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ASH Annual Meeting and Exposition

Scientific Workshops

The 2024 Scientific Workshops will take place on Friday, December 6, and will also be streamed on the ASH annual meeting platform for virtual participants. ASH will host 10 workshops this year, featuring interactive discussions covering the latest scientific developments in a range of hematologic topics.

All are welcome to attend each workshop. There is no additional fee, but ASH annual meeting registration is required.

Don't Miss Your Chance to Present at a Scientific Workshop

Lend your speaking abilities and get involved in one of the most anticipated events of the annual meeting! If you are interested, each workshop has specific instructions on how you can indicate interest in speaking.

Friday, December 6, 2024, 2:00 p.m. - 5:00 p.m.

Co-Chairs:

Megan Weivoda , PhD
Mayo Clinic
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Kah Poh Loh , MD, MBBCH BAO, MS
University of Rochester Medical Center
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Hematologic disorders disproportionately affect older adults; however, our understanding of the aging mechanisms that drive hematologic dysfunction remains limited. This workshop will interrogate the roles for cell intrinsic (epigenetics, proteostasis) vs. extrinsic (microenvironment, immune function) factors on aging in hematologic diseases. The workshop which aims to be interactive and fostering debate-style discourse, will also address the translation of geroscience biomarkers that can be used to understand and treat patients across the aging continuum.

Target Audience:

Laboratory-based scientists, clinical/translational investigators, and early career investigators with an interest in aging and hematology (malignant and classical).

Objectives:

  • Foster interaction and engagement among aging scientists and investigators conducting research at the interface of hematology and aging.
  • Provide a forum within ASH to advance discussions on novel aging science related to hematologic disorders and implications for translation to clinical practice.
  • Provide opportunities for early career investigators to participate and interact with established leaders in the field of hematology and aging.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:03Ìý±è.³¾.
San Diego Convention Center , Room 7

Moderator:

Megan Weivoda , PhD
Mayo Clinic
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Speaker:

Megan Weivoda , PhD
Mayo Clinic
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Opening Remarks

Cell Intrinsic Mechanisms Driving Hematologic Aging

2:03Ìý±è.³¾.Ìý-Ìý2:45Ìý±è.³¾.
San Diego Convention Center , Room 7

Moderators:

Tanya M. Wildes , MD,MSc
University of Nebraska Medical Center
Omaha,  NE

Francesca Cottini , MD
The Ohio State University
Columbus,  OH

Speakers:

Tanya M. Wildes , MD,MSc
University of Nebraska Medical Center
Omaha,  NE
Moderator Introduction and Session 1 Initial Audience Voting

Kristina Kirschner , PhD
Mayo Clinic
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Clonal Mutations Drive Hematopoietic Stem and Progenitor Cell Fitness

Fanny Jiahua Jiahua Zhou , BS,MS
University of California San Diego
San Diego,  CA
Defective Misfolded Protein Response and Impaired Proteostasis Drive Hematopoietic Stem Cell Aging

Enrico Derenzini , MD, PhD
European Institute of Oncology
Milan,  Italy
DNA Damage Signaling and Telomere Dysfunction in B Cell Malignancies

Adapting to the Aged Bone Marrow Microenvironment (BMME)

2:46Ìý±è.³¾.Ìý-Ìý3:28Ìý±è.³¾.
San Diego Convention Center , Room 7

Moderators:

Heidi Klepin , MD
Wake Forest University School of Medicine
Winston Salem,  NC

Patrizia Mondello , MD,PhD,MSc
Mayo Clinic
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Speakers:

Patrizia Mondello , MD,PhD,MSc
Mayo Clinic
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Moderator Introduction and Session 2 Initial Audience Voting

Marco De Dominici , PhD
University of Colorado
Aurora,  CO
Aging and Inflammation Select for Hematopoietic Stem Cell Oncogenic Mutations

Madison Doolittle , PhD
University of Connecticut
Farmington,  CT
Senescent in the Aging Bone Marrow Microenvironment is Driven by Mesenchymal Lineage Cells

Bo Shen , PhD
Tsinghua University
Beijing,  CHN
Mesenchymal Stromal Cells-Nerve Cross Talk Regulates Changes in Hematopoiesis with Aging

The Contribution of Immune System to Hematologic Aging

3:30Ìý±è.³¾.Ìý-Ìý4:12Ìý±è.³¾.
San Diego Convention Center , Room 7

Moderators:

Rebecca Olin , MD
University of California San Francisco
San Francisco,  CA

Othman S Akhtar , MD
Medical College of Wisconsin
Milwaukee,  WI

Speakers:

Othman S Akhtar , MD
Moffit Cancer Center
Tampa,  FL
Moderator Introduction and Session 3 Initial Audience Voting

Emily R Quarato , PhD
University of Rochester
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Innate Immune Failure Activates Efferocytic Activity in Bone Marrow Stromal Cells That Drives Hematological Aging

Gabriel Alvares Borges , PhD,MSc
Mayo Clinic
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Inflamm-Aging Impairs Anti-Tumor Immune Responses in Clonal Hematologic Disease

Sheng Li , PhD
University of California San Diego
San Diego,  CA
Altered Immune Environment in Clonal Hematopoiesis

Translational Geroscience Biomarkers in Hematologic Malignancies

4:15Ìý±è.³¾.Ìý-Ìý4:57Ìý±è.³¾.
San Diego Convention Center , Room 7

Moderators:

Andrew Artz , MD,MS
City of Hope National Medical Center
Duarte,  CA

Mazie Tsang , MD
Mayo Clinic
Phoenix,  AZ

Speakers:

Andrew Artz , MD,MS
City of Hope National Medical Center
Duarte,  CA
Moderator Introduction and Session 4 Initial Audience Voting

Ashley Rosko , MD
The Ohio State University
Columbus,  OH
Biomarkers of Aging: Chasing the Holy Grail in Hematologic Malignancies?

Nadine Abdallah , MD
Mayo Clinic
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Clinical Frailty Assessments and Biomarkers of Biological Aging

Annalynn M Williams , PhD,MS
University of Rochester
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Promising Biomarkers of Premature Cognitive Aging in Survivors of Adolescent and Young Adult Hodgkin Lymphoma

Closing Remarks

4:57Ìý±è.³¾.Ìý-Ìý5:00Ìý±è.³¾.
San Diego Convention Center , Room 7

Moderator:

Kah Poh Loh , MD, MBBCH BAO, MS
University of Rochester
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Speaker:

Kah Poh Loh , MD, MBBCH BAO, MS
University of Rochester
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Closing Remarks

Friday, December 6, 2024, 2:00 p.m. - 5:00 p.m.

Co-Chairs:

Umut Gurkan , PhD
Case Western Reserve University
Cleveland,  OH

O. Berk Usta , PhD
Massachusetts General Hospital
Boston,  MA

Red Blood Cell (RBCs) transfusion, afforded by a global network of blood storage and banking, is one of the most common and lifesaving medical treatments and the oldest cell therapy. The quality assessment for stored RBC products and their subsequent allocation currently follows rudimentary approaches and should benefit from novel technologies. Given the rapid and astounding advances in -omics, machine learning, and lab-on-a-chip technologies in the last decade, their standalone and integrated use have been suggested in multiple recent articles by many in the field.

This workshop will feature themes and experts in a wide range of areas including but not limited to biopreservation, blood storage and transfusion, blood banking, blood quality assessment, lab-on-a-chip and microfluidic technologies, multi-omics technologies, ethics and public health law, with a focus on advancing technologies both for storage and quality assessment of blood products.

