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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 firstname.lastname@example.org or 781-972-5457
8:30-8:55 Evaluation of Predictive Biomarkers in CTCs in NSCLC Patients Undergoing Targeted Therapies
Edith Szafer-Glusman, Ph.D., Senior Research Associate, Development Sciences, Genentech
Some of the challenges in the management of NSCLC patients are the lack of biomarkers that indicate response to therapy, and the difficulty in obtaining biopsies to evaluate biomarkers of targeted therapies. Here we show that molecular characterization of circulating tumor cells is feasible in advance lung cancer clinical trials and provide information about patient biomarker status. In addition, changes in CTC numbers are associated with clinical response in patients treated with erlotinib and pertuzumab. Finally, I’ll elaborate on the development of novel CTC biomarkers for the monitoring of patient molecular status in ongoing clinical trials.
8:55-9:20 Role of CTC-Based Pharmacodynamic Biomarkers in Drug Development and Clinical Trials of Targeted Anti-Cancer Therapeutics
Lihua Wang, Ph.D., Senior Scientist, PADIS, LHTP, Applied Developmental Directorate, SAIC-Frederick, NCI/NIH
In combination with circulating tumor cells (CTC) counts, monitoring of biomarker changes within CTCs provide evidence for pharmacodynamic effects of targeted anti-cancer agents in patients enrolled in clinic trials. This presentation describes a strategy for development and validation of assays for pharmacodynamic biomarkers such as γH2AX or p16INK4a in CTCs. These CTC-based assays are in use in ongoing clinical trials of targeted anti-cancer agents within the National Cancer Institute. We found that in comparison to CTC counts alone, assaying molecular biomarker levels in CTCs is more sensitive for assessing pharmacodynamic effects in patients.
9:20-9:45 Molecular Characterization of CTCs: Tool for Optimizing Patient Management
Ryan Dittamore, Director, Medical Innovation, Ventana Medical Systems
Molecular characterization of CTCs is becoming more relevant in personalized disease management of patients with solid malignancies. Data will presented regarding the development of an automated multiplexed immunofluorescence and in situ hybridization assay for molecular profiling of CTCs. The results from these studies further support the hypothesis that the biological characteristics of CTCs will improve predicting risk, monitoring response, and tailoring treatment strategies for patients with solid tumors.
9:45-10:45 Coffee Break in the Exhibit Hall with Poster Viewing
10:45-11:10 Answering a Question with a Question: Circulating Tumor Cells from a Surgeon’s Perspective
Daniel Boffa, M.D., Assistant Professor of Surgery, Yale School of Medicine
Circulating tumor cells have raised as many questions as they have shown the potential to answer. The surgical perspective to metastatic progression has been particularly influenced by understanding of the fluid phase of cancer progression, challenging the concept of localized disease, and radiographic staging. The presentation will focus on the surgical aspects of cancer care most likely to benefit from the use of CTC enumeration and characterization.
11:10-11:40 Characterization of Circulating Tumor Cells With the CellSearch Platform
Brad Foulk, Scientist, Janssen R&D
Beginning in 2004 the CellSearch platform has been FDA approved to enumerate circulating tumor cells(CTC)in metastatic breast, prostate, and colorectal cancer patients. Since that time the platform has proven to be a valuable research tool for the characterization of CTCs as well. A review of techniques to characterize CTC by molecular and cellular methods will be presented.
11:40 am-12:10 pm Sponsored Presentations (Opportunities Available)
Contact Ilana Quigley at email@example.com or 781-972-5457
12:10-1:30 Enjoy Lunch on Your Own
1:30-1:35 Chairperson’s Opening Remarks
1:35-2:00 Advances in Circulating Tumor Cell Analysis: Analytical Validation Must Precede Clinical Application
Martin Fleisher, Ph.D., Chief, Clinical Chemistry Service, Department of Laboratory Medicine, Memorial Sloan-Kettering Cancer Center
Detecting circulating tumor cells (CTCs) in peripheral blood of patients with cancer has become a clinically relevant and important prognostic biomarker and has been shown to be a predictive biomarker post-therapy. Currently, one analytical method, the Veridex CellTrack Analyzer, has been FDA approved for use in monitoring therapeutic response in patients with breast, prostate and colon cancer. This enrichment technique requires expression of EpCAM by CTCs. Other methods to enrich CTCs that rely on size, shape and phenotypic characteristics are: (a) flow cytometry that sorts cells by size and surface antigen expression; (b) microchips designed to capture CTCs as blood flows past EpCAM-coated mircoposts; (c) filters with pore size designed to retain CTCs but permits smaller cells to pass; (d) imaging techniques that rely on Fiber-Optic Array Scanning Technology (FAST) that identify CTCs based on fluorescent labels; and (e) negative enrichment that eliminates all cells from blood samples, except CTCs. The successful clinical application of these emerging CTC technologies depends on rigorous analytical validation. A validation protocol, consistent with CLIA regulator guidelines will be presented.
