Scientific Workshops
Scientific Workshops are interactive discussions covering the latest scientific developments in a range of hematologic topics. The 2025 Scientific Workshops will take place on Friday, December 5, and will also be streamed on the ASH annual meeting platform for virtual participants.
Friday, December 5, 2025, 3:00 p.m. - 6:00 p.m.
Co-Chairs:
Megan Weivoda, PhD
Mayo Clinic, Division of Hematology
Rochester, MN
[email protected]
Richard Lin, MD, PhD
Memorial Sloan Kettering Cancer Center
New York, NY
[email protected]
Hematologic disorders disproportionately affect older adults and often exhibit hallmarks of biological aging. Moreover, standard treatments for hematologic diseases, including chemotherapy, radiation, and stem cell transplantation, can accelerate aging, compounding disease burden and complicating recovery. Despite growing recognition of the impact of biological aging on hematological health, therapeutic strategies to target and mitigate age-related dysfunction remain underdeveloped.
This year’s workshop will focus on critically evaluating interventions that could meaningfully alter biological aging in the context of hematologic disease. In each session, speakers will advocate for a specific class of intervention, presenting basic and translational evidence to support their position. To promote engagement and debate, sessions will include pre- and post-session audience polling and interactive cross-examination by moderators and attendees. This format is designed to challenge assumptions, sharpen proposed strategies, and build consensus — or constructive dissent — around the most promising therapeutic directions.
Discussions from this workshop are likely to inform the development of a white paper that will summarize key insights and issue a call to action.
Target Audience:
Laboratory-based scientists and clinical/translational investigators with an interest in aging and hematologic disorders — both malignant and non-malignant.
Objectives:
- Evaluate and compare emerging strategies for mitigating hematologic aging, including lifestyle, immune-based, and pharmacologic interventions.
- Highlight innovative therapeutic approaches that target cellular and molecular drivers of hematologic aging, such as senescence and chronic inflammation.
- Foster interdisciplinary collaboration aimed at discovering, validating, and advancing strategies to slow or reverse hematologic aging.
Workshop Schedule
Session Description
Speaker:
Megan Weivoda, PhD
Mayo Clinic
OPENING REMARKS
The Natural Intervention: Diet and Exercise-Based Strategies for Reversing Hematologic Aging
3:03 p.m. - 3:44 p.m., OCCC - W312
Session Description
Speakers:
Nadine Abdallah, MD
Mayo Clinic
Moderator Introduction and Audience Pre-Session Polling
Mary Riwes, DO
University of Michigan
Prebiotic Interventions to Modulate Gut Microbiota and Inflammation to Prevent Transplant-related Aging
Charity Oyedeji, MD
Duke University School of Medicine
Targeting Aging in Sickle Cell Disease Through Exercise and Nutrition
Nadine Abdallah, MD
Mayo Clinic
Panel Discussion and Cross-Examination of the Position; Post-Session Polling
Session Description
Speakers:
Haitham Abdelhakim, MD
University of Kansas Medical Center
Immune Checkpoint Blockade as a Strategy for Reversing Hematologic Aging
Yue Liu
Vaccine Targeting of Senescence for Age-Related Pathologies
Small Molecules, Big Impact: Reversing Hematologic Aging by Targeting Senescence and Inflammaging
4:35 p.m. - 5:16 p.m., OCCC - W312
Session Description
Speaker:
Jane Liesveld Jr, MD
University of Rochester
Rapamycin and the Rewiring of Aging
A New Horizon in Hematology: Novel Strategies for Reversing Aging
5:16 p.m. - 5:57 p.m., OCCC - W312
Session Description
Speakers:
Conor Lynch, PhD
H. Lee Moffitt Cancer Center And Research Institute
Moderator Introduction
Jonathan Yen, PhD
St. Jude Children's Research Hospital
Precision Editing: Harnessing CRISPR to Rejuvenate Blood Cells
Conor Lynch, PhD
H. Lee Moffitt Cancer Center And Research Institute
Panel Discussion and cross-examination of the position; post-session polling
Friday, December 5, 2025, 3:00 p.m. - 6:00 p.m.
Co-Chairs:
Jane S. Hankins, MD, MS
St. Jude Children’s Research Hospital
Memphis, TN
[email protected]
Dunia Hatabah, MD
Emory University School of Medicine
Atlanta, GA
[email protected]
Caterina Minniti, MD
Albert Einstein College of Medicine
Bronx, NY
[email protected]
Claudia R. Morris, MD
Emory University School of Medicine; Children’s Healthcare of Atlanta
Atlanta, GA
[email protected]
There is currently a lack of consensus on how to classify disease severity in sickle cell disease (SCD), which has hindered progress in the field. This uncertainty has greatly limited the ability to characterize the disease phenotype, affecting optimal decision-making and accurate risk prediction. Moreover, the inability to properly define disease severity has significantly impaired the design of suitable endpoints for clinical trials.
Traditionally, the severity of SCD has been primarily defined by the frequency of vaso-occlusive episodes (VOEs). This metric has served as the foundation for clinical trial eligibility and the development of all currently U.S. Food and Drug Administration (FDA)-approved therapies for SCD. However, VOE frequency is a narrow and inadequate measure of the overall disease burden. VOEs are inconsistently defined and do not capture the broader spectrum of complications and organ damage associated with the disease. Additionally, VOEs are difficult to quantify and susceptible to variability in clinical practice, which threatens the internal validity of studies that rely on this outcome measure. Unsurprisingly, many randomized controlled trials that showed promise in phase II studies — using VOE as the primary outcome (e.g., time to VOE resolution for acute pain studies, number of VOEs for prophylactic therapies), which the FDA prefers for orphan drug approval — failed to meet their primary endpoints in phase III studies.