Target Audience:

Investigators and personnel with interest in blood banking, bio-preservation, transfusion medicine, -omics (i.e, metabolomics, proteomics, lipidomic), large scale data, machine learning, bioengineering, medical devices, cell and gene therapy, ethics, regulatory affairs; and ultimately patients who are recipients of blood products.

Objectives:

  • Bring together stakeholders and experts from diverse backgrounds who have pioneered advances to improve RBC quality assessment.
  • Engage a diverse audience on this important topic.
  • Spur discussion, innovation, and meaningful progress towards a new era of precision transfusion medicine of stored RBC products.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:10Ìý±è.³¾.
San Diego Convention Center , Room 3

Moderator:

Umut Gurkan , PhD
Case Western Reserve University
Cleveland,  OH

Speaker:

Umut Gurkan , PhD
Case Western Reserve University
Cleveland,  OH
Opening Remarks

Challenges in Red Blood Cell Quality Testing

2:10Ìý±è.³¾.Ìý-Ìý3:25Ìý±è.³¾.
San Diego Convention Center , Room 3

Moderator:

Umut Gurkan , PhD
Case Western Reserve University
Cleveland,  OH

Speakers:

Umut Gurkan , PhD
Case Western Reserve University
Cleveland,  OH
Moderator Introduction

Jose ANTONIO Cancelas , MD
Dana-Farber Cancer Institute
Boston,  MA
The US Blood Banking Perspective on Stored Red Blood Cell Quality and Its Assessment

Jason Acker , MBA PHD
University of Alberta
Edmonton,  AB, CAN
Canada Blood Banking Perspective on Stored Red Blood Cell Quality and Its Assessment

Julia Xu , MD,MS
Division of Classical Hematology, Department of Medicine, University of Pittsburgh
Pittsburgh,  PA
Global Burden of Red Blood Cell Disorders and the Need for New Assessment Technologies for Blood Products

Vivien Sheehan , MD,PhD
Emory University School of Medicine
Atlanta,  GA
The Need for Red Cell Function Testing: From Clinical Trials to Patient Care

Novel Technologies in Red Blood Cell Quality Assessment

3:30Ìý±è.³¾.Ìý-Ìý4:45Ìý±è.³¾.
San Diego Convention Center , Room 3

Moderator:

O. Berk Usta , PhD
Massachusetts General Hospital
Boston,  MA

Speakers:

O. Berk Usta , PhD
Massachusetts General Hospital
Boston,  MA
Moderator Introduction

Travis Nemkov , PhD
University of Colorado Anschutz Medical Campus
Aurora,  CO
Multi-Omics Technologies for Red Blood Cell Quality Assessment

Amin T. Turki , MD
University Hospital Essen
Essen,  Germany
Leveraging Large Scale Data and AI for Red Blood Cell Quality Assessment and Precision Transfusion Medicine

Lars Kaestner , PhD
Saarland University
Homburg,  Saar, Germany
European Perspective and in Vitro Diagnostics on Stored Red Blood Cell Quality and Its Assessment

Allan Doctor , MD
University of Maryland
Baltimore,  MD
Defining Oxygen Transport Potency for Red Bed Cells and Bio-Synthetic Alternatives

Closing Remarks

4:50Ìý±è.³¾.Ìý-Ìý5:00Ìý±è.³¾.
San Diego Convention Center , Room 3

Moderator:

O. Berk Usta , PhD
Massachusetts General Hospital
Boston,  MA

Speaker:

O. Berk Usta , PhD
Massachusetts General Hospital
Boston,  MA
Closing Remarks

Friday, December 6, 2024, 2:00 p.m. - 5:00 p.m.

Co-Chairs:

Michael R. Savona , MD
Vanderbilt University Medical Center
Nashville,  TN

Lambert Busque , MD
University of Montreal
Montreal,  QC, Canada

Clonal hematopoiesis of indeterminate potential (CHIP) is linked to various disease states, and is heterogeneous with respect to its impact, based on specific mutations, variant allele frequency of the mutations, and germline context. As CHIP may occur in any of dozens of genes typically found aberrant in myeloid neoplasms, it follows that the mechanism of pathogenesis varies based on the CHIP that occurs, as will, accordingly, the potential treatment opportunities.

The first decade after the discovery of CHIP was dedicated to describing this profound phenomenon in large cohorts, and its relation to different diseases. This has led to considerable insight into the molecular epidemiology of CHIP. Now, the onus is to understand how CHIP leads to disease. Overtures toward understanding how different CHIP mutants influence pathobiology have been made, and there is hope in addressing CHIP-associated effects, largely via addressing specific pro-inflammatory signaling patterns upregulated in CHIP-mutation-specific states.

This workshop will highlight novel mechanistic insights into CHIP-associated pathobiology including the challenge of translating these insights to novel secondary prevention clinical trials to prevent CHIP associated disease from occurring.

Target Audience:

Myeloid biologists, vascular/coagulopathy researchers, molecular geneticists and clinical researchers, particularly those who design clinical trials.

Objectives:

  • Communicate recent discoveries and highlight the disparate means by which CHIP can initiate or accelerate disease.
  • Illustrate proof-of-principle therapeutic interventions in the lab to initiate a discussion on potential therapeutic opportunities in the clinic.
  • Engage the audience in a thoughtful discussion around the expectations from regulatory agencies for a secondary prevention study in healthy persons. The differences between this and a treatment trial in patients already stricken with a lethal hematologic malignancy.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:05Ìý±è.³¾.
San Diego Convention Center , Room 8

Moderator:

Lambert Busque , MD
Institut universitaire d'hématologie-oncologie et de thérapie cellulaire (IHOT)
Montréal,  QC, Canada

Speaker:

Lambert Busque , MD
Maisonneuve-Rosemont Hospital
Montreal,  QC, Canada
Opening Remarks

Germline Predisposition for Clonal Hematopoiesis

2:05Ìý±è.³¾.Ìý-Ìý2:45Ìý±è.³¾.
San Diego Convention Center , Room 8

Moderator:

Kelly Bolton , MD, PhD
Washington University School of Medicine
St. Louis,  MO

Speakers:

Kelly Bolton , MD, PhD
Washington University School of Medicine
St. Louis,  MO
Moderator Introduction

Kelly Bolton , MD, PhD
Washington University School of Medicine
St. Louis,  MO
Introduction of Germline Context for Clonal Hematopoiesis

Alexander G. Bick , MD,PhD
Vanderbilt University School of Medicine
Nashville,  TN
TCL1A to mCA's

Clonal Hematopoiesis Lesions and Gene Editing

2:45Ìý±è.³¾.Ìý-Ìý3:50Ìý±è.³¾.
San Diego Convention Center , Room 8

Moderator:

Michael R. Savona , MD
Vanderbilt University Medical Center
Nashville,  TN

Speakers:

Michael R. Savona , MD
Vanderbilt University Medical Center
Nashville,  TN
Moderator Introduction

Ravi Majeti , MD, PhD
Stanford University
Stanford,  CA
Gene Editing to Model Clonal Hematopoiesis

Esther A. Obeng , MD,PhD
St. Jude Children's Research Hospital
Memphis,  TN
Modeling Deleterious Clonal Hematopoiesis Mutations In Vivo

Michael R. Savona , MD
Vanderbilt University Medical Center
Nashville,  TN
Using Intraallelic Gene Conversion to Reduce Variant Allele Frequencies of Deleterious CH Mutations

Clinical Trial Approaches in Clonal Hematopoiesis

3:50Ìý±è.³¾.Ìý-Ìý4:55Ìý±è.³¾.
San Diego Convention Center , Room 8

Moderator:

Mrinal M. Patnaik , MD, MBBS
Mayo Clinic
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Speakers:

Mrinal M. Patnaik , MD, MBBS
Mayo Clinic
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Moderator Introduction

Luisa Cimmino , PhD
University of Miami-Miller School of Medicine
Miami,  FL
Targeting TET2 in Clonal Hematopoiesis

Amit Verma , MD
Albert Einstein College of Medicine
Bronx,  NY
Addressing Inflammaging As Secondary Prevention in Clonal Hematopoiesis

David P. Steensma , MD
Ajax Therapeutics
Cambridge,  MA
Serendipity and Lessons Learned about Clonal Hematopoiesis in the Cantos Study

Closing Remarks

4:55Ìý±è.³¾.Ìý-Ìý5:00Ìý±è.³¾.
San Diego Convention Center , Room 8

Moderator:

Michael R. Savona , MD
Vanderbilt University Medical Center
Nashville,  TN

Speaker:

Michael R. Savona , MD
Vanderbilt University Medical Center
Nashville,  TN
Closing Remarks

Friday, December 6, 2024, 2:00 p.m. - 5:00 p.m.