2:00-2:25 Clinical Microfluidics: Isolation and Molecular Characterization of Circulating Tumor Cells Using the CTC-Chip
David T. Ting, M.D., Assistant Physician and Instructor in Medicine, Massachusetts General Hospital Cancer Center, Harvard Medical School
This presentation will describe the engineering design and clinical validation of a high throughput microfluidic mixing device, the “CTC-chip,” that allows the isolation and characterization of CTCs from the peripheral blood of cancer patients. Multi-parameter characterization was conducted on CTCs to help better understand their origin and metastatic potential.
2:25-2:55 Molecular Analysis of Captured Circulating Melanoma Cells
John Viator, Ph.D., Associate Professor, Biological Engineering, University of Missouri
Photoacoustic flowmetry is a method to test blood samples for circulating tumor cells. By irradiating the samples under flow with nanosecond duration laser light, we induced high frequency acoustic waves in melanoma cells from metastatic patients. The melanin pigment within the melanoma cells absorbed the laser light and generated the photoacoustic waves. Using a microfluidic technique known as two phase flow, we captured the melanoma cells and performed molecular assays to verify the nature of these rare tumor cells. These results suggest that photoacoustic flowmetry may be used for early detection of metastatic disease in melanoma patients.
2:55-3:20 Single-Cell Functional and Genomic Analysis of Circulating Tumor Cells Captured with Geometrically Enhanced Differential Immunocapture
Brian J. Kirby, Ph.D., Associate Professor, Sibley School of Mechanical and Aerospace Engineering, Cornell University; Associate Professor of Engineering in Medicine, Division of Hematology and Oncology, Weill-Cornell Medical College
We present recent work in which single circulating tumor cells captured with GEDI (geometrically enhanced differential immunocapture) are probed to identify genomic content and functional response to chemotherapeutics. GEDI microdevices are used to isolate circulating tumor cells from prostate and pancreatic cancer patients while rejecting contaminating leukocytes, and both genomic analysis and in situ immunostaining are used to probe mutation and functional response.
3:20-4:20 Refreshment Break in the Exhibit Hall with Poster Viewing
4:20-4:45 4-Dimensional Biopsy of Lung Cancer
Lyudmila Bazhenova, M.D., Associate Clinical Professor, Medicine, Division of Hematology-Oncology; Lung Cancer Team Leader, Moores UCSD Cancer Center
We have established a fluid phase biopsy approach that identifies CTCs in way that preserves cytologic features in high-definition (HD) for diagnostic pathology imaging requirements without using immune- or surface receptor-based enrichment. HD-CTCs identified with this approach can be used for enumerations and molecular characterization-based studies. We report performance of this assay in patients with NSCLC. 106 patients were recruited (I-10, II-3, III-34, IV-59); 85%, 79%, and 53% of patients with stages I+II, III, and IV had detectable HD-CTCs. 28 patients with stage IV disease were analyzed with multiple draws over time and showed differential survival based on CTC counts. Proposed clinical utility of CTC in NSCLC will also be discussed.
4:45-5:10 Nano-Velcro Embedded Devices for Detection of Circulating Tumor Cells in Whole Blood: A Validation Study in Prostate Cancer Clinic
Hsian-Rong Tseng, Ph.D., Associate Professor, Molecular and Medical Pharmacology, Crump Institute for Molecular Imaging, UCLA
Our joint research team at UCLA has developed a new cell capture technology for detecting circulating tumor cells (CTCs) in blood samples collected from metastatic cancer patients. Similar to most of the existing approaches, anti-EpCAM was grafted onto the surfaces to distinguish CTCs from the surrounding hematologic cells. The uniqueness of our technology is the use of nanostructured surfaces, which facilitates local topographical interactions between CTCs and substrates at the very first cell/substrate contacting time point. We demonstrated the ability of these nanostructured substrates to capture CTCs in whole blood samples with significantly improved efficiency and selectivity.
5:10-5:35 Circulating Tumor Cell (CTC) Technologies for Melanoma
Gavin P. Robertson, Ph.D., Professor, Pharmacology, Pathology, Dermatology and Surgery, Penn State University; Director, Penn State Melanoma Center, Penn State Melanoma Therapeutics Program, and Foreman Foundation Melanoma Research Laboratory
Melanoma is a highly metastatic cancer disseminating through CTCs in the blood and lymphatic fluids. One gram of tumor tissue in a patient is predicted to slough off one million CTCs per day. Use of these cells as a liquid biopsy of the tumor is important for diagnosis, prognosis and therapeutic agent selection. Technological development for the isolation, characterization and study of melanoma CTCs for research and clinical applications are proceeding rapidly. This presentation discusses recent technological advances in the isolation, characterization and study of melanoma CTCs. Human, animal and bioengineered models will be highlighted, detailing studies on melanoma CTCs.
5:35-6:00 Identification and Separation of CTC Subsets
Patricia Burke, Ph.D., Scientist, Translational Medicine Oncology, MedImmune
This talk will address the identification and enrichment of CTCs and subsets of CTCs, and molecular characterization and utility of CTC subsets.
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