Scientific Program

The 2013 Scientific Committee Sessions will be held Saturday, December 7, and Sunday, December 8. Each session will be offered twice, including a session organized by the new ASH Ad Hoc Scientific Committee on Epigenetics and Genomics. A question-and-answer period will occur at the end of each individual speaker presentation. Invited abstracts of these sessions will be published in the Program Book and on the flash drive containing the annual meeting abstracts. In addition, this information will be available online in early November.

Scientific Program Co-Chairs:

José López, MD
Puget Sound Blood Center
Seattle, WA

Kevin Shannon, MD
University of California - San Francisco
San Francisco, CA

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Ad Hoc Scientific Committee on Epigenetics and Genomics

Histone Modifications in Normal and Malignant Hematopoiesis

Sessions Offered Twice:
Saturday, December 7, 2013
2:00 PM - 3:30 PM

Ernest N. Morial Convention Center (243-245)
Sunday, December 8, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (343-345)

This session will focus on the role of epigenetic modifications in normal and malignant hematopoiesis, including recently described DNA and histone modifications that affect gene expression, self-renewal, and transformation. The session will begin with a discussion of recent studies of DNA modifications and alterations in histone state and how these alterations affect gene expression and hematopoietic function. Subsequent talks will focus on how alterations in these epigenetic modifications contribute to disease states and how epigenetic alterations in hematologic malignancies have led to the identification and validation of novel therapeutic targets.

Dr. Kristian Helin will discuss recent data elucidating the role of TET proteins and of novel histone modifications in gene regulation and hematopoietic function, including insights from genome-wide epigenomic studies.

Dr. Brian Huntly will discuss recent studies of how epigenetic readers, which recognize specific histone marks, contribute to hematopoietic stem cell function and to malignant transformation, and how these studies have led to new insights into mechanisms of leukemic transformation.

Dr. Robert Copeland will discuss recent studies elucidating the role of mutations in epigenetic modifiers in hematologic malignancies and the subsequent development of specific epigenetic therapies targeting DOT1L and EZH2.

Chair:

Ross Levine, MD
Memorial Sloan-Kettering Cancer Center
New York, NY

Speakers:

Kristian Helin, PhD
University of Copenhagen
Copenhagen, Denmark
TET Proteins and Histone Modifications in Hematopoiesis

Brian J. P. Huntly, MD, PhD
University of Cambridge
Cambridge, United Kingdom
Role of Epigenetic Readers in Pathogenesis and Therapy of Acute Leukemias

Robert A. Copeland, PhD
Epizyme, Inc.
Cambridge, MA
Protein Methyltransferase Inhibitors as Personalized Cancer Therapeutics

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Ad-Hoc Scientific Committee on Bone Marrow Failure

Shwachman-Diamond Syndrome

Sessions Offered Twice:
Saturday, December 7, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (278-282)
Saturday, December 7, 2013
4:00 PM - 5:30 PM

Ernest N. Morial Convention Center (265-268)

Shwachman-Diamond Syndrome (SDS) is an inherited bone marrow failure syndrome characterized by exocrine pancreatic insufficiency, neutropenia, and metaphyseal dysostosis. Patients with SDS have an increased risk of developing myelodysplastic syndromes and acute myeloid leukemia. In the majority of patients, the disease is caused by mutations in the SBDS gene.

Dr. Akiko Shimamura will review the wide spectrum of clinical disease manifestations of SDS as well as the challenges in clinical and molecular diagnosis, the course of the disease, and current treatment options.

Dr. Johanna Rommens will discuss insights into the diverse disease pathology gained through modeling SDS in the mouse.

Dr. Alan Warren will discuss studies from his lab identifying a fundamental conserved role for the SBDS protein in maturation of the large ribosomal subunit. He will discuss new data identifying pathways responsible for disease that suggest potential targets for drug development.

Chair:

Monica Bessler
The Children's Hospital of Philadelphia
Philadelphia, PA

Speakers:

Akiko Shimamura, MD, PhD
Fred Hutchinson Cancer Research Center
Seattle, WA
Clinical Spectrum and Molecular Pathophysiology of Shwachman-Diamond Syndrome

Johanna M. Rommens, PhD
University of Toronto
Toronto, ON, Canada
Lessons from the Mouse Model

Alan John Warren, PhD, FRCP, FRCPath, FMedSci
University of Cambridge
Cambridge, United Kingdom
Linking Defective Ribosome Maturation to Shwachman-Diamond Syndrome

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Ad-Hoc Scientific Committee on Plasma Cell Neoplasia

Genomic Instability and Plasma Cells

Sessions Offered Twice:
Saturday, December 7, 2013
2:00 PM - 3:30 PM

Ernest N. Morial Convention Center (293-296)
Sunday, December 8, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (293-296)

This session will focus on understanding the genomic changes in myeloma, with emphasis on the role of aberrant somatic hypermutation in genome alterations and the landscape of myeloma mutations. The session will highlight the heterogeneous mutational spectrum, discuss the findings that the driver mutations are often subclonal and may co-exist in the same patient, and that in most cases, there is a complex subclonal structure which evolves over time.

