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
11:50-12:05 pm Sponsored Presentation (Opportunity available)
12:05-12:50 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
1:30-1:55 Biomarkers in Translational Medicine: From Bench to the Clinic and Back
Suso Platero, Ph.D., Director, Oncology Biomarkers, Janssen Pharmaceuticals
Translational medicine could be defined as the application of biomarkers from preclinical models to clinical trials. But also, once we start dosing patients, the generation of hypothesis using clinical samples is a key feature of translational medicine for our understanding of the interaction of the drug with the specific disease. By looking at patients’ samples and correlating their status to drug responses we can identify biomarkers that are important for mechanism of action of the drug and sensitivity of the patient’s disease to the drug. Uniting both approaches will yield better biomarkers that can later on be used in subsequent phases of drug development.
1:55-2:20 Translation of Emerging Biomarkers from Preclinical Species to Human Populations
Jiri Aubrecht, Pharm.D., Ph.D., Senior Director and Safety Biomarker Group Lead, Drug Safety Research & Development, Pfizer
Biomarkers provide essential tools for refining of therapeutic index in drug development, monitoring disease progression and/or examining effects of environmental exposure to chemicals. Despite the success of routine markers used in preclinical and clinical settings a new cohort of safety biomarkers with better sensitivity and specificity is being evaluated. This includes tissue-specific proteins, metabolites and/or circulating miRNAs. Since drug development relies on preclinical evaluation of lead compounds, the biomarker translation across animal species to humans is essential. To study the performance of emerging biomarkers we employ a variety of approaches including animal models and clinical studies. The presentation will introduce recent progress in applying state-of-the-art technologies such as next-generation sequencing in biomarker development as well as several case studies documenting advances in cross species translation for biomarkers of hepatotoxicity and nephrotoxicity.
2:20-2:45 Translational Biomarkers: Need, Progress and Challenges
Donna L. Mendrick, Ph.D., Director, Systems Biology, NCTR, FDA
Biomarkers used in animal testing and in the clinic to identify adverse drug events are inadequate to identify population-based safety risks and individual susceptibilities. System biological approaches offer new discovery modalities to improve the identification and mechanistic understanding of new biomarkers. Published literature is filled with reports of potentially novel and useful biomarkers; however, academic labs do not receive funding to work toward regulatory qualification of such biomarkers so they tend to remain the subject of single publications. To effect change, new biomarkers need to be accepted by the community at large.
2:45-3:45 Refreshment Break in the Exhibit Hall with Poster Viewing
3:45-4:15 Sponsored Presentation
Denis Smirnov, Ph.D., Associate Scientific Director, US Biomarker Oncology, Janssen R&D US
4:15-4:30 A Simple Blood Test to Assess Insulin Resistance (IR) in Clinical Trials: Quantose IRTM
Nelson Rhodes, Ph.D., Associate Study Director, Metabolon, Inc.
4:30-5:00 Sponsored Presentation (Opportunity Available)
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
8:30-8:55 Big Data and Small Trials: Translating Biological Data into Clinical Biomarkers
Nicholas C. Dracopoli, Ph.D., Vice President, Janssen R&D, Johnson & Johnson
All of the companion diagnostic tests approved by the FDA for use in oncology are for “driver mutations” in genes involved in signal transduction pathways. These tests are for single analytes predicting the functional status of the drug target or pathway. There are no approved companion diagnostics for drugs that work through alternative mechanisms such as chemotherapy or immunomodulation. This presentation will discuss why so few biomarkers have been developed, and why we have mostly failed to develop molecular profiles that predict drug response.
8:55-9:20 Talk Title to be Announced
Emmanuelle Di Tomaso, Ph.D., Global Correlative Science Lead, Novartis Institutes for BioMedical Research
9:20-9:45 Clinical Trial Biomarkers in a World of Big Data and Predictive Analytics
Michael Hale, Ph.D., Executive Director, Medical Sciences Biostatistics, Amgen
9:45-10:45 Coffee Break in the Exhibit Hall with Poster Viewing
10:45-11:10 IBM Watson and the Valley of Death
Mark S. Boguski, M.D., Ph.D., Associate Professor, Pathology, Center for Biomedical Informatics, Harvard Medical School
There is a large and widening gap created by our ability to generate data much faster than we can ever ascribe meaning to it via traditional approaches. This gap has been evident in biomedicine since the late 1990s and has now become a "Valley of Death" in the application of new technologies for biomarker discovery and clinical diagnostics. These technologies produce more data than we can ever hope to interpret by consulting the literature, for two reasons. First, there is no literature pertaining to most of the findings and perhaps never will be because the traditional model for follow-up and validation does not scale. Second, even when literature is available, most of it may represent non-reproducible results. For example, it has been estimated that the majority of "breakthrough" research in oncology, women's health and cardiovascular disease cannot be replicated or confirmed. Systems like IBM Watson that heavily leverage a literature corpus to find connections and make inferences suffer from obvious limitations in this context. There is a way out of the Valley of Death but it will require both researchers and technology developers to approach the problem differently.
11:10-11:35 Big Data and Reliability of Biomarker Identification
Andreas Schuppert, Ph.D., Vice President, Technology Development, Bayer Technology Services GmbH; Professor, AICES, RWTH Aachen University
“Big Data” offers great promise both to industry and clinics. Systematic analysis of very large, multivariate data sets is expected to be a powerful technology to improve the overall efficiency in the pharma pipeline as well as to enable the expected benefits of personalized medicine. However, the reality suffers from an apparent lack of reproducibility of the data resulting in a lack of reliability in the biomarkers for complex diseases. We demonstrate on a heterogeneous set of gene expression data from cancer studies that a systematic utilization of the intrinsic correlations in highly multivariate data can be used to extract scores enabling the quantitative characterization of tumor status with a high degree of stability.
11:35-12:00 Talk Title to be Announced
Iya Khalil, Ph.D., Executive Vice President and Co-Founder, GNS Healthcare
12:00-12:30 Sponsored Presentation (Opportunity available)
12:30-1:55 Enjoy Lunch on Your Own