Co-Chairs:

Uma Borate , MD
The Ohio State University
Columbus,  OH

Stephanie Valerie Seremetis , MD
Novo Nordisk
Plainsboro,  NC

Decentralized Clinical Trials (DCTs) are of great interest to the hematology community (classical and malignant hematology practice providers), pharmaceutical sponsors and regulatory agencies because these studies have enormous potential to remove patient access barriers to clinical trials and enroll more 'real world' patients. Despite this interest, there are many outstanding questions about the practical aspects of performing these trials: from trial design to data integrity and result analysis challenges.

This workshop will address these questions providing perspectives from academia, industry, and regulatory stakeholders. In the advent of artificial intelligence (AI), there is a unique opportunity to understand how to leverage it in all aspects of DCTs to ensure real world patient participation. To that end, this workshop will also highlight possible applications of AI to DCT design, implementation, and execution.

Target Audience:

Translational investigators, Clinical investigators (including those in the practice setting), advance practice providers, industry collaborators/sponsors, clinical trial staff (research coordinators, data managers), regulators, biostatisticians and early career investigators.

Objectives:

  • Bring together all key stakeholders -clinical investigators, industry, regulatory agencies ad patient advocates who share a common interest in the conduct of successful decentralized clinical trials.
  • Learn about the operational opportunities and challenges in conducting DCTs including leveraging AI tools for these studies.
  • Spark new multidisciplinary collaborations across different stakeholder groups that continue long after the workshop.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:05Ìý±è.³¾.
San Diego Convention Center , Room 4

Moderators:

Uma Borate , MD
The Ohio State University
Columbus,  OH

Stephanie Valerie Seremetis , MD
Novo Nordisk
Plainsboro,  NC

Speakers:

Uma Borate , MD
The Ohio State University
Columbus,  OH
Opening Remarks

Stephanie Valerie Seremetis , MD
Novo Nordisk
Plainsboro,  NC
Opening Remarks

Trial Designs for Decentralized Clinical Trials

2:05Ìý±è.³¾.Ìý-Ìý2:52Ìý±è.³¾.
San Diego Convention Center , Room 4

Moderator:

Jean M Connors , MD
Brigham and Women's Hospital
Boston,  MA

Speakers:

Jean M Connors , MD
Brigham and Women's Hospital
Boston,  MA
Moderator Introduction

Jean M Connors , MD
Brigham and Women's Hospital
Boston,  MA
Are We Ready for Innovative Decentralized Clinical Trials in Hematology? Learnings from the COVID19 Pandemic

Andrew Srisuwananukorn , MD
The Ohio State University
Columbus,  OH
How Can AI Help in 'real World' Patient Selection?

Shirley D'Sa , MD(Res)
University College London Hospitals NHS Foundation Trust
London,  United Kingdom
Using Wearables for Patient Monitoring in Decentralized Clinical Trials- Ready for Prime Time?

Implementation of Decentralized Clinical Trials

3:04Ìý±è.³¾.Ìý-Ìý3:51Ìý±è.³¾.
San Diego Convention Center , Room 4

Moderator:

Uma Borate , MD
The Ohio State University
Columbus,  OH

Speakers:

Uma Borate , MD
The Ohio State University
Columbus,  OH
Moderator Introduction

Giulia Petrone , MD,MBBS
Washington University
Saint Louis,  MO
Innovative Decentralized Clinical Trial Designs

Irene Ghobrial , MD
Dana-Farber Cancer Institute
Boston,  MA
Challenges in Operationalizing Decentralized Clinical Trials: The Institutional Perspective

Ankit J. Kansagra , MD
University of Texas Southwestern Medical Center
Dallas,  TX
The Sponsor Perspective of Decentralized Clinical Trials - Pleasure or Pain?

Data Management and Analysis of Decentralized Clinical Trials

4:03Ìý±è.³¾.Ìý-Ìý4:55Ìý±è.³¾.
San Diego Convention Center , Room 4

Moderator:

Jiasheng Wang , MD
The Ohio State University
Columbus,  OH

Speakers:

Jiasheng Wang , MD
The Ohio State University
Columbus,  OH
Moderator Introduction

Jiasheng Wang , MD
The Ohio State University
Columbus,  OH
Data Collection and Monitoring in Decentralized Clinical Trials: Challenges and AI Solutions

Megan Othus , PhD
SWOG Statistics and Data Management Center
Seattle,  WA
What's New, What's Old? - Data Management and Analysis of Decentralized Clinical Trials

Kelly J Norsworthy , MD
Food and Drug Administration
Silver Spring,  MD
Decentralized Clinical Trial/ Pragmatic Trial Results and Practice Change: ‘Reality or Optimism’?

Closing Remarks

4:55Ìý±è.³¾.Ìý-Ìý5:00Ìý±è.³¾.
San Diego Convention Center , Room 4

Moderators:

Uma Borate , MD
The Ohio State University
Columbus,  OH

Stephanie Valerie Seremetis , MD
Novo Nordisk
Plainsboro,  NC

Speakers:

Uma Borate , MD
The Ohio State University
Columbus,  OH
Closing Remarks

Stephanie Valerie Seremetis , MD
Novo Nordisk
Plainsboro,  NC
Closing Remarks

Friday, December 6, 2024, 2:00 p.m. - 5:00 p.m.

Co-Chairs:

Nathan Singh , MD
Washington University School of Medicine
Saint Louis,  MO

Stefano Rivella , PhD
Children's Hospital of Philadelphia
Philadelphia,  PA

The facile application of gene editing technologies has fueled a revolution in the management of hematologic diseases. The two areas that have seen the most significant changes are hemoglobinopathies and blood cancers. The increasing use of gene edited therapies provides great promise for the future but also brings toxicities related to genome engineering. Understanding the biological basis of these toxicities is critical to managing patients receiving these therapies and designing next-generation products.

This workshop aims to bring together investigators with distinct and complementary expertise to discuss the biological implications of gene editing and accelerate progress by sharing lessons from each discipline. Specifically, this workshop will focus on emerging questions following the use of gene and cellular therapies for the treatment of hematologic (classical and malignant) diseases. Beginning with the recent application of these novel therapies, the program will focus on emerging toxicities, potential scientific and clinical roadblocks, and discussions of regulatory considerations.

Target Audience:

Basic scientists, translational researchers, clinical trialists and regulatory experts studying cell and gene therapies, developing novel gene editing platforms, and/or conducting adverse event reporting and monitoring.