Dr. David Schatz will focus on the mechanism by which somatic hypermutation (SHM) is targeted preferentially to immunoglobulin (Ig) genes. He will discuss recent data showing that Ig enhancers and enhancer-like elements constitute the central components of the DNA elements that recruit SHM preferentially to Ig loci and the protein factors that bind these DNA elements to facilitate SHM. These findings have implications for the mistargeting of SHM to non-Ig loci, including proto-oncogenes such as BCL6 and MYC, and are likely relevant to the pathogenesis of B-cell malignancies including multiple myeloma.

Dr. Andy Futreal will discuss the current status of somatic genetic changes in multiple myeloma. He will discuss striking heterogeneity of somatic mutations affecting both known cancer genes as well as novel candidate gene mutations previously not implicated in myeloma. Dr. Futreal will correlate identified cancer genes with disease subtypes and their significance, and compare the results in myeloma with other malignancies, especially B-cell tumors.

Dr. Peter Campbell will discuss diverse mutational processes that often act at different times, leading to complex clonal architecture and diverse processes contributing to the mutational repertoire. He will characterize subclonal and overlapping driver mutations, implying the striking variability of the initiation of molecular events that are associated with progression. Use of serial sample data and diverse patterns of clonal evolution including linear evolution, differential clonal response, and branching evolution will be discussed and compared with similar processes ongoing in other malignancies with implications for therapeutic decisions.

Chair:

Nikhil C. Munshi, MD
Dana-Farber Cancer Institute, Harvard Medical School, Boston Veterans Affairs Healthcare System
Boston, MA

Speakers:

David G. Schatz, PhD
Yale University
New Haven, CT
Protecting the Genome: Mechanisms Targeting Somatic Hypermutation

Andy Futreal, PhD
University of Texas MD Anderson Cancer Center
Houston, TX
The Mutational Profile of Plasma Cell Disorders

Peter J. Campbell, PhD
Wellcome Trust Sanger Institute
Cambridge, United Kingdom
Genomic Evolution and Clinical Correlates

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Scientific Committee on Blood Disorders in Childhood

Developmental Hematopoiesis Gone Awry

Sessions Offered Twice:
Saturday, December 7, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (271-273)
Sunday, December 8, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (271-273)

This session will explore recent advances in our understanding of the molecular pathways regulating hematopoiesis and how aberrations in these pathways contribute to hematologic disease. Presentations will span basic scientific and patient-oriented studies to address fundamental questions in developmental hematopoiesis.

Dr. Elaine Dzierzak will describe how hematopoietic stem cells form in the embryo and will also review key regulatory pathways involved in this process.

Dr. Nancy Speck will focus on the RUNX1 gene that is mutated in a familial leukemia syndrome and is essential for hematopoietic stem cell formation in the embryo. She will describe how loss-of-function RUNX1 mutations affect hematopoietic stem cell formation and function.

Dr. Marshall Horwitz will present recent insights from genetic studies of familial myelodysplastic syndromes and familial leukemia syndromes. He will also discuss the role of these genes in hematopoiesis and how mutations in these genes contribute to clonal evolution.

Chair:

Michael A. Pulsipher, MD
University of Utah School of Medicine/Primary Children's Medical Center
Salt Lake City, UT

Speakers:

Elaine Dzierzak, PhD
Erasmus Medical Center
Rotterdam, Netherlands
Ontogeny of Hematopoiesis

Nancy A. Speck, PhD
University of Pennsylvania
Philadelphia, PA
Inherited and Acquired RUNX1 Mutations in Hematologic Disorders

Marshall Horwitz, MD, PhD
University of Washington
Seattle, WA
Insights from Familial Leukemia and Myelodysplastic Syndromes

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Scientific Committee on Hematopathology and Clinical Laboratory Hematology

Innovations in Analyzing Hematopoietic Cell Phenotype and Function

Sessions Offered Twice:
Saturday, December 7, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (293-296)
Saturday, December 7, 2013
4:00 PM - 5:30 PM

Ernest N. Morial Convention Center (293-296)

This session will focus on innovative techniques that are being used to analyze hematopoietic cell phenotype and function. Speakers will cover new insights that these techniques have contributed to our knowledge of the pathogenesis of leukemias and lymphomas as well as our knowledge of immune cell function in benign conditions.

Dr. Garry Nolan and his colleagues have developed a novel mass flow cytometry technique that enables examination of large numbers of parameters on a single cell and allows for analysis of signaling responses in complex systems. Dr. Nolan will discuss the application of this technique and its impact on increasing our knowledge of leukemia cell biology.

Dr. Kojo Elenitoba-Johnson utilizes mass spectrometry-driven proteomics to identify proteins and post-translational modifications expressed by specific types of lymphomas. Dr. Elenitoba-Johnson will discuss the use of this technology for the identification of novel biomarkers and novel biologic insights underlying lymphoma pathogenesis and their therapeutic implications.

