Cambridge Healthtech Institute’s 2nd Annual

Biomarkers for Immunotherapy

Predictive and Prognostic Biomarker Discovery for Successful Therapy

7-8 May 2019


Despite the revolution of immunotherapy for cancer treatment in recent years, many treated patients do not respond, not mentioning some potential serious side effects associated with these treatments. There is a pressing need for the discovery of prognostic and predictive biomarkers that will improve the selection of patients who will best respond to therapy and help tailor therapy regimens. CHI’s Second Annual “Biomarkers for Immunotherapy” will bring together biomarkers experts from industry and academia to address advances in the discovery and development of predictive and prognostic biomarkers for immunotherapy.

Final Agenda

Recommended Short Course* Monday 6 May | 13:30 - 17:00

SC2: Biomarkers in Liquid Biopsy: CTCs, ctDNA and Exosomes

Lorena Diéguez, PhD, Group Leader, Department of Life Sciences, Nano4Health Unit, Medical Devices Research Group, International Iberian Nanotechnology Laboratory, Portugal

Roberto Piñeiro Cid, PhD, Cancer Modeling Lab, Instituto de Investigación Sanitaria de Santiago de Compostela- Roche-Chus Joint Unit, Spain

Biomarkers for early disease detection, therapeutic efficacy monitoring and outcome prediction are the key to precision medicine. Liquid Biopsy studies disease biomarkers in body fluids and can be paramount for precision medicine in cancer. The analysis of biomarkers in peripheral blood improves cancer diagnosis and treatment success. This course will give you a comprehensive overview and update on the established biomarkers, available technologies and clinical applications of liquid biopsy.

*Separate registration required.


08:00 Registration and Morning Coffee


08:55 Organizer’s Opening Remarks

Ngoc 'Emily' Le, PhD, Conference Producer, Cambridge Healthtech Institute, United States

09:00 Chairperson's Remarks

Ed Schuuring, PhD, Professor & Head, Molecular Pathology, University Medical Center Groningen, The Netherlands

09:05 Immune Contexture Heterogeneity and Clinical Impact

Galon_JeromeJérôme Galon, Director, Integrative Cancer Immunology Laboratory, Cordeliers Research Center, Research INSERM (French NIH) (first class, DR1), France

We developed and validated worldwide a standardized digital-pathology-based immune assay, “Immunoscore”, with prognostic power superior to the currently used cancer staging-system. Immunoscore and immunoediting were demonstrated to affect metastatic dissemination in humans. The newly-proposed “parallel immune selection model” of tumor evolution incorporates the effects of the immune system in shaping metastatic spread. The impact of the immune contexture on predicting response to immunotherapy, including CAR-T cells, will also be discussed.

09:35 The 10,000 Immunomes Project: Building a Resource for Human Immunology

Bhattacharya_SanchitaSanchita Bhattacharya, PhD, Bioinformatics Project Scientist, Bakar Computational Health Sciences Institute, University of California San Francisco, United States

One of the major barriers for understanding human immunological mechanisms is that immune assays have not been reproducibly characterized for a sufficiently large and diverse healthy human cohort. Here, we present the 10,000 Immunomes Project (10KIP), a framework for growing a diverse human immunology reference, from ImmPort, a publicly available resource of subject-level immunology data.

10:05 Comparing Immune Cell Populations and Novel Biomarkers in Subtypes of NSCLC

Juncker-Jensen_AnnaAnna Juncker-Jensen, PhD, Medical Science Liasion, NeoGenomics Laboratories, USA

10:35 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing


11:15 Chairperson's Remarks

Ed Schuuring, PhD, Professor & Head, Molecular Pathology, University Medical Center Groningen, The Netherlands


11:15 KEYNOTE PRESENTATION: Development of a TMB Assay and the Challenges of Harmonizing across Products

Michael Doherty, Senior Vice President, Head of Product Development, Foundation Medicine, United States

A novel, technically robust, blood-based assay to measure TMB in plasma (bTMB) that is distinct from tissue-based approaches will be described. Using a retrospective analysis of two large randomized trials as test and validation studies, we show that bTMB reproducibly identifies patients who derive clinically significant improvements in progression-free survival from atezolizumab (an anti-PD-L1) in second-line and higher NSCLC. Our data show that high bTMB is a clinically actionable biomarker for atezolizumab in NSCLC. Challenges and efforts to harmonize across products will also be discussed.

11:45 NEW: Is It Time for an International Genomic Reference Panel for MSI and TMB?