Objectives:

  • Bring together two parallel groups of investigators who share the scientific goal of editing human genomes to treat blood disorders.
  • Promote discussions that share lessons and insights from one disease context to another.
  • Spark new multidisciplinary collaborations.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:03Ìý±è.³¾.
San Diego Convention Center , Room 31

Moderator:

Stefano Rivella , PhD
Children's Hospital of Philadelphia
Philadelphia,  PA

Understanding Immediate Toxicities of Current Gene and Cell Therapy Tools

2:03Ìý±è.³¾.Ìý-Ìý3:00Ìý±è.³¾.
San Diego Convention Center , Room 31

Moderators:

Marina Cavazzana , MD,PhD
Assistance Publique-Hôpitaux de Paris
Paris,  France

Saar Gill , MD, PhD
University of Pennsylvania
Philadelphia,  PA

Speakers:

Marina Cavazzana , MD,PhD
Assistance Publique-Hôpitaux de Paris
Paris,  France
Moderator Introduction (Classical Hematology Perspective)

Saar Gill , MD, PhD
University of Pennsylvania
Philadelphia,  PA
Moderator Introduction (Malignant Hematology Perspective)

Annarita Miccio , PhD
Imagine Institute
Paris,  France
Ex Vivo Genetic Modification and Hematopoietic Cell Transplantation

Marcela Maus , MD
Harvard Medical School
Lexington,  MA
Ex Vivo Genome Editing of Immune Cells

Janet L. Kwiatkowski , MD, MSCE
Children's Hospital of Philadelphia
Philadelphia,  PA
Hematopoietic Cell Transplantation Versus Gene Therapy for Hemoglobinopathies

Noelle Frey , MD, MS
University of Pennsylvania
Philadelphia,  PA
Hematopoietic Cell Transplantation Versus Gene Therapy for Blood Cancers

Emerging and Long-term Toxicities

3:15Ìý±è.³¾.Ìý-Ìý4:14Ìý±è.³¾.
San Diego Convention Center , Room 31

Moderators:

Jennifer N Brudno , MD
National Cancer Institute, National Institutes of Health
Bethesda,  MD

John Tisdale , MD
National Institutes of Health
Bethesda,  MD

Speakers:

John Tisdale , MD
National Institutes of Health
Bethesda,  MD
Moderator Introduction (Classical Hematology Perspective)

Jennifer N Brudno , MD
National Cancer Institute, National Institutes of Health
Bethesda,  MD
Moderator Introduction (Malignant Hematology Perspective)

Maria Rosa Lidonnici , PhD
SR-TIGET
Milan,  Italy
Clonal Hematopoiesis in Hemoglobinopathies

Marina Cavazzana , MD,PhD
Assistance Publique-Hôpitaux de Paris
Paris,  France
The Role of In?ammation in Sickle Cell Disease Pathology

Bruce Levine , PhD
University of Pennsylvania
Philadelphia,  PA
Consequences of Engineered E?ector Cell Therapies

Mark Hamilton , MD
Stanford University
Palo Alto,  CA
Damage to Engineered T Cell Genomes

How Do We Effectively Capture Adverse Events, Particularly Infections Post Hematopoietic Stem Cell Transplantation?

4:14Ìý±è.³¾.Ìý-Ìý4:53Ìý±è.³¾.
San Diego Convention Center , Room 31

Moderators:

Matthew Hsieh , MD
NIH-NHLBI-MCHB
Bethesda,  MD

Rayne Rouce , MD
Clinical Care Center-Texas Children's Hospital
Houston,  TX

Speakers:

Matthew Hsieh , MD
NIH-NHLBI-MCHB
Bethesda,  MD
Moderator Introduction (Classical Hematology Perspective)

Rayne Rouce , MD
Clinical Care Center-Texas Children's Hospital
Houston,  TX
Moderator Introduction (Malignant Hematology Perspective)

Adetola A. Kassim , MD, MBBS, MS
Vanderbilt University Medical Center
Nashville,  TN
Benign Diseases

Kathryn Cappell , MD,PhD
National Institutes of Health
Bethesda,  MD
Malignant Diseases

Closing Remarks

4:55Ìý±è.³¾.Ìý-Ìý5:00Ìý±è.³¾.
San Diego Convention Center , Room 31

Moderator:

Nathan Singh , MD
Washington University School of Medicine
Saint Louis,  MO

Speaker:

Nathan Singh , MD
Washington University School of Medicine
Saint Louis,  MO
Closing Remarks

Friday, December 6, 2024, 2:00 p.m. - 5:00 p.m.

Co-Chairs:

Lucy A. Godley , MD,PhD
Northwestern University
Chicago,  IL

Marcin Wlodarski , MD, PhD
St. Jude Children's Research Hospital
Memphis,  TN

Deleterious germline variants conferring susceptibility to hematopoietic malignancies (HMs) and bone marrow failure (BMF) are recognized now to occur much more commonly than previously assumed. Furthermore, assessing for germline predisposition is now advocated by multiple clinical guidelines. Although the genes identified as conferring HM cancer risk have identified new molecular pathways important for hematopoiesis and tumorigenesis, the mechanisms by which these gene variants cause HMs and BMF are not understood fully.

This year, we anticipate sessions on the science and clinical translation of germline RUNX1-mutant HMs; complex genetics and variant analysis; mechanistic insights from disease models; and new scientific discoveries in this field.

Target Audience:

International investigators who are actively studying HMs and BMF, trainees, and clinicians.

Objectives:

  • Discuss on-going research efforts in specific predisposition syndromes.
  • Strengthen existing and develop new international collaborations in these diseases.
  • Discuss the novel biology of predisposition syndromes.
  • Highlight model systems for mechanistic exploration of HMs and BMF syndromes.
  • Discuss the clinical implications of scientific progress in the area of germline predisposition to HMs BMF.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:02Ìý±è.³¾.
San Diego Convention Center , Room 6CF

Moderator:

Marcin Wlodarski , MD, PhD
St. Jude Children's Research Hospital
Memphis,  TN

Speaker:

Marcin Wlodarski , MD, PhD
St. Jude Children's Research Hospital
Memphis,  TN
Opening Remarks

Germline RUNX1-Mediated Disease

2:02Ìý±è.³¾.Ìý-Ìý2:41Ìý±è.³¾.
San Diego Convention Center , Room 6CF

Moderators:

Abbye McEwen , MD, PhD
The University of Washington
Seattle,  WA

Lea Cunningham , MD
National Institutes of Health
Bethesda,  MD

Speakers:

Abbye McEwen , MD, PhD
The University of Washington
Seattle,  WA
Moderator Introduction

Hiroki Goto , MD, PhD
Albert Einstein College of Medicine
Bronx,  NY
Molecular and Phenotypic Analysis of RUNX1-Familial Platelet Disorder-to-Acute Myeloid Leukemia Progression Using a Novel RUNX1-FPD Mouse Model

Erica Bresciani , PhD
NHGRI/NIH
Bethesda,  MD
Single Cell Transcriptome Analysis of CD34+ Cells from Patients with RUNX1-Fpdmm at Different Stages of Disease Progression

Kivilcim Ozturk , PhD
University of California San Diego
San Diego,  CA
Reclassifying Variants of Uncertain Significance with Transcriptional Profiling

Mona M.Hosseini , PhD,BS
Oregon Health and Science University
Portland,  OR
The Impact of Inflammatory and Prosurvival Pathways Inhibition in Rescuing Hematopoietic Differentiation Defects in Familial Platelet Disorder

Serine Avagyan , MD,PhD
University of California San Francisco
San Francisco,  CA
Effects of Somatic BCOR Mutations on Hematopoiesis