Dr. Mark Miller will discuss the principles and advantages of two-photon (2P) microscopy for studying cellular immune responses in vivo. Dr. Miller will present multiple examples in which imaging single-cell dynamics have uncovered novel immunological phenomena during infection, autoimmunity, and cancer. While 2P imaging has been used extensively to study immune cell trafficking and function in mice, more recently the technique has helped scientists acquire a multidimensional picture of disease in unfixed human biopsy tissues.

Chair:

LoAnn Peterson, MD
Northwestern University Feinberg School of Medicine
Chicago, IL

Speakers:

Garry Nolan, PhD
Stanford University
Palo Alto, CA
All Roads Lead to Rome: Phenotypic Channeling to Uniform Differentiation Structures from Diverse Leukemia Genotypes by Mass Cytometry

Kojo S.J. Elenitoba-Johnson, MD
University of Michigan
Ann Arbor, MI
Lost in Post-Translation, Found by Mass Spectrometry: Identification of Novel Biomarkers and Biologic Insights in Lymphoma Pathogenesis

Mark J. Miller, PhD
Washington University in St. Louis
St. Louis, MO
Two-Photon Imaging of Immune Cell Dynamics: Moving from 2D Fixed Tissue Sections to 3D Dynamic Histology In Vivo

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Scientific Committee on Hematopoiesis

Non-Coding RNAs in Normal and Malignant Hematopoiesis

Sessions Offered Twice:
Saturday, December 7, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (343-345)
Sunday, December 8, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (343-345)

The dark side, or non-coding portion of the human genome, comprises only one and a half percent of the entire genome. If one also considers the untranslated regions (UTRs), this increases to just two percent. Although there has been an explosion of data demonstrating the clinical relevance of one class of short, non-coding RNAs (ncRNAs) called microRNAs (miRNAs) in both normal development and in diseases such as cancer, less is known about the structure and function of other midsized and long ncRNAs (lncRNAs) such as large intergenic ncRNAs (lincRNAs), transcribed ultraconserved regions (T-UCRs), and small nucleolar RNAs (snoRNAs). This session will provide the latest scientific evidence for the roles of ncRNAs in health and disease with particular focus on their critical contributions to both normal and malignant hematopoiesis.

Dr. Mitchell Weiss will provide a comprehensive review of the field of non-coding RNAs and define the various classes and members. He will then provide a more focused review of the structure and function of the heterogeneous group of ncRNAs, called long non-coding RNAs (lncRNAs). Finally, he will discuss the work of his laboratory and others to identify and characterize long non-coding RNAs that are expressed during hematopoiesis and their roles specifically during erythro-megakaryopoiesis.

Dr. Howard Chang will discuss complicated interactions of lncRNAs, lincRNAs with various transcription factors resulting in altered chromatin states. These interactions are likely to play important roles in both normal and altered biologic processes, including cancer. Dr. Chang will attempt to define the rules that determine how lncRNAs alter transcriptional activity, the genes that they regulate, and their roles in dysregulated states such as cancer.

Dr. Pier Paolo Pandolfi will discuss exciting new data focused on the roles of pseudogenes, lincRNAs, and miRNAs in the pathogenesis of leukemia and lymphoma as also studied in vivo in the mouse. He will also focus on circular competing endogenous RNA (ceRNAs) and pseudo-ceRNAs and give important attention to how understanding the ceRNA language will facilitate efforts to deconvolute ceRNA networks in hematopoietic malignancies.

Chair:

John F. DiPersio, MD, PhD
Washington University School of Medicine
Saint Louis, MO

Speakers:

Mitchell J. Weiss
The Children's Hospital of Philadelphia
Philadelphia, PA
Role of Long Coding RNAs in Epigenetic Modulation of Hematopoiesis

Howard Y. Chang, MD, PhD
Stanford University
Stanford, CA
Genome Regulation by Long Non-Coding RNAs

Pier Paolo Pandolfi, MD, PhD
Beth Israel Deaconess Medical Center, Harvard Medical School
Boston, MA
ceRNAs and ceRNA Networks in Normal and Malignant Hematopoiesis and Their Therapeutic Implications

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Scientific Committee on Hemostasis

Engineering a New Generation of Hemostatic Agents

Sessions Offered Twice:
Saturday, December 7, 2013
4:00 PM - 5:30 PM

Ernest N. Morial Convention Center (271-273)
Sunday, December 8, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (260-262)

This is an exciting time for the hemophilia community, as a number of new hemostatic agents are in various stages of development or in clinical trials, including variants of naturally occurring coagulation proteins that have been engineered to have greater activity and/or longer duration of action. Such novel therapeutics hold the promise of improving the quality of life for hemophilia patients. This session will explore the scientific basis for some of the novel approaches.

Dr. Egon Persson will describe the conformational changes induced when factor VIIa binds to cofactor, tissue factor, and how changing a limited number of amino acids can induce similar conformational changes. This allows the design of FVIIa variants with increased intrinsic activity.

Dr. Robert Peters will discuss how the neonatal Fc receptor acts to prolong the lifetime of immunoglobulins and albumin and how this property can be utilized to enhance the half-life of therapeutic agents.

