strategies. Biomarker assays (irrespective of platform) need to answer extremely well defined questions in drug development, and should employ associated metrics that can be objectively evaluated after study conclusion to determine whether the biomarker studies generated sufficient value to the program or therapeutic area in question. The present talk will focus on these principles and describe a general approach to 1) defining the potential value of biomarker assays prior to clinical study execution; 2) tracking biomarker assays and programs in clinical trials at a large pharma company via a specifically designed database strategy; and 3) objectively assessing the impact of biomarker assays following clinical study conclusion.
9:35-10:00 Successful Implementation of Global Biomarker Strategies Requires Laser Focus on Preanalytical Processes
Marisa Dolled-Filhart, Ph.D., Associate Director, Pathology and Companion Diagnostics, Merck
Generation of quality biomarker data begins with the development of an accurate and precise method. However, this is simply not sufficient. Studies show that the activities preceding sample analysis, the preanalytical activities, are as important, if not more important than method performance. Many reports cite significant assay variability being attributed to what happens prior to the sample arriving at the bioanalytical lab. Paying close attention to how the sample is collected, processed and shipped will ultimately determine your success.
10:00-10:30 Networking Coffee Break
10:30-10:55 Talk Title to be Announced
Andrew Schade, M.D., Ph.D., Senior Director, Diagnostics and Experimental Pathology, Tailored Therapeutics, Eli Lilly and Company
10:55-11:20 Companion Diagnostics: How Personal Does It Have to Get?
Daniel Edelman, Ph.D., Core Manager, Clinical Molecular Profiling Core, National Cancer Institute, NIH
Companion diagnostics are becoming an important and critical tool for clinicians in the treatment of patients suffering from diseases for which individualized targeted therapy is available. The FDA has approved 19 laboratory tests (as of December 2013) to aid in this improved treatment of patients. Yet, their impact on public health could depend on how personal they have to get. Also, laboratory-developed tests may provide a greater benefit in some circumstances. In this talk I will compare and contrast these and other relevant issues.
11:20-11:50 Sponsored Presentation
Jeremy Bridge-Cook, Senior Vice President, Research and Development, Luminex Corporation
The development of biomarkers from discovery to clinical implementation as a Companion Diagnostics is a process which is inherently unpredictable; no two development pathways are alike. Given this unpredictability, it is desirable to develop biomarkers using a platform which provides flexibility, but which also reduces complexity as much as possible. The xMAP ® platform has been used extensively at all stages of biomarker development. The speaker will highlight some examples of discovery, validation and clinical implementation of biomarkers on xMAP ®
11:50am-12:15pm CyPlex: A Transformational Immunoassay Technology
Rajiv Pande, Ph.D., Vice President, Scientific Affairs, CyVek Inc.
CyPlex Systems is a novel quantitative immunoassay platform that integrates a disposable microfluidic cartridge with a fully automated desktop analyzer. Multiple samples can be loaded onto a single CyPlex cartridge, and multiple analytes per sample can be quantified simultaneously, within an hour. CyPlex cartridges combine a unique solid phase approach (glass nanoreactors)with microfluidics toprovide high quality multi-analyte results without the typical multiplexing compromises. CyPlex assays showexcellent robustness and are extremely easy to perform.
12:15-12:50 Luncheon Presentation: Validated Multiplexed Cytokine Assays: A New Standard for Immunoassays
Pankaj Oberoi, Ph.D., Vice President, Commercial Assays, Meso Scale Discovery
Inconsistencies between biomarker studies can be attributed to variability between assay kit lots. Even CE‐marked kits may lack reproducibility because the CE mark is self‐regulated. With the use of well‐characterized, purified reagents and highly optimized assays, MSD’s V‐PLEX™ product portfolio demonstrates consistent and robust immunoassays. MSD has developed a 30‐plex human biomarker assay that is analytically validated and shows excellent lot‐to‐lot reproducibility and superior sensitivity, precision, and accuracy.
1:25-1:30 Chairperson’s Opening Remarks
Corinne Solier, Ph.D., Pharmaceutical Sciences Head, Extramural Research, F. Hoffmann-La Roche
1:30-1:55 The Dawn of a New Era for Lung Cancer Early Detection: CT Screening in Combination with Molecular Diagnostics
Pierre Floriano, Ph.D., Scientific Manager, Translational Medicine, McCombs Institute for the Early Detection and Treatment of Cancer, MD Anderson Cancer Center
Findings from the National Lung Cancer Screening Trial have confirmed the benefits of CT screening for lung cancer detection. Moreover, numerous studies have reported biomarker sets that have utility for lung cancer early detection. It has become timely to launch a trial to determine the merits of combining CT-based screening with biomarkers for improved performance compared to CT alone and eventually determine the need for CT based on initial biomarker-based screening.
