2014 Archived Content

Cambridge Healthtech Institute’s Inaugural

Clinical Epigenetics

Interrogating Disease Epigenomes for Diagnostic, Prognostic & Therapeutic Utility

February 10-12, 2014 | Moscone North Convention Center | San Francisco, CA


Day 1 | Day 2 | Day 3 | Download Brochure 

Wednesday, February 12

7:00 am Breakfast Presentation (Sponsorship Opportunity Available) or Morning Coffee

8:00 Plenary Keynote Session (Click Here For More Details) 

9:45 Refreshment Break and Poster Competition Winner Announced in the Exhibit Hall


10:35 Chairperson’s Remarks

Michelle M. Hanna, Ph.D., CEO & Scientific Director, RiboMed Biotechnologies, Inc.

10:40 Identification of Genome-Wide Methylated CpG Island Profiles of Coding, Non-Coding and Repeated Regions as Molecular Markers for Human Melanomas

Ranjan J. Perera, Ph.D., Associate Professor & Scientific Director, Genomics and Bioinformatics, Sanford-Burnham Medical Research Institute

We have identified genome-wide methylated CpG island distributions by subjecting melanoma genomic DNA to methyl binding domain 2 (MBD2) pull-down and NGS. CpG islands in the upstream regulatory regions of many coding and non-coding RNA genes exhibit extensive hypermethylation, whereas several repeated elements, such as LINE 2, and several LTR elements, are hypomethylated in advanced stage melanoma cell lines. Focused assays of melanoma patient tissue samples for CpG island methylation near the non-coding RNA genes demonstrated high specificity.

11:10 miRNA Biomarkers for Colorectal Neoplasia

Ajay Goel, Ph.D., Director, Epigenetics and Cancer Prevention, Baylor Research Institute

MicroRNAs (or miRNAs) are small transcripts of 20-24 nucleotides that have emerged as important regulators of gene expression in cancer cells. Overexpression of specific miRNAs has been linked to the stepwise disease progression during the normal-adenoma-cancer sequence in the colorectal cancer (CRC). Given their cancer-specific pattern of expression, remarkable stability and presence in blood and other body fluids, miRNAs are considered to be highly promising cancer biomarkers.

11:40 PANEL DISCUSSION: Developing and Commercializing Epigenetic Diagnostics


Perry Dimas, VP, Business Development, Premier Source Diagnostics


- Michelle M. Hanna, Ph.D., CEO & Scientific Director, RiboMed Biotechnologies, Inc.

- Noel Doheny, CEO, Epigenomics, Inc.

- Babak Alizadeh, Ph.D., Co-founder & COO, PrognosDx Health, Inc.

- Jason Ross, Ph.D., Research Scientist, Animal Food and Health Sciences, CSIRO

Epigenetic marks, such as DNA methylation and histone modifications, comprise part of the epigenetic machinery leading to abnormal gene expression and chromatin instability in disease. Epigenetic changes, particularly in human cancers, are now being considered as novel biological markers for diagnostic and therapeutic utility. This panel will discuss current challenges in developing and commercializing epigenetic diagnostics by addressing three specific areas of concern:

  • DNA Methylation as Viable Biomarkers
  • Validation of Assay and Technologies
  • CPT Coding and Reimbursement Challenges

12:10 pm Session Break

12:20 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

1:00 Refreshment Break in the Exhibit Hall and Last Chance for Poster Viewing


1:40 Chairperson’s Remarks

Peter Staller, Ph.D., Director, Oncology Research, EpiTherapeutics ApS

1:45 Development of Specific and Reversible Inhibitors of Lysine Specific Demethylase 1 (LSD1)

Sunil Sharma, M.D., Division Chief, Medical Oncology, Jon and Karen Huntsman Professor, Cancer Research, Huntsman Cancer Institute, University of Utah; CMO, Salarius Pharmaceuticals

I will describe the efforts to develop inhibitors of LSD1. LSD1 is an important histone demethylase that is important for histone K4 and K9 demethylation. It has an emerging role in various cancers. In this presentation, I will review the biology and rationale for LSD1 inhibition and various strategies to inhibit its activity.