Dr. Alan Mast will discuss the prospect that inhibition of tissue factor pathway inhibitor might lead to increased thrombin generation and improved hemostasis in hemophilia.

Chair:

Maureane Hoffman, MD, PhD
Durham Veterans Affairs Medical Center
Durham, NC

Speakers:

Egon Persson, PhD
Novo Nordisk A/S
Måløv, Denmark
Engineering FVIIa Variants with Enhanced Activity

Robert Peters, Ph.D.
Biogen Idec Hemophilia
Cambridge, MA
Prolonging the Half-Life of Hemostatic Factors via the Neonatal Fc Receptor

Alan E. Mast, MD, PhD
Blood Center of Wisconsin
Milwaukee, WI
The Potential to Enhance Hemostasis by Inhibiting Tissue Factor Pathway Inhibitor

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Scientific Committee on Immunology and Host Defense

T-Cell Tolerance Exhaustion

Sessions Offered Twice:
Saturday, December 7, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (278-282)
Saturday, December 7, 2013
4:00 PM - 5:30 PM

Ernest N. Morial Convention Center (278-282)

Immune responses are dynamically regulated by a complex interplay between activating and inhibitory signals. This session will explore recent advances in our understanding of some of the factors that limit T-cell responses, and thus result in prevention of autoimmunity or alloimmunity as well as diminished anti-tumor or anti-viral immunity.

Dr. Maria Grazia Roncarolo will review current understanding of the spectrum of regulatory T cells and the results of clinical approaches to utilize such cells to prevent or treat autoimmunity and graft-versus-host disease. She will also discuss current concepts regarding mechanisms of T-cell-mediated immune suppression.

Dr. E. John Wherry will discuss the emerging understanding of cell intrinsic factors that regulate whether T cells exposed to chronic antigens maintain functionality or become senescent. Using studies of chronic viral infection in mice and in humans, Dr. Wherry will discuss the role of T-box transcription factors and other pathways that regulate T-cell exhaustion versus memory.

Dr. Stanley Riddell will discuss the role that T-cell exhaustion plays in limiting the effectiveness of adoptive cell therapies for chronic viral infection and cancer. He will discuss current approaches to preempt exhaustion in the context of adoptive immunotherapy trials, including the use of selected subsets of naïve, central memory, or stem cell memory cells for genetic manipulation.

Chair:

Gay M. Crooks, MBBS
University of California - Los Angeles David Geffen School of Medicine
Los Angeles, CA

Speakers:

Maria Grazia Roncarolo, MD
San Raffaele Scientific Institute
Milan, Italy
T-Cell-Mediated Suppression: From Bench to Bedside

E. John Wherry, Ph.D.
University of Pennsylvania School of Medicine
Philadelphia, PA
Molecular Basis of T-Cell Exhaustion

Stanley Riddell, MD
Fred Hutchinson Cancer Research Center
Seattle, WA
Selecting T-Cell Subsets for Adoptive T-Cell Therapy to Optimize Potency and Persistence

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Scientific Committee on Iron and Heme

Heme, Not Just for Globins

Sessions Offered Twice:
Saturday, December 7, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (271-273)
Sunday, December 8, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (271-273)

This scientific session will focus on the biochemistry of heme biosynthesis, the cell biology of heme transporters, and human diseases resulting from defects in heme synthesis and its transport to various subcellular compartments. The role of heme in various biological processes, besides its well-known role in hemoglobin production, will be reviewed. Recent advances in the identification of novel transporters of heme will be discussed in reference to the cell biology of erythropoiesis and iron/heme homeostasis. Disorders of heme production and transport will be reviewed.

Dr. Iqbal Hamza will present an overview of the role of heme in various biological processes, its biochemical synthesis, and the recent identification of novel heme transporters and their functional implications. Genetic model systems that have provided insight into the identification and functional characterization of these novel heme transporters will be presented, including the effects on iron/heme homeostasis.

Dr. Miguel Soares will discuss the pro-apoptotic signaling cascades initiated by heme release during severe systemic infections. These signaling processes controlling the metabolic adaptation to cellular iron overload will be discussed.

Dr. Hervé Puy will discuss the clinical disorders resulting from defects in the production of heme and its transport. Interventional therapies for these heme-related blood disorders will be reviewed.

Chair:

Barry H. Paw, MD, PhD
Brigham and Women's Hospital, Harvard Medical School
Boston, MA

Speakers:

Iqbal Hamza, PhD
University of Maryland
College Park, MD
Hematology from the Ground Up: Lessons from C. elegans

Miguel Soares, PhD
Instituto Gulbenkian de Ciencia
Oeiras, Portugal
Targeting Iron/Heme in Infectious Disease

Hervé Puy Sr., MD, PhD
INSERM U773, Université Paris Diderot
Paris, France
Heme-Related Blood Disorders

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Scientific Committee on Lymphoid Neoplasia

Targeting Apoptosis in Lymphoid Malignancies

Sessions Offered Twice:
Saturday, December 7, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (293-296)
Sunday, December 8, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (293-296)

This session will describe the mechanisms of programmed cell death, their deregulation in lymphoid cancers, and the development of new clinical approaches. Recent discoveries have provided new insights into the mechanisms of programmed cell death. Our understanding of how these processes are corrupted in cancer development is expanding rapidly and leading to new opportunities for therapeutic intervention.