1:55-2:20 Antibody-Based Proteomics and Biomarker Research: Current Status and Limitations
Corinne Solier, Ph.D., Vice Director, Pharmaceutical Sciences Head of Extramural Research, F. Hoffmann-La Roche
Antibody-based proteomics plays a very important role in biomarker discovery and validation. Mass spectrometry is a method of choice for hypothesis-free, high-throughput evaluation of candidate markers; however, antibody-based technologies remain the main solution to achieve optimal sensitivity in complex samples. This presentation will review the benefits and limitations of antibody-based proteomics in biomarker research for discovery and validation in body fluids and tissue. The combination of antibodies and mass spectrometry utilizing the best of both worlds opens new avenues in biomarker research.
2:20-2:45 Clinical Biomarker Method Development and Fit-for-Purpose Validation in Support of Biotherapeutics Drug Development: Challenges, Learning and Opportunities
Jenny Zhang, Ph.D., Manager, Biomarker Assay Specialist, Clinical Assay Group, Global Innovative Pharmacology, Pfizer
For a clinical study to support biotherapeutics drug development, it is important to apply a bioanalytical method that is capable of generating precise and accurate biomarker PD data for critical decision making, PK/PD modeling, and dose selection. When multiple platforms (such as ELISA and LC/MS/MS assays) are available, a suitable bioanalytical method must be identified. The presentation will focus on the strategy of method selection, fit-for-purpose method validation, and data impact on the clinical trials. Three unique case studies will be discussed.
2:45-3:45 Refreshment Break in the Exhibit Hall with Poster Viewing
3:45-4:15 Proteomics-Assisted Discovery of a Host Response Plasma Biomarker Panel to Accurately Diagnose Persistent Coughers with Active TB Disease
Rushdy Ahmad, Ph.D., Research Scientist, The Broad Institute of Harvard and MIT
A simple to use, robust and accurate blood-based rapid diagnostic test is needed for tuberculosis control and prevention. Each year approximately 9 million people are infected with TB. The lack of a rapid TB diagnostic test contributes to nearly 1.5 million deaths every year. TB is one of the top killers of women worldwide, resulting in half a million deaths annually. At the Broad Institute of MIT and Harvard, the Proteomics group has utilized high quality plasma samples from TB and control patients in conjunction with Myriad-RBM's Human Inflammation MAP to develop a specific and sensitive host response biomarker panel to diagnose adult persistent coughers with active TB disease. We will present results from this 5-year long prospective study and chart next steps to translate these promising results into a field deployable rapid TB diagnostic test. Such a test has the potential to be a game changer, saving many hundreds of thousands of lives every year.
4:15-4:45 Assay and Kit Lot Bridging Considerations for Single and Multiplex Biomarker Analysis in Support of Clinical Studies
Afshin Safavi, Ph.D., Founder and CSO, BioAgilytix Labs
Biomarker analysis has become a common practice by many pharmaceutical companies to help PK/PD modeling. The reliability of outcomes is heavily influenced by the quality of the reagents. One of the challenges that bioanalytical labs face when running biomarker studies is the control of lot-to-lot variability of critical reagents and commercial immunoassay kits. Case studies will be presented to highlight the key bioanalytical considerations involved in running successful biomarker analyses in support of clinical studies.
4:45-5:00 Quantitative Low-Abundance Biomarker Measurement and Its Impact on Disease Management
Joe Barco, Ph.D., Senior Product Manager, Life Sciences, Singulex, Inc.
Several considerations must be taken into account to establish a clinical biomarker, including the ability to measure normal states. Singulex’s proprietary single molecule counting technology helps overcome challenges in biomarker translation from discovery to clinic. This presentation reviews applications of quantitative and sensitive detection technology in ‘real life’ applications.
5:00-6:00 Welcome Reception in the Exhibit Hall with Poster Viewing
6:00-9:00 Dinner Courses*
- Fit-for-Purpose Biomarker Assay Development and Validation
- Non-Coding RNAs as Biomarkers and Diagnostics
(*Separate registration required)
Thursday, May 1
7:30-8:15 am Breakfast Presentation (Sponsorship Opportunity Available) or Morning Coffee
8:25-8:30 Chairperson’s Opening Remarks
Seth Crosby, M.D., Director, Partnerships & Alliances, Washington University School of Medicine
8:30-8:55 Does NGS Make Sense for Prospective Trials?