Lo-Cascio_LeandroLeandro Lo-Cascio, PhD, Standard Development Senior Scientist, Advanced Therapies, National Institute for Biological Standards and Control (NIBSC)

For the generation of a reference material our approach includes sourcing and characterization of MSS and MSI-H cell lines, as well as generation and characterization of novel MSI-H cell lines via CRISPR-engineering. At present a panel of 10 cancer and control cell lines has been sourced and characterised for MSI using a PCR-based approach. WGS and targeted panel sequencing was performed on candidate cell lines. MSIsensor was used to calculate MSI status, to confirm PCR-based findings. Further characterization will be performed with other commercially available MSI assays and further bioinformatics approaches. Furthermore, we are aiming to generate MSI-H cell lines via CRISPR, targeting genes of the MMR system in a MSS cell line. Benefits of this approach will be the generation of MSI-H/MSS paired samples. MSI and TMB represent useful biomarkers to select cancer patients that will benefit from targeted therapies. Standards are required for accurate and sensitive diagnostic testing. Thus, we appreciate the necessity of a reference material intended as a non-assay-specific primary standard to calibrate MSI and TMB measurements.

12:15 Expression of Endogenous Retrovirus as a Potential Biomarker of Response to Immune Checkpoint Therapy in Low Mutation Burden Cancers

Ganesan_ShridarShridar Ganesan, MD, PhD, Chief, Molecular Oncology; Associate Director, Translational Research; Omar Boraie Chair, Genomic Science, Rutgers University, United States

To better understand response of low mutation burden cancers to ICB, we investigated the expression of endogenous retroviruses (ERVs) and their association with markers of immune infiltration and immune checkpoint activation. Endogenous retroviruses are normally not expressed in most somatic tissues, but abnormal expression has been reported in multiple cancer types. Expression of certain classes of ERV was associated with markers of immune activation in several tumor classes, but most strikingly in clear cell renal cancer. ERV expression was associated with evidence of chromatin abnormalities, and with increased response to immune checkpoint blockade in ccRCC. These observations suggest that ERV expression may be novel biomarker of response to ICB in certain cancers including ccRCC.

12:45 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own


14:15 Chairperson's Remarks

Shridar Ganesan, MD, PhD, Chief, Molecular Oncology; Associate Director, Translational Research; Omar Boraie Chair, Genomic Science, Rutgers University, United States

14:20 Circulating Tumor DNA as a Molecular Bloodborne-Based Biomarker to Predict Tumor Response in Lung Cancer Patients Treated with Immunotherapy

Schuuring_EdEd Schuuring, PhD, Professor & Head, Molecular Pathology, University Medical Center Groningen, The Netherlands

A significant minority of patients treated with immune checkpoint inhibitors shows durable responses but no adequate biomarkers are available for predicting which patients will benefit. Immunotherapy is expensive and potentially toxic. Tumor response is monitored by tumor volume using CT scanning. The aim of our study is to assess plasma levels of non-targetable tumor-specific mutations as molecular biomarkers and monitoring tool for durable responses to immunotherapy in advanced NSCLC.

14:50 Tumor Immunoprofiling: Novel Tissue-Based Biomarkers in Cancer Immunotherapy

Giraldo-Castillo_NicolasNicolas A. Giraldo-Castillo, MD, PhD, Pathology Resident, Johns Hopkins University School of Medicine, United States

Therapeutic blockade of the PD-1/PD-L1 checkpoint has been embraced as a strategy to enhance antitumor T-cell immunity, with durable efficacy in multiple tumor types. The best-studied biomarker to predict response to these agents is PD-L1 protein expression, measured by immunohistochemistry and graded “positive” or “negative.” Using digital pathology-assisted methods, our laboratory has found that quantitative assessments of PD-1+ and PD-L1+ cell densities, as well as geographic interactions between these two cell populations, correlate with clinical response to anti-PD-1 therapy in Merkel cell carcinoma, melanoma, and renal cell carcinoma and is superior to binomial scoring systems.

Adaptive-Biotechnologies 15:20 Implementing Immunosequencing as New Molecular Biomarkers of Response to Immunotherapy

Catherine Sanders, PhD, Senior Director, Research and Business Development, Adaptive Biotechnologies, USA

Adaptive Biotechnologies’ immunosequencing technology combines bias-controlled multiplex PCR, high-throughput sequencing, and sophisticated bioinformatics. In solid tumors, the immunoSEQ Assay accurately quantifies TIL density and clonality, with preclinical and clinical applications to inform repertoire changes in response to single agent or combination therapies that have potential prognostic and predictive value.

15:50 Immunophenotyping and Functional Assays for Predictive Marker Discovery

Wang_JunxiaJunxia Wang, PhD, Director, Analytical Development, Mustang Bio, Inc., United States

The discovery and development of predictive for CAR T cells have unique challenges as compared to conventional drugs as the clinical outcome is largely dependent on a viable cellular drug. To overcome the challenges, we have implemented an integrated clinical and CMC biomarker strategy to extend our biomarker study for CAR T cells to include the characterization of the cells prior to engineering and the final product at the site of manufacturing facility. To this purpose, a series of immunophenotyping and functional assays have been developed for the discovery of predictive markers of treatment efficacy and toxicity.