Dr. Douglas Green will describe the current understanding of programmed cell death and highlight recently defined cell death mechanisms that cells employ in response to inflammatory, metabolic, and nutritional derangements.

Dr. Andreas Strasser will present work from mouse models of lymphoid malignancies and define the role of specific cell death proteins in the maintenance of lymphoid malignancies that set the stage for clinical translation.

Dr. Anthony Letai will present recent insights from translational studies that demonstrate a relationship between the activity of cell death mechanisms and the ability to predict response to cytotoxic chemotherapy. He will also update recent clinical studies that assess small molecules targeting antiapoptotic mechanisms in lymphoid malignancies.

Chair:

Scott A. Armstrong, MD, PhD
Memorial Sloan-Kettering Cancer Center
New York, NY

Speakers:

Douglas R. Green, PhD
St. Jude Children's Research Hospital
Memphis, TN
Matters of Life and Death: Give and Take in the Bcl-2 Family

Andreas Strasser, PhD
The Walter and Eliza Hall Institute Of Medical Research
Melbourne, Australia
Role of Bcl-2 Family in Lymphoid Malignancies

Anthony Letai, MD, PhD
Dana Farber Cancer Institute
Boston, MA
Therapeutic Targeting of the Bcl2 Family

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Scientific Committee on Myeloid Biology

The Molecular Biology of Congenital Neutropenia

Sessions Offered Twice:
Saturday, December 7, 2013
2:00 PM - 3:30 PM

Ernest N. Morial Convention Center (271-273)
Sunday, December 8, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (243-245)

Neutrophils are an essential component of the innate immune response and are a major contributor to inflammation. Consequently, neutrophil homeostasis is tightly regulated. This is achieved by balancing neutrophil production, release from the bone marrow, and senescence. Genetic alterations that impair neutrophil production or release result in severe congenital neutropenia (CN). CN is complicated by recurrent life-threatening opportunistic bacterial infections and increased incidence of acute myeloid leukemia (AML). To alleviate neutropenia, CN patients are routinely treated with granulocyte colony-stimulating factor (G-CSF), a major regulator of granulopoiesis that induces proliferation and differentiation of hematopoietic stem cells toward granulocytes and monocytes. Transformation of CN to AML is associated with the acquisition of somatic mutations in the gene encoding the G-CSF receptor (CSF3R).

Dr. Daniel Link will review the chemokine signals that regulate neutrophil trafficking from the bone marrow and the congenital neutropenias associated with alterations in this pathway.

Dr. Ivo Touw will discuss the consequences of CSF3R mutations on G-CSF responses and the leukemic evolution of CN. Development of CSF3R mutations is followed by the acquisition of additional genetic defects in CSF3R mutant clones over time.

Dr. Julia Skokowa will describe signaling systems that operate downstream of G-CSFR and have implications for this process, including activation of the WNT pathway, SIRT deacetylation, and activation of the nuclear transport protein HCL S1.

Chair:

Elizabeth Eklund, MD
Northwestern University
Chicago, IL

Speakers:

Daniel Link, MD
Washington University School of Medicine
Saint Louis, MO
Mechanisms of Neutrophil Release from the Bone Marrow

Ivo P. Touw, PhD
Erasmus Medical Center
Rotterdam, Netherlands
Malignant Transformation in Congenital Neutropenia

Julia Skokowa, Prof. Dr. med.
Hannover Medical School
Hannover, Germany
Aberrant G-CSFR Signaling in Congenital Neutropenia

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Scientific Committee on Myeloid Neoplasia

Molecular Complexity and Novel Targeting Therapies for Myeloid Neoplasms: The Future is Here!

Sessions Offered Twice:
Saturday, December 7, 2013
2:00 PM - 3:30 PM

Ernest N. Morial Convention Center (278-282)
Sunday, December 8, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (278-282)

Acute and chronic myeloid neoplasms have been well characterized for cytogenetic and molecular aberrations and this knowledge has been used for the improvement of disease classifications, outcome risk stratification, and treatment guidance. Emerging genomic sequencing technologies have provided insight into the landscape of genomic and epigenetic alterations that occur in these myeloid diseases and have led to the subsequent formulation of novel mechanistic hypotheses, the identification of previously unrecognized molecular subsets of patients, and the design of new molecularly-targeting therapies. However, the discovered molecular complexity of the myeloid neoplasms has also emphasized the challenges that can be encountered in devising targeting therapies that are specific for distinct molecular aberrations as well as minimally toxic and highly effective. This session will be devoted to the review of the molecular targeting approaches that are currently available for patients with myeloid neoplasms and to the discussion of the novel emerging therapies.

Dr. Michael Deininger will review the state of the art of tyrosine kinase inhibitors in chronic myeloid leukemia, the challenge of broadening their application to other acute and chronic myeloid neoplasms, and the utility of emerging novel pharmacologic strategies that interfere with leukemogenic kinase signaling.