Seth Crosby, M.D., Director, Partnerships & Alliances, Washington University School of Medicine
NGS data that informs patient stratification decisions must be generated in a clinically certified lab. The challenges and expense of validating and reporting a clinical NSG panel are quite distinct from that of translational research. What is involved in this process? With the falling prices and changing regulatory environment of NGS, has the time come to pre-profile prospective trial participants? If so, should NGS be done internally or should you outsource?
8:55-9:20 Developing a Next-Generation Sequencing Test for Lung Cancer Mutations
Derek Chiang, Ph.D., Research Investigator, Novartis Institutes for Biomedical Research
9:20-9:50 Next-Generation Cancer Diagnostics at Illumina
Frank S. Ong, M.D., Associate Director, Medical Affairs, Illumina, Inc.
Illumina provides a comprehensive line of products that address the scale and breadth of functional analysis required to achieve the goals of molecular medicine. From biomarker discovery to the development of MDx assays, our offering includes leading-edge solutions for NGS, genotyping, CNVs, GEx profiling, and DNA methylation. In this session we will provide an update on our clinical oncology strategy and discuss the work we are doing to position ourselves as a leading partner for the development of next-gen cancer diagnostics.
9:50-10:45 Coffee Break in the Exhibit Hall with Poster Viewing
10:45-11:10 Practical Applications of NGS-Based Assays in a Clinical Molecular Oncology Laboratory
Helen Fernandes, Ph.D., Associate Professor, Pathology and Laboratory Medicine, Weill Cornell Medical College
The utility of targeted next-generation sequencing-based assays for identification of genomic variants continues to revolutionize the field of molecular diagnostics. Several organizations are working on new guidelines to standardize indications for testing and reporting of NGS-based assays. This presentation will focus on validation of analytic performance characteristics of assays for the identification of mutations and genomic variants in solid tumors. The issues involved for optimal performance, implementation in a routine clinical laboratory and reporting of results will be discussed.
11:10-11:35 A High-Throughput Microfluidics-Based Mutation and Copy Number Variation (CNV) Detection Panel for Analysis of Clinical Samples
Rajesh Patel, Ph.D., Scientific Manager, Oncology Biomarker Development, Genentech
Molecular profiling of tumor DNA has taken center stage for determining treatment options for patients and for enrolling patients in clinical trials for testing targeted therapies. Detecting somatic mutations in targeted genes — for example, BRAF in melanoma, EGFR for lung cancer and KRAS for colorectal cancer — has become an integral part of personalized medicine. We have developed a high-throughput microfluidics-based mutation detection (MUT-MAP) panel for detecting over 120 hotspots across 13 genes using 2-100 ng of extracted DNA from fresh frozen and formalin-fixed paraffin-embedded tissue samples. Additionally, the same chip integrates CNV analysis of 24 genes.
11:35-12:00 pm Next-Generation Sequencing of Hematologic Malignancies in the CLIA Environment
Rajyalakshmi Luthra, Ph.D., Professor, Hematopathology; Director, Molecular Genetic Pathology Fellowship Program; Director, Molecular Diagnostic Laboratory, MD Anderson Cancer Center
Clinical NGS-based testing for simultaneous detection of somatic mutations in multiple genes provides insights into prognosis and therapeutic choices for clinical management of patients with leukemia. However, the constantly changing NGS landscape presents challenges in terms of clinical validation and implementation. This presentation will discuss clinical validation, data processing, interpretation and reporting of NGS-based targeted sequencing in the CLIA environment.
12:00-12:25 Circulating Tumor DNA as a Biomarker of Response and Resistance to Treatment
Abhijit Patel, M.D., Ph.D., Assistant Professor, Therapeutic Radiology, Yale University School of Medicine
Our group has developed an ultrasensitive, multi-target assay that can identify and quantify mutant ctDNA using error-suppressed next-generation sequencing. Broad coverage of mutation hotspots and warm-spots allows detection of ctDNA without prior knowledge of the tumor’s mutation profile. Clinical examples will be presented in which this approach is used to non-invasively monitor changes in ctDNA levels in response to treatment and to track the emergence of mutations that confer resistance to targeted therapies.
12:25-1:55 Enjoy Lunch on Your Own