Lea Cunningham , MD
National Institutes of Health
Bethesda,  MD
Outcomes of Patients with Germline RUNX1 Hematologic Malignancies Undergoing Allogeneic Hematopoietic Stem Cell Transplantation: A Retrospective Analysis

DDX41-Mediated Malignancies

2:41Ìý±è.³¾.Ìý-Ìý3:25Ìý±è.³¾.
San Diego Convention Center , Room 6CF

Moderators:

Satish Nandakumar , MBBS, PhD
Albert Einstein College of Medicine
New York,  NY

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University Hospital Vall d´Hebron
Barcelona,  Spain

Speakers:

Satish Nandakumar , MBBS, PhD
Albert Einstein College of Medicine
New York,  NY
Moderator Introduction

Honghao Bi , PhD
Northwestern University
Chicago,  IL
DDX41 Dissolves G-Quadruplexes to Maintain Erythroid Genomic Integrity and Prevent Cgas Activation-Induced Erythropoiesis Dysfunction

Ayana Kon , MD,PhD
Graduate School of Medicine, Kyoto University
Kyoto,  Japan
Pathogenic Mechanisms of DDX41 Mutations in the Development of Myeloid Malignancies

Ludovica Marando , MD,PhD
Mayo Clinic
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Investigating the Role of the R525H DDX41 Somatic Variant in the Context of DDX41 Mutant Germline Predisposition Syndrome

Yael Kusne , MD, PhD
Mayo Clinic
Phoenix,  AZ
Therapy Related Myeloid Neoplasms in Patients with DDX41 Mutant Germline Predisposition Syndrome

Marie Sebert , MD,PhD
Saint-Louis Hospital
Paris,  France
DDX41 in Real Life

Kirsten Gronbaek , MD, PhD, PR
Rigshospitalet, University of Copenhagen
Copenhagen N,  Denmark
Characterizing Dead-Box ATP-Dependent RNA Helicase 41 (DDX41) Germline Variants in Hereditary Myeloid Neoplasms

Carmelo Gurnari , MD,PhD
Cleveland Clinic Foundation
Cleveland,  OH
Germline/Somatic Genomic Constellations Predict Outcomes in DDX41-Mutant Myeloid Neoplasia

Timothy Chlon , PhD
Cincinnati Children's Hospital Medical Center
Cincinnati,  OH
Functional Analysis of Germline DDX41 Variants Associated with Myelodysplastic Syndromes (MDS)/ Acute Myeloid Leukemia (AML)

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Health Research Institute of Santiago de Compostela (IDIS)
Santiago De Compostela,  Spain
In silico Analysis of DDX41 Inhibitors Binding Mechanisms

Rapid Presentations of New Insights

3:26Ìý±è.³¾.Ìý-Ìý4:06Ìý±è.³¾.
San Diego Convention Center , Room 6CF

Moderators:

Seishi Ogawa , MD,PhD
Kyoto University
Sakyoku,  KYO, Japan

Outi Kilpivaara , PhD
University of Helsinki
Helsinki,  Finland

Speakers:

Seishi Ogawa , MD,PhD
Kyoto University
Sakyoku,  KYO, Japan
Moderator Introduction

Lauren Harmon , PhD
Van Andel Institute
GRAND RAPIDS,  MI
Germline Genetic Variation in Acute Leukemia

Lara Wahlster , MD, PhD
Dana-Farber Cancer Institute
Boston,  MA
Elucidating the Mechanisms of Genetic Susceptibility in Childhood Acute Lymphoblastic Leukemia through the Lens of Genomics

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Nagoya University
Nagoya,  Japan
Proteogenomic Diagnosis for Inherited Bone Marrow Failure Syndrome

Sushree Sahoo , PhD
St. Jude Children's Research Hospital
Memphis,  TN
Molecular Mechanisms of Monosomy 7 in Pediatric MDS Predisposing Syndromes: Stochastic Versus Germline-Driven Nonrandom Evolution

Terra L. Lasho , PhD
Mayo Clinic
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Spectrum of Bone Disease in Adult Patients with Telomere Biology Disorders

Ira Kraft , MD
National Institutes of Health
Bethesda,  MD
Allogeneic Hematopoietic Cell Transplantation with Briquilimab (JSP191)-Based Conditioning for GATA2 Deficiency

William Mannherz , PhD
Boston Children's Hospital
Boston,  MA
Towards the Therapeutic Manipulation of Nucleotide Metabolism for Telomere Biology Disorders

Richard Voit , MD,PhD
University of Texas Southwestern Medical Center
Dallas,  TX
Risks and Rewards of a Unified Gene Therapy for Diamond-Blackfan Anemia

Carlos Bravo-Perez , MD
Taussig Cancer Institute, Cleveland Clinic
Cleveland,  OK
Inborn Errors of Immunity in T-Cell Large Granular Lymphocyte Leukemia

Varun Gupta , Masters in Bioinformatics
Albert Einstein College of Medicine
Bronx,  NY
Clonal Hematopoiesis and Ethnicity: Uncovering Genetic Predispositions in a Multi-Ethnic NYC Cohort

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Applied Tumor Genomics, Research Programs Unit, Faculty of Medicine, University of Helsinki
Helsinki,  Finland
Single Cell Transcriptome Analysis on ERCC6L2 Disease Patients’ Bone Marrow Samples

Sara Torres-Esquius , MSc
Vall d'Hebron Universitary Hospital
Barcelona,  Spain
Performance and Clinical Utility of Germline Genetic Testing Criteria for Predisposition to Myeloid Neoplasms in Adults

New Scientific Discoveries

4:06Ìý±è.³¾.Ìý-Ìý4:56Ìý±è.³¾.
San Diego Convention Center , Room 6CF

Moderators:

Akiko Shimamura , MD,PhD
Boston Children's Hospital
Boston,  MA

Alejandro Ferrer , PhD
Mayo Clinic
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Speakers:

Akiko Shimamura , MD,PhD
Boston Children's Hospital
Boston,  MA
Moderator Introduction

³§±ð²¹²ÔÌý±á²¹°ù°ù´Ç±è
Royal Melbourne Hospital and Peter MacCallum Cancer Centre
Melbourne,  VIC, Australia
Heterozygous Germline TET2 Loss Function Variants Predispose to Development of Nodular Lymphocyte Predominant Hodgkin Lymphoma

Lili Kotmayer , MD, PhD
St. Jude Children's Research Hospital
Memphis,  TN
Genetic Landscape and Clinical Correlates in 965 Individuals with Germline GATA2 Mutations

Lauren Banaszak , MD
University of Wisconsin-Madison
Madison,  WI
ANKRD26-Related Thrombocytopenia: Hematologic Malignancy Characteristics, Outcomes, and Precursor States

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University of Bologna
Bologna,  Italy
Biallelic Germline Variants in SH2B3 Cause a Multisystem Developmental Disorder Characterized By a Transient Neonatal Myeloproliferative Disorder, Subsequent Thrombocytosis, and Autoimmune Manifestations in Childhood

Kristen E. Schratz , MD
Johns Hopkins University School of Medicine
Baltimore,  MD
Divergent Clonal Populations Have Distinct Telomere Lengths in POT1 Mutation Carriers

Agnieszka D. Czechowicz , MD,PhD
Stanford University
Stanford,  CA
Reduced-Toxicity Hematopoietic Stem Cell Therapies for Fanconi Anemia

Maryam Rafati , MD,PhD
National Cancer Institute, NIH
Rockville,  MD
Germline Pathogenic Variants in DNA Damage Response and Repair Genes and Their Impact on HCT Outcomes in 2008 Patients with Severe Aplastic Anemia, Myelofibrosis and Myelodysplastic Syndrome

Closing Remarks

4:56Ìý±è.³¾.Ìý-Ìý5:00Ìý±è.³¾.
San Diego Convention Center , Room 6CF

Moderator:

Lucy A. Godley , MD,PhD
Northwestern University
Chicago,  IL

Speaker:

Lucy A. Godley , MD,PhD
Northwestern University
Chicago,  IL
Closing Remarks

Friday, December 6, 2024, 2:00 p.m. - 4:40 p.m.