Dr. Craig Thompson will review recent discoveries regarding the biology and clinical impact of emerging mutations in epigenetic modifiers and how they can be effectively targeted in myeloid neoplasms with new compounds currently being tested in early clinical trials.

Dr. Guy Sauvageau will review the role of leukemia-initiating cells in the mechanisms of chemoresistance of myeloid neoplasms, and how newly devised high-throughput strategies can be utilized for the screening of novel, effective compounds targeting this highly resistant disease niche.

Chair:

Guido Marcucci, MD
The Ohio State University
Columbus, OH

Speakers:

Michael Deininger, MD, PhD
The University of Utah
Salt Lake City, UT
Targeting Tyrosine Kinase Receptors

Craig B. Thompson, MD
Memorial Sloan-Kettering Cancer Center
New York, NY
Targeting Metabolic Inputs into Epigenetic Regulations of Acute Leukemia

Guy Sauvageau, MD, PhD
Maisonneuve-Rosemont Hospital, Université de Montréal
Montréal, QC, Canada
Novel Targeting Strategies for Leukemia-Initiating Cells in Myeloid Neoplasms

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Scientific Committee on Platelets

Platelets and Cancer

Sessions Offered Twice:
Saturday, December 7, 2013
2:00 PM - 3:30 PM

Ernest N. Morial Convention Center (343-345)
Sunday, December 8, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (353-355)

Animal studies published 45 years ago showed that thrombocytopenia protected against tumor cell metastases, but mechanisms of this effect and its relevance to the biology of human malignancies has been uncertain. Research is now beginning to elucidate specific mechanisms by which platelets affect the biology of cancer, suggesting areas of clinical translation applicable to a broad spectrum of human malignancies. The purpose of this session is to present new data about how platelets influence the growth and spread of cancer and how recent discoveries about platelet/cancer interactions might be applied to clinical therapeutics.

Dr. Chris Holmes will review the essential concepts of oncogenesis and cancer spread, provide an overview of the current state of the emerging field of platelets and cancer, and provide a framework for presenting the state-of-the-art work of the session.

Dr. Richard Hynes will discuss how platelets promote cancer cell metastases, including prometastatic changes in solid tumor cell phenotype, blood stream invasion, vascular transit, and microvascular arrest followed by tumor cell adhesion and extravasation at distant sites.

Dr. Tatiana Byzova will discuss how platelets actively modify the tumor microenvironment to support the growth of metastatic foci, emphasizing the interaction between platelets and the skeleton in the spread of prostate cancer.

Dr. Anil Sood will discuss the phenomenon of cancer-associated thrombocytosis, focusing on its cause and consequences, and will interpret data describing mechanisms by which thrombocytosis promotes the growth and spread of ovarian cancer.

Chair:

Michael H. Kroll, MD
The University of Texas MD Anderson Cancer Center
Houston, TX

Speakers:

Chris Holmes, MD, PhD
University of Vermont
Burlington, VT
Tumor Biology and our Current Understanding of the Role of Platelets: An Overview

Richard O. Hynes, PhD
Howard Hughes Medical Institute, Massachusetts Institute of Technology
Cambridge, MA
Platelets, Tumor Cell Invasiveness, and Metastasis

Tatiana V. Byzova, PhD
Cleveland Clinic
Cleveland, OH
Platelets and the Tumor Cell Microenvironment

Anil Sood, MD
The University of Texas MD Anderson Cancer Center
Houston, TX
Causes and Consequences of Cancer-Associated Thrombocytosis

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Scientific Committee on Red Cell Biology

From Red Cell Biology to Principles of Cell Regulation

Sessions Offered Twice:
Saturday, December 7, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (265-268)
Saturday, December 7, 2013
2:00 PM - 3:30 PM

Ernest N. Morial Convention Center (265-268)

This session will focus on the use of red cells to elucidate principles of cell regulation relevant to diverse aspects of hematology. In recent years, genomic data have been accrued at a rate that severely challenges existing analytical methodologies, and negotiating complex datasets can be daunting. The presentations will illustrate cutting-edge genomic and mechanistic analyses to dissect problems in chromosome biology, erythroid cell maturation, and hemoglobin switching that have considerable importance from fundamental and clinical perspectives.

Dr. John Stamatoyannopoulos will discuss the ENCODE project and the development of principles to understand chromosome biology and gene regulation.

Dr. Merav Socolovsky will describe epigenetic and cell cycle regulatory mechanisms governing erythroid cell maturation.

Dr. Stuart Orkin will present recent insights into transcriptional networks that control hemoglobin switching.