16:20 Refreshment Break in the Exhibit Hall with Poster Viewing

17:00 Breakout Discussions View Details

18:00 Welcome Reception in the Exhibit Hall with Poster Viewing

19:00 Close of Day


08:00 Registration and Morning Coffee


09:00 Chairperson's Remarks

Christopher M. Hartshorn, PhD, Program Director, NIH NCI, United States

09:05 Current Initiatives and Research Efforts at the National Cancer Institute Dedicated to Cancer Immunotherapy

Hartshorn_ChristopherChristopher M. Hartshorn, PhD, Program Director, NIH NCI, United States

The US National Cancer Institute (NCI) of the National Institutes of Health has played an integral role in the research and funding of cancer immunotherapies over the last several decades. As the core understanding as to the interface between cancer biology and immunological response have evolved, so has the NCI’s conceptual vision of this unique therapeutic modality and the needs of the field. This talk will focus on the most recent and relevant efforts focused at developing markers and tools for patient stratification, deciphering the complex nature of responders vs non-responders, and earlier intervention after dosing.

09:35 Interpretation and Validation of Longitudinal Serum Tumor Biomarker Changes for Early Prediction of Immunotherapy Non-Responsiveness

Rossum_Huub_vanHuub H. van Rossum, PhD, EuSpLM, Specialist, Laboratory Medicine and Clinical Chemistry, Laboratory Medicine, The Netherlands Cancer Institute, The Netherlands

For NSCLC, only a modest number of patients treated with immune checkpoint inhibitors will respond to this treatment. Serum tumor biomarkers may be of value and alert the clinician of possible treatment failure. The Re-marker platform was developed to support basal longitudinal biomarker investigations including BReC-plot generation and the diagnostic validation of biomarker-response based tests. Using this tool, biomarker response-based tests could be designed that accurately predict non-responsiveness to immunotherapy.

10:05 Predicting Immunotherapy Response and Toxicity in Melanoma

Rizos_HelenHelen Rizos, PhD, Head, Biomedical Sciences, Faculty of Medicine and Health Sciences, Macquarie University, Australia

There is an urgent need to identify biomarkers that accurately predict for treatment response, and guide the selection of novel combination therapies for patients who acquire resistance. There is also a requirement to accurately predict whether response will be associated with significant adverse events. In this presentation, the utility of circulating biomarkers as predictive and prognostic markers in melanoma, including circulating cytokines, exosomes and circulating tumour DNA, will be discussed.

10:35 Highly-Characterized TMB Reference Standards to Standardize and Harmonize TMB Measurements

Dan Brudzewsky, Field Application Scientist, Clinical Genomics, SeraCare Life Sciences, USA

Tumor mutational burden (TMB) is a pan-cancer biomarker for assessing immuno-oncology drug response. SeraCare is collaborating with FOCR and IQN-Path to develop TMB reference standards with confirmed mutational burden to standardize and harmonize TMB measurements. We describe the characterization of a panel of gDNA and FFPE-based TMB reference materials by WES.

11:05 Coffee Break in the Exhibit Hall with Poster Viewing



11:35 Moderator’s Remarks

Charlotte Ryckman, Associate, Covington & Burling LLP, Belgium

11:45 Precision Diagnostics in Oncology: Expanding Roles of Liquid Biopsies

Nitzan Rosenfeld, PhD, Senior Group Leader, Cancer Research UK Cambridge Institute, University of Cambridge; CSO, Inivata Ltd., United Kingdom

Effective clinical management relies on accurate diagnostic information, which requires effective techniques and the right samples. Next generation sequencing can provide a wealth of information, but implementing innovative technologies into clinical routine can be a challenge. We’ll examine how analysis of cell-free DNA can provide an opportunity to re-examine many of the current clinical decision points, and a test case for adoption of new diagnostic tools.

12:15 Legal and Regulatory Developments in Precision Medicine and Diagnostic Devices

Erik Vollebregt, Partner, Axon Lawyers, The Netherlands





  • What changes will be brought about by the IVDR?
  • What is the impact of the GDPR in the field of precision medicine and diagnostic devices?
  • What are the practical implications of implementation of new European regulations?
  • What are the consequences of the interplay of the IVDR and the GDPR?

12:45 PANEL DISCUSSION: Challenges and Opportunities in European Diagnostic Investments



Philippe Peltier, Partner, Kurma Partners, France


Florian Kainzinger, PhD, Managing Partner, Founder, Think.Health Ventures, Germany

Makinen_SeppoSeppo Mäkinen, Partner, Pathena Investments

  • What is different in Europe versus other markets (e.g., US and Israel). How do different European markets compare?
  • What has changed in the landscape of European investments over the past few years? What can be improved?
  • The role of regulators and governments
  • How can start-ups stand out and get attention in the current landscape?

13:30 Close of Biomarkers for Immunotherapy