Co-Chairs:

Jeffrey I. Zwicker , MD
Memorial Sloan Kettering Cancer Center
New York,  NY

Lisa Baumann Kreuziger , MD,MS
Versiti
Milwaukee,  WI

Thrombosis is a common complication of cancer and its therapy. This workshop will provide a forum for the hemostasis and thrombosis community to discuss ongoing investigations into mechanisms of thrombosis in cancer, the interplay of coagulation factors and tumor progression, and address more broadly the mechanisms of thrombo-inflammation.

The workshop will feature short research presentations with equal focus on discussion to foster interactions between basic and translational researchers interested in cancer and thrombosis.

Target Audience:

Basic scientists and researchers interested in the mechanisms of thrombosis in cancer patients, thrombo-inflammation, and how thrombosis may influence the progression of cancer. Clinical researchers may benefit from the workshop to understand the mechanisms of disease and identify potential collaborations. Participation from early career and senior investigators will also be encouraged to facilitate mentoring/networking opportunities.

Objectives:

  • Provide a unique forum to discuss the latest scientific developments in cancer and thrombosis.
  • Enhance current collaborations, develop new collaborations, and provide opportunities for interaction between early career and established investigators in cancer and thrombosis.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:05Ìý±è.³¾.
San Diego Convention Center , Room 24

Moderator:

Jeffrey I. Zwicker , MD
Memorial Sloan Kettering Cancer Center
New York,  NY

Speaker:

Jeffrey I. Zwicker , MD
Memorial Sloan Kettering Cancer Center
New York,  NY
Opening Remarks

Tissue Factor(TF) and Tissue Factor Pathway Inhibitor(TFPI) in Cancer Associated Thrombosis

2:05Ìý±è.³¾.Ìý-Ìý3:05Ìý±è.³¾.
San Diego Convention Center , Room 24

Moderator:

Alan E. Mast , MD,PhD
Versiti Blood Research Institute
Milwaukee,  WI

Speakers:

Alan E. Mast , MD,PhD
Versiti Blood Research Institute
Milwaukee,  WI
Moderator Introduction

Florian Moik , MD, PhD
Medical University of Graz
Graz,  Austria
Tissue Factor Pathway Inhibitor As Biomarker in Cancer Associated Thrombosis

Rory R. Koenen
Maastricht University
Maastricht,  Netherlands
Inactivation of Tissue Factor Pathway Inhibitor

Irene Martínez-Martínez , PhD
BioMedical Research Institute of Murcia (IMIB-Pascual Parrilla)
Murcia,  Spain
mir-5683 Inhibition Endothelial Tissue Factor Pathway Inhibitor in Cancer

Yohei Hisada , PhD
University of North Carolina at Chapel Hill
Chapel Hill,  NC
Mouse Xenograft Models of Acute Promyelocytic Leukemia and Coagulopathy

Thromboinflammation of Cancer Associated Thrombosis

3:10Ìý±è.³¾.Ìý-Ìý4:35Ìý±è.³¾.
San Diego Convention Center , Room 24

Moderators:

Nicola Potere , MD
G. D'Annunzio University of Chieti-Pescara
Chieti,  Italy

Lalitha Nayak , MD
University Hospitals Cleveland Medical Center
Cleveland,  OH

Speakers:

Nicola Potere , MD
G. D'Annunzio University of Chieti-Pescara
Chieti,  Italy
Moderator Introduction

Steven Grover , PhD
University of North Carolina at Chapel Hill
Chapel Hill,  NC
PAR-1 and Doxorubicin Induced Cardiotoxicity

Ang Li , MD,MS
Baylor College of Medicine
Houston,  TX
Circulating Tumor DNA and Thrombosis in Cancer

Marisa Brake , PhD
Harvard Medical School
Boston,  MA
CD200R1 and IL-17 Axis in Cancer Associated Thrombosis

Joan D Beckman , MD,PhD
University of Minnesota
Minneapolis,  MN
JAK-STAT Inhibition of Endothelial Activation

Alexandre Guy , MD, PhD
Biology of Cardiovascular Diseases, University of Bordeaux, INSERM, UMR1034
Pessac,  France
Thromboinflammation Markers in Myeloproliferative Neoplasms (MPNs)

Marina Marchetti , PhD
Papa Giovanni XXIII Hospital
Bergamo,  Italy
Hypercoagulability and Inflammatory Biomarkers Predict Disease Progression Non-Small Cell Lung Cancer Patients

Closing Remarks

4:35Ìý±è.³¾.Ìý-Ìý4:40Ìý±è.³¾.
San Diego Convention Center , Room 24

Moderator:

Lisa Baumann Kreuziger , MD,MS
Versiti
Milwaukee,  WI

Speaker:

Lisa Baumann Kreuziger , MD,MS
Versiti
Milwaukee,  WI
Closing Remarks

Friday, December 6, 2024, 2:00 p.m. - 5:00 p.m.

Co-Chairs:

Aaron D Schimmer , MD
Princess Margaret Cancer Centre
Toronto,  ON, Canada

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Albert Einstein College of Medicine
Bronx,  NY

Mitochondrial and metabolic pathways play an important role in the pathogenesis of blood cancers. In addition, they represent a biological vulnerability that can be targeted therapeutically in some patients. This workshop will bring together a multidisciplinary group of researchers to discuss mitochondrial pathways and metabolism in blood cancers with the goal of facilitating collaborations between investigators from diverse fields who would likely not otherwise interact.

The workshop will feature sessions on:

  • Fundamental discoveries in mitochondria and metabolism that will offer new insight into blood cancer biology and potential therapies. Talks may include discoveries in model organisms.
  • Translational research – Targeting mitochondria and metabolism in blood cancer. Speakers will discuss preclinical studies of novel mitochondrial and metabolic targets for the treatment of blood cancers.
  • Clinical research – Talks will include discussions of therapies targeting mitochondrial and metabolic pathways.

Target Audience:

Basic scientists, translational researchers, and clinician investigators studying mitochondrial pathways and metabolism in the lab and/or clinic.