Chair:

Emery Bresnick, PhD
University of Wisconsin School of Medicine and Public Health
Madison, WI

Speakers:

John Stamatoyannopoulos, MD
University of Washington
Seattle, WA
Gene Regulation: A Genomic Perspective

Merav Socolovsky, MBBS, PhD
University of Massachusetts Medical School
Worcester, MA
Systems Biology and Epigenetic Mechanisms in Erythropoiesis

Stuart H. Orkin, MD
Howard Hughes Medical Institute
Cambridge, MA
Genetics and Epigenetics of Fetal Hemoglobin Control

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Scientific Committee on Stem Cells and Regenerative Medicine

The Stem Cell Epigenome

Sessions Offered Twice:
Saturday, December 7, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (243-245)
Saturday, December 7, 2013
4:00 PM - 5:30 PM

Ernest N. Morial Convention Center (243-245)

The hematopoietic system is a major paradigm for understanding how epigenetic cues guide the differentiation of stem cells into distinct functional lineages. The direct chemical modification of DNA and chromatin-associated proteins is orchestrated by a complex network of enzymes, some of which are mutated or dysregulated in leukemia, myelodysplasia, lymphoma, and a host of other malignancies and developmental disorders. Defining the key molecular features of epigenetic regulation promises to inform efforts to model blood diseases, direct stem cell differentiation, and develop new therapeutic strategies. In this session, three leading scientists will present the latest insights into transcriptional regulation and chromatin control with particular attention to hematopoiesis and stem cell dynamics.

Dr. Richard Young, a leader in defining transcriptional regulatory mechanisms, will discuss his lab’s progress in mapping the regulatory circuitry that controls cell state and differentiation in embryonic stem cells and differentiated cells.

Dr. Andrew Feinberg pioneered the field of cancer epigenetics and has led efforts to develop and apply genome-scale epigenetic tools generally to common human disease. He will describe the role of epigenetic changes in normal development and reprogramming in induced pluripotent stem cells and cancer.

Dr. Leonard Zon, who has revealed many fundamental insights into blood development by exploiting the zebrafish genetic system, will discuss genetic and chemical screens that have revealed new chromatin targets critical for hematopoietic stem cell development, function, and malignancy.

Chair:

George Q. Daley, MD, PhD
Boston Children's Hospital
Boston, MA

Speakers:

Richard A. Young, PhD
Whitehead Institute for Biomedical Research, Massachusetts Institute of Technology
Cambridge, MA
Control of the Stem Cell Gene Expression Program

Andrew P. Feinberg, MD
The Johns Hopkins University School of Medicine
Baltimore, MD
Epigenetics of Hematopoiesis, Stem Cell Reprogramming, and Cancer

Leonard I. Zon, MD
Howard Hughes Medical Institute, Boston Children's Hospital, Harvard Medical School
Boston, MA
The Role of Chromatin Factors in Hematopoietic Development

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Scientific Committee on Thrombosis & Vascular Biology

Vessel Wall, Atherosclerosis, and Arterial Thrombosis

Sessions Offered Twice:
Saturday, December 7, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (265-268)
Sunday, December 8, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (265-268)

Atherosclerosis is a chronic degenerative process of the arterial wall characterized by the subendothelial accumulation of lipids in critical areas of the arterial tree, affecting millions of men and women worldwide. Alterations of lipid metabolism accompanied by changes in carbohydrate metabolism with insulin resistance are key determinants of atherosclerotic plaque formation. In addition, it is now generally recognized that inflammation, the second key hallmark of atherosclerosis, plays a major role in the onset of the disease, and most importantly, in causing plaque disruption and instability that may be followed by local thrombosis. This session will highlight three major topics representative of ongoing research efforts aimed at achieving a detailed understanding of the pathogenic molecular mechanisms underlying atherosclerosis and its acute thrombotic complications.

Dr. Meinrad Gawaz will discuss the role of platelet interaction with leukocytes in atherothrombosis, with emphasis on the reciprocal influence of leukocyte-platelet crosstalk in the atherosclerotic plaque environment. Platelets are an important link between inflammation, thrombosis, and atherogenesis. Leukocyte, as well as platelet activation, influence the balance of inflammatory procoagulant and anticoagulant pathways; and increased thrombin production is likely a key determinant of both atherosclerotic plaque progression and acute thrombotic arterial occlusion.

Dr. Gwendalyn Randolph will discuss the role of monocyte trafficking in the onset and development of atherosclerotic lesions. Her studies are based on earlier literature concluding that the removal of monocyte-derived cells from sites of inflammation requires emigration to lymph nodes through lymphatic vessels. More recent experimental work in the context of atherosclerosis and acute inflammation indicates that the process of emigration plays a quantitatively minor role in the removal of macrophages. Instead, macrophage burden is primarily regulated by monocyte recruitment and local apoptosis. Dr. Randolph’s laboratory is seeking to understand how changes in cholesterol metabolism in the artery wall regulate monocyte recruitment to plaques and how this signal can be turned off to promote disease regression.

Dr. Daniel Simon will concentrate on research aimed at determining genetic predictors of myocardial infarction using transcriptional profiling of platelets. This functional genomic approach has identified new targets that regulate vascular inflammation and thrombosis, including the elucidation of a novel pathway of thrombosis that does not affect bleeding time or hemostasis. Therefore, therapeutic inhibition of this pathway may achieve antithrombotic effects with a reduced risk of bleeding complications.