2:15 Inhibition of LSD1 as a Therapeutic Strategy for the Treatment of Acute Myeloid Leukemia

Helai Mohammad, Ph.D., Investigator, Cancer Epigenetics DPU, Oncology R&D, GlaxoSmithKline

Lysine specific demethylase 1 (LSD1) is a H3K4me1/2 demethylase found in various transcriptional co-repressor complexes. The current study describes the anti-tumor effects of a novel, potent, selective, irreversible GSK LSD1 inhibitor. Our pre-clinical data demonstrate that pharmacological inhibition of LSD1 may provide a promising treatment for AML by promoting differentiation and subsequent growth inhibition of AML cells.

2:45 Co-Presentation: The Constellation-Genentech Bromodomain Drug Discovery Platform

Andrea Cochran, Ph.D., Senior Scientist, Early Discovery Biochemistry, Genentech, Inc.

Steve Bellon, Ph.D., Director, Structural Biology, Constellation Pharmaceuticals

We will describe a platform for discovery of potent and selective bromodomain probes for use in target validation studies. The platform consists of medicinal chemistry, biophysics and structural biology applied to understanding how bromodomains recognize both chromatin and small molecules. Cell-permeable small-molecule inhibitors are useful tools to investigate the function of bromodomain proteins in activating transcription and may provide starting points for the development of novel drugs.

3:15 Mechanisms of Response to BET Bromodomain Inhibition in Malignant Peripheral Nerve Sheath Tumors

Lu Q. Le, M.D., Ph.D., Assistant Professor, Department of Dermatology, Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center

Malignant Peripheral Nerve Sheath Tumors (MPNSTs) are highly aggressive sarcomas that are typically fatal. To gain insights into MPNST pathogenesis, we took advantage of a novel MPNST mouse model that permits the study of tumor evolution, which allowed us to identify and elucidate mechanisms for chromatin regulator BRD4 in promoting MPNST tumorigenesis. These findings provide a strong preclinical basis for evaluating BRD4 inhibitors as novel therapies for these life-threatening tumors.

3:45 Refreshment Break

4:05 The Potential Applications of Enzymatic Inhibitors of KDM5 in Oncology

Peter Staller, Ph.D., Director, Oncology Research, EpiTherapeutics ApS

The histone demethylases KDM5A and KDM5B target the methylation of histone H3 at lysine 4 and contribute to cancer cell proliferation and to the induction of drug tolerance. EpiTherapeutics has developed specific and potent inhibitors of KDM5. The pharmacological properties of selected compounds and their in vivo activity as well as potential therapeutic applications will be discussed.


4:35 Resminostat, A Novel Histone Deacetylase Inhibitor in Development for the Treatment of Advanced Hepatocellular Carcinoma (HCC): A New Biomarker-Driven Epigenetic Therapy Option

Bernd Hentsch, Ph.D., Chief Development Officer, 4SC AG

Resminostat has been clinically studied in patients with hepatocellular carcinoma (HCC), Hodgkin´s Lymphoma (HL) and colorectal cancer. A biomarker program was established identifying transcriptional profiles modulated upon resminostat exposure. ZFP64 was identified as a biomarker strongly downregulated by resminostat. Expression levels of ZFP64 measured prior to resminostat treatment in peripheral blood cells correlated with the clinical outcome, i.e., patients displaying high ZFP64 baseline levels experienced a substantially longer overall survival.

5:05 Bromodomain Inhibition for Treating Human Diseases

Norman C.W. Wong, M.D., CSO & Co-Founder, Resverlogix

The knowledge gained from 5 years of human clinical trials using RVX-208, an orally active BET-protein inhibitor, has provided valuable information in the design of a discovery platform. New compounds identified using this platform have proven to be active in cellular and animal models of cancerous and inflammatory diseases. The latest data detailing characteristics of novel BET-protein inhibitors will be presented.

5:35 Close of Conference Program

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