Objectives:

  • Discuss fundamental discoveries in mitochondrial and metabolic pathways that will inform on new biology in blood cancers.
  • Identify new therapeutic strategies to target dysregulated metabolism in blood cancer and discuss their effectiveness in preclinical models and clinical trials.
  • Spark discussion and collaboration among investigators with diverse background who share a common interest in metabolism.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:05Ìý±è.³¾.
San Diego Convention Center , Room 2

Moderator:

Aaron D Schimmer , MD
Princess Margaret Cancer Centre
Toronto,  Canada

Speaker:

Aaron D Schimmer , MD
Princess Margaret Hospital, University of Toronto
Toronto,  ON, Canada
Opening Remarks

Mitochondria and Metabolism - Fundamental Discovery

2:05Ìý±è.³¾.Ìý-Ìý2:50Ìý±è.³¾.
San Diego Convention Center , Room 2

Moderator:

Steven M. Chan , MD,PhD
Princess Margaret Cancer Centre
Toronto,  ON, Canada

Speakers:

Steven M. Chan , MD,PhD
Princess Margaret Cancer Centre
Toronto,  ON, Canada
Moderator Introduction

Kelsey H Fisher-Wellman , PhD
Wake Forest University
Winston-Salem,  NC
Mitochondrial Polarization and Venetoclax Sensitivity

Stuart A Rushworth , PhD
The University of East Anglia
Norwich,  United Kingdom
Blood Cancer Tumor Microenvironment and Metabolism

Translational Research- Targeting Mitochondria and Metabolism in Cancer

3:05Ìý±è.³¾.Ìý-Ìý3:50Ìý±è.³¾.
San Diego Convention Center , Room 2

Moderator:

Aditi Shastri , MD
Montefiore Medical Center
Bronx,  NY

Speakers:

Aditi Shastri , MD
Montefiore Medical Center
Bronx,  NY
Moderator Introduction

Ilaria Iacobucci , PhD
St. Jude Children's Research Hospital
Memphis,  TN
Metabolic Dependencies and Vulnerabilities in Acute Erythroid Leukemia

Aaron D Schimmer , MD
Princess Margaret Cancer Centre
Toronto,  Canada
Mitochondrial Stress in Acute Myeloid Leukemia- Biology to Therapy

Mitochondria and Metabolism - Blood Cancer Clinical Trials

4:05Ìý±è.³¾.Ìý-Ìý4:50Ìý±è.³¾.
San Diego Convention Center , Room 2

Moderator:

Sarah Skuli , MD,PhD
University of Pennsylvania
Philadelphia,  PA

Speakers:

Sarah Skuli , MD,PhD
University of Pennsylvania
Philadelphia,  PA
Moderator Introduction

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Albert Einstein College of Medicine
Bronx,  NY
Glutaminolysis in Myelodysplastic Syndromes/Acute Myeloid Leukemia as a Therapeutic Target

Kathleen M. Sakamoto , MD,PhD
Stanford University
Stanford,  CA
Targeting Oxidative Phosphorylation for Pediatric Blood Cancer- from the Lab to the Clinic

Closing Remarks

4:55Ìý±è.³¾.Ìý-Ìý5:00Ìý±è.³¾.
San Diego Convention Center , Room 2

Moderator:

²Ñ²¹°ù¾±²Ô²¹Ìý°­´Ç²Ô´Ç±è±ô±ð±¹²¹
Albert Einstein College of Medicine
Bronx,  NY

Speaker:

²Ñ²¹°ù¾±²Ô²¹Ìý°­´Ç²Ô´Ç±è±ô±ð±¹²¹
Albert Einstein College of Medicine
Bronx,  NY
Closing Remarks

Friday, December 6, 2024, 2:00 p.m. - 5:00 p.m.

Co-Chairs:

Christina Glytsou , PhD
Rutgers University
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Daniel Starczynowski , PhD
University of Cincinnati College of Medicine
Cincinnati,  OH

Panagiotis Ntziachristos , PhD
Ghent University
Corneel Heymanslaan 10,  Ghent, Belgium

Resistance to therapy poses a significant challenge in the treatment of blood disorders, largely due to the limited study of the underlying mechanisms. This workshop will cover aspects of resistance to therapy in blood malignancies, focusing on genetic as well as non-genetic molecular mechanisms. Topics will include epigenetic, epitranscriptomic, translational, post-translation, and metabolic aspects, as well as their potential interaction.

Pre-existing or adaptive, cell autonomous or non-autonomous mechanisms of resistance will be discussed, along with novel systems to investigate them and potential therapeutic interventions to tackle them. Experts in molecular mechanisms of therapy resistance in tumor cells will share the latest updates and insights from their research. Participants will gain a comprehensive understanding of the complexities of therapy resistance and explore new avenues for therapeutic approaches. Lastly, this interdisciplinary workshop will serve as a forum for the exchange of ideas and collaboration among investigators from various fields.

Target Audience:

Researchers and clinical investigators interested in the underlying causes of therapy resistance in blood disorders. Both early-career and experienced investigators are encouraged to participate to foster mentoring, networking, and collaboration.

Objectives:

  • Help scientists working in the field of hematology understand mechanisms of resistance to therapy.
  • Interrogate various mechanisms of resistance.
  • Provide a forum for open discussion and exchange of ideas that will assist the development of solutions to overcome the challenge of relapse in blood cancers.
  • Promote collaboration and exchange of technologies.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:03Ìý±è.³¾.
San Diego Convention Center , Room 33

Moderator:

Panagiotis Ntziachristos , PhD
Ghent University
Corneel Heymanslaan 10,  Ghent, Belgium

Speaker:

Panagiotis Ntziachristos , PhD
Ghent University
Corneel Heymanslaan 10,  Ghent, Belgium
Opening Remarks

Epigenetic And Chromatin Mechanisms

2:03Ìý±è.³¾.Ìý-Ìý2:44Ìý±è.³¾.
San Diego Convention Center , Room 33

Moderator:

Panagiotis Ntziachristos , PhD
Ghent University
Corneel Heymanslaan 10,  Ghent, Belgium

Speakers:

Panagiotis Ntziachristos , PhD
Ghent University
Corneel Heymanslaan 10,  Ghent, Belgium
Moderator Introduction

Yadira Soto-Feliciano , PhD
Massachusetts Institute of Technology
Cambridge,  MA
Mechanisms of Gene Regulation By Chromatin Adaptors in Development and Disease

Stavroula Kousteni , PhD
Columbia University
New York,  NY
Aberrant ß-catenin Signaling from the Niche Transforms IDH1/2-Mutant Pre-malignant Stem Cells and Compromises Response to IDH1/2 Inhibitors

Constantine S Mitsiades , MD
Dana-Farber Cancer Institute
Boston,  MA
Cooperation of Non-Genomic and Genomic Mechanisms of Persistence /Resistance to Pharmacological and Immune Based Therapies

Post-transcriptional Mechanisms

2:44Ìý±è.³¾.Ìý-Ìý3:25Ìý±è.³¾.
San Diego Convention Center , Room 33

Moderator:

Christina Glytsou , PhD
Rutgers University
±Ê¾±²õ³¦²¹³Ù²¹·É²¹²â,Ìý NJ

Speakers:

Christina Glytsou , PhD
Rutgers University
±Ê¾±²õ³¦²¹³Ù²¹·É²¹²â,Ìý NJ
Moderator Introduction

Jianjun Chen , PhD
City of Hope Comprehensive Cancer Center
Duarte,  CA
RNA Modification and Therapy Resistance in Acute Leukemia

Stephanie Halene , MD
Yale Univ. School of Medicine
New Haven,  CT
Exploiting Epitranscriptomic Vulnerabilities: M6a and Splicing Factor Mutations in Myelodysplastic Syndrome (MDS)/Leukemia

Wei Tong , PhD
Children's Hospital of Philadelphia
Philadelphia,  PA
Targeting Casitas B-lineage Lymphoma (CBL) Mutant Myeloid Malignancies

Translational and Post-translational Mechanisms

3:35Ìý±è.³¾.Ìý-Ìý4:16Ìý±è.³¾.
San Diego Convention Center , Room 33

Moderator:

Daniel Starczynowski , PhD
University of Cincinnati College of Medicine
Cincinnati,  OH

Speakers:

Daniel Starczynowski , PhD
University of Cincinnati College of Medicine
Cincinnati,  OH
Moderator Introduction

Kira Gritsman , MD,PhD
Albert Einstein College of Medicine
Bronx,  NY
Targeting an Epigenetic Resistance Mechanism to PI3 Kinase Inhibition in Acute Myeloid Leukemia