Chair:

Zaverio Marcello Ruggeri, MD
The Scripps Research Institute
La Jolla, CA

Speakers:

Meinrad Gawaz, MD
Eberhard Karls-Universität
Tübingen, Germany
Leukocyte Adhesion and Activation in Atherothrombosis

Gwendalyn J. Randolph, PhD
Washington University School of Medicine
St. Louis, MO
Monocyte Trafficking, Inflammation, and Atherosclerosis

Daniel I. Simon, MD
Case Western Reserve University School of Medicine
Cleveland, OH
Platelet Transcriptional Profiling and Atherothrombosis

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Scientific Committee on Transfusion Medicine

Transfusion Medicine for the Pregnant Mother, Fetus, and Neonate: From Bedside to Bench

Sessions Offered Twice:
Saturday, December 7, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (220-222)
Sunday, December 8, 2013
7:30 AM - 9:00 AM

Ernest N. Morial Convention Center (220-222)

This session will focus on recent developments of some of the transfusion medicine issues that affect the health of the pregnant mother, her fetus, and the resulting newborn child. Specifically, speakers will describe recent results from animal model experiments that elucidate the development of maternal alloimmunity against red blood cell and platelet alloantigens and the mechanisms and consequences of immune-mediated blood cell destruction in the fetus and neonate. In addition, speakers will discuss how to apply this new knowledge in the clinic by describing cutting-edge molecular methods for identifying and preventing high-risk human pregnancies.

Dr. Jeanne Hendrickson will describe new mouse models for studying the mechanisms underlying the immunogenicity of red blood cell alloantigens. She will also describe new mouse models for studying the pathophysiology, treatment, and prevention of hemolytic disease of the fetus and newborn (HDFN).

Dr. Heyu Ni will describe new mouse models for studying fetal and neonatal alloimmune thrombocytopenia (FNAIT). He will also discuss how these models allow rigorous testing of hypotheses addressing the pathophysiology and therapies of this disorder.

Dr. C. Ellen van der Schoot will describe new methods of prenatal fetal DNA testing for predicting which human pregnancies are at risk for HDFN and/or FNAIT. She will also discuss how these approaches can be used clinically to determine which women should receive Rh immune globulin to prevent Rh disease.

Chair:

Steven L. Spitalnik, MD
Columbia University Medical Center
New York, NY

Speakers:

Jeanne E. Hendrickson, MD
Yale University
New Haven, CT
Red Blood Cell Alloimmunization and Hemolytic Disease of the Fetus and Newborn: New Approaches Using Animal Models

Heyu Ni, MD, PhD
Keenan Research Center, Li Ka Shing Knowledge Institute, St. Michael’s Hospital, Department of Laboratory Medicine and Pathobiology, and Toronto Platelet Immunobiology Group, University of Toronto
Toronto, ON, Canada
Fetal and Neonatal Alloimmune Thrombocytopenia: Lessons Learned from Animal Models

C. Ellen van der Schoot, MD, PhD
Sanquin Research Institute
Amsterdam, Netherlands
Prenatal Fetal DNA Testing for Predicting HDFN, FNAIT, and RhIG Candidacy

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Scientific Committee on Transplantation Biology

Burgeoning Roles of B Cells in Transplant

Sessions Offered Twice:
Saturday, December 7, 2013
9:30 AM - 11:00 AM

Ernest N. Morial Convention Center (243-245)
Saturday, December 7, 2013
4:00 PM - 5:30 PM

Ernest N. Morial Convention Center (343-345)

This session will focus on the evidence delineating how B cells act as potential pathologic mediators or immune regulators in human diseases. Beyond their ability to produce antibodies and act as antigen-presenting cells in anti-microbial responses, B cells are now identified as vital contributors to immune pathology.

Dr. Ann Marshak-Rothstein will lay the foundation for active discussion by relaying how B-cell tolerance is maintained. She will present data behind the current understanding of important mechanisms by which autoantibody-antigen complexes trigger receptor signaling and promote autoimmunity.

Dr. Thomas Tedder will present evidence supporting the role of B cells as negative regulators of graft rejection. He will also outline the mechanisms by which interleukin 10-producing B cells regulate T cells to maintain immune tolerance.

Dr. Jerome Ritz will summarize work demonstrating the relevance of B-cell and antibody responses to the development of chronic graft-versus-host disease (GVHD) and graft-versus-leukemia (GVL) responses. Modulation of B-cell immunity in patients with GVHD and GVL may improve outcomes for patients undergoing allogeneic hematopoietic stem cell transplantation.

Chair:

Stefanie Sarantopoulos, MD, PhD
The University of North Carolina at Chapel Hill
Chapel Hill, NC

Speakers:

Ann Marshak-Rothstein, PhD
University of Massachusetts
Worcester, MA
From B-Cell Tolerance to Autoimmunity

Thomas F. Tedder, PhD
Duke University
Durham, NC
Regulatory B Cells (B10 Cells) in Human Disease

Jerome Ritz, MD
Dana-Farber Cancer Institute
Boston, MA
Role of B Cells in Graft-Versus-Leukemia and Graft-Versus-Host Disease

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