Stephen Oh , MD,PhD
Washington University School of Medicine
Saint Louis,  MO
Aberrant DUSP6-RSK1 Signaling Axis Mediates Treatment Resistance and Disease Progression in Myeloproliferative Neoplasms

Marek Mraz , MD
Central European Institute of Technology (CEITEC), Masaryk University
Brno,  Czech Republic
Non-Genetic Adaptation to Bruton Tyrosine Kinase (BTK) Inhibitors in Chronic Lymphocytic Leukemia

Metabolic Mechanisms and Mitochondria Biology

4:17Ìý±è.³¾.Ìý-Ìý4:58Ìý±è.³¾.
San Diego Convention Center , Room 33

Moderator:

Christina Glytsou , PhD
Rutgers University
±Ê¾±²õ³¦²¹³Ù²¹·É²¹²â,Ìý NJ

Speakers:

Christina Glytsou , PhD
Rutgers University
±Ê¾±²õ³¦²¹³Ù²¹·É²¹²â,Ìý NJ
Moderator Introduction

David C.S. Huang , MBBS,PhD,MRCP
Walter and Eliza Hall Institute of Medical Research
Melbourne,  Australia
Impact of Mitochondrial Biology on BCL2 Inhibition

Victoria da Silva-Diz , PhD
Rutgers University
Highland Park,  NJ
Targeting Novel Metabolic Vulnerabilities in T-Cell Leukemia

Nina Cabezas-Wallscheid , Prof. Dr.
Max Planck Institute
Freiburg,  Germany
Targeting Dormant Hematopoietic Stem Cells in Leukemogenesis

Closing Remarks

4:58Ìý±è.³¾.Ìý-Ìý5:00Ìý±è.³¾.
San Diego Convention Center , Room 33

Moderator:

Christina Glytsou , PhD
Rutgers University
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Speaker:

Christina Glytsou , PhD
Rutgers University
±Ê¾±²õ³¦²¹³Ù²¹·É²¹²â,Ìý NJ
Closing Remarks

Friday, December 6, 2024, 2:00 p.m. - 5:00 p.m.

Co-Chairs:

Jonathan H. Schatz , MD
University of Miami
Miami,  FL

Francesco Maura , MD
University of Miami
Coral Gables,  FL

To date, resistance studies have examined properties of chimeric antigen receptor (CAR) products and clinical factors like inflammatory markers and tumor burden. Lymphoma microenvironments (LMEs) associated with outcome also have been described, but further mechanistic details are lacking. Tumor-intrinsic resistance mechanisms mediated by malignant genomes is even less well understood, likely because of the functional studies necessary to establish the bases of genotype-phenotype and genotype-outcome relationships. Increasingly, these key unknowns are the focus of leaders in the CAR-T research community.

This workshop will examine new approaches to understand resistance to CD19-directed chimeric-antigen receptor (CAR) T-cell products. Specifically, the workshop will focus on unique genomic changes that impact CAR-T outcomes compared to standard therapies, functional laboratory studies to define mechanisms, and their connections to LMEs.

Lastly, the workshop will utilize a multidisciplinary focus on work in progress paired with interactive sessions to foster participant understanding of emerging approaches necessary to tackle new challenges in hematology.

Target Audience:

Basic scientists, pathologists, translational researchers and clinicians

Objectives:

  • Promote wider understanding of the unique genomic changes that associate with CAR-T responses in contrast to standard therapies and the techniques and specialized analyses necessary to define them.
  • Highlight emerging data and techniques that examine relationships between lymphoma genotypes and immunophenotypes.
  • Examine relationships between infused CAR-T cells and non-CAR host T cells and how their interactions shape CAR-T outcomes.
  • Propose paradigms to examine the impact of individual genes altered in tumor genomes able to promote CAR-T resistance.
  • Provide discussion frameworks for using results of resistance studies to inform design of next-generation cellular immunotherapies with potential to benefit greater numbers of patients.

Workshop Program:

The full workshop program with speakers will be available at a later date.

Workshop Schedule


Opening Remarks

2:00Ìý±è.³¾.Ìý-Ìý2:05Ìý±è.³¾.
San Diego Convention Center , Room 28 A-D

Moderator:

Jonathan H. Schatz , MD
University of Miami
Miami,  FL

Speaker:

Jonathan H. Schatz , MD
University of Miami
Miami,  FL
Opening Remarks

Examining the Malignant Genome in CAR-T Patients

2:05Ìý±è.³¾.Ìý-Ìý2:41Ìý±è.³¾.
San Diego Convention Center , Room 28 A-D

Moderator:

Jonathan H. Schatz , MD
University of Miami
Miami,  FL

Speakers:

Jonathan H. Schatz , MD
University of Miami
Miami,  FL
Moderator Introduction

Francesco Maura , MD
University of Miami
Coral Gables,  FL
The Unique Power of Whole Genome Sequencing

Ash A. Alizadeh , MD,PhD
Stanford University
San Mateo,  CA
Circulating Tumor DNA: All You Need Is Blood

Engaging Elusive Tumor Cells with Immune Effectors

2:41Ìý±è.³¾.Ìý-Ìý3:27Ìý±è.³¾.
San Diego Convention Center , Room 28 A-D

Moderator:

Francesco Maura , MD
University of Miami
Coral Gables,  FL

Speakers:

Francesco Maura , MD
University of Miami
Coral Gables,  FL
Moderator Introduction

Frederick Locke , MD
Moffitt Cancer Center
Tampa,  FL
Response and Resistance: A Tale of Tumor and T Cells

Justin Kline , MD
University of Chicago
Chicago,  IL
Integrative Genomic Analysis Identifies Unique Immune Environments Associated with Immunotherapy Response in Diffuse Large B-Cell Lymphoma

Jay Y Spiegel , MD,FRCPC
University of Miami
Miami,  FL
How Resistance Mechanisms Can Inform the Design of Next-Generation Cars Aimed at Improved Efficacy

Functional Laboratory Studies of Resistance

3:42Ìý±è.³¾.Ìý-Ìý4:18Ìý±è.³¾.
San Diego Convention Center , Room 28 A-D

Moderator:

Frederick Locke , MD
Moffitt Cancer Center
Tampa,  FL

Speakers:

Frederick Locke , MD
Moffitt Cancer Center
Tampa,  FL
Moderator Introduction

Jonathan H. Schatz , MD
University of Miami
Miami,  FL
Alterations in Individual Genes As Resistance Drivers: Beyond CD19

Damian Green , MD
University of Miami
Miami,  FL
Combinatorial Strategies to Improve Target Engagement for Myeloma Cars

CAR-19 Resistance in Acute Lymphoblastic Leukemia

4:18Ìý±è.³¾.Ìý-Ìý4:54Ìý±è.³¾.
San Diego Convention Center , Room 28 A-D

Moderator:

Ash A. Alizadeh , MD,PhD
Stanford University
San Mateo,  CA

Speakers:

Ash A. Alizadeh , MD,PhD
Stanford University
San Mateo,  CA
Moderator Introduction

Kara L. Davis , DO
Stanford University
Stanford,  CA
Tumor Intrinsic Factors Predicting Antigen Loss

Sara Ghorashian , FRCPath, PhD
Great Ormond Street Children's Hospital
London,  United Kingdom
Targeting CD22 and Other Strategies to Overcome CD19 Antigen Escape

Closing Remarks

4:54Ìý±è.³¾.Ìý-Ìý5:00Ìý±è.³¾.
San Diego Convention Center , Room 28 A-D

Moderator:

Francesco Maura , MD
University of Miami
Coral Gables,  FL

Speaker:

Francesco Maura , MD
University of Miami
Coral Gables,  FL
Closing Remarks

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