A universally accepted and formalized grading system for categorizing disease severity has yet to be established. Such a system could standardize clinical assessments, guide personalized therapeutic interventions, improve predictive modeling for morbidity and mortality, and facilitate the calculation of cumulative morbidity. Moreover, a severity grading system with discrete outcomes that reflect incremental disease severity over time would more accurately represent the disease phenotype, potentially serving as a better clinical endpoint for future trials in SCD, both for acute and chronic complications, as well as preventive treatments (e.g., new disease-modifying agents).
In addition to establishing discrete and cumulative clinical endpoints informed by a standardized grading of severity, clinical trial designs for both acute (i.e., pain and acute chest syndrome) and chronic complications (i.e., anemia, end-organ damage, chronic pain) could be considerably enhanced through the use of surrogate biomarkers. Laboratory biomarkers such as fetal hemoglobin (HbF), sickling point, mitochondrial function, oxidative stress, adhesion properties, and arginine bioavailability are all promising surrogate biomarkers that could be utilized, either independently or as complementary measures to clinical endpoints.
This workshop will engage a panel of global experts in hematology: clinical trial design, implementation science, and patient-centered outcomes. The discussion will cover topics related to classifying disease severity in SCD, selecting surrogate biomarkers, and optimizing the use of such classifications and biomarkers to improve endpoint selection for future clinical trials in SCD.
Target Audience:
Hematologists, translational researchers, laboratory scientists, emergency medicine physicians, SCD researchers and trainees, regulatory agencies (e.g., FDA, clinical investigators, and SCD clinical trialists), federal funders such as the National Institutes of Health (NIH) and the National Heart, Lung, and Blood Institute (NHLBI), and SCD advocacy groups including the Sickle Cell Disease Association of America, as well as patients with SCD and their families.
Objectives:
- Discuss the existing severity scoring systems in SCD.
- Understand the utility and current availability of biomarkers in SCD in defining disease severity.
- Initiate dialogue between the FDA, ASH leadership, SCD researchers/clinical trialists, and basic scientists/biomarker experts to identify more optimal and objective endpoints to be used for future clinical trials in SCD to address both acute and chronic complications of this orphan disease.
Workshop Schedule
Session Description
Speaker:
Dunia Hatabah
Opening Remarks
Session Description
Speakers:
Jane Hankins, MD,MS
St Jude Children's Research Hospital
Moderator Introduction: Topic Only
Elizabeth Klings, MD
Boston University
Prior Disease Severity Scoring Systems
Courtney Thornburg, MD, MS
Rady Children's Health - Orange County
Defining Disease Severity for NHLBI Innovative Trials
Tarun Aurora, MD, MSCI
Emory University
A New Severity Classification System (SCOGS)
Jane Hankins, MD,MS
St Jude Children's Research Hospital
Engagement/Discussion/Q&A
Session Description
Speakers:
Caterina Minniti, MD
Einsten College of Medicine
Moderator Introduction
Patrick Hines, MD
Functional Fluidics
Adhesions and Red Cell Health as a Marker of Propensity of VOC
Martin Steinberg
HbF is the Most Powerful Disease Modifier in SCD: What is Next for This Biomarker?
Vence L. Bonham Jr, J.D.
National Human Genome Research Institute
Patient Reported Outcomes, Indicators of Severity and End Points for Clinical Trials
Caterina Minniti, MD
Einsten College of Medicine
Engagement/Discussion/Q&A
Acute Vaso-occlusive Pain Clinical Trial Endpoints Beyond Time-to-Crisis-Resolution in Sickle Cell Disease (SCD)
4:35 p.m. - 5:15 p.m., OCCC - W315
Session Description
Speakers:
Claudia R. Morris, MD
Emory University School of Medicine
Moderator Introduction
Claudia R. Morris, MD
Emory University School of Medicine
Challenges with Time-to-Crisis-Resolution as an Endpoint in Acute SCD Pain Clinical Trials
Nitya Bakshi, MBBS, MS
Yale University
Challenges with PROs in Acute SCD Pain Clinical Trials: Why We Need Surrogate Biomarkers
Sruti Shiva
Mitochondria as a Surrogate Biomarker: Much more than ATP Makers
Claudia R. Morris, MD
Emory University School of Medicine
Engagement/Discussion/Q&A
Session Description
Speakers:
Dunia Hatabah
Moderator Introduction
Dunia Hatabah
Panel Discussion/Q&A
Friday, December 5, 2025, 3:00 p.m. - 6:00 p.m.
Co-Chairs:
Lucy A. Godley, MD, PhD
Northwestern University
Chicago, IL
[email protected]
Marcin Wlodarski, MD, PhD
St. Jude Children's Research Hospital
Memphis, TN
[email protected]
Germline variants conferring susceptibility to hematopoietic malignancies (HMs) and bone marrow failure (BMF) are now recognized to occur far more frequently than previously assumed. Current clinical guidelines strongly advocate for assessing germline predisposition in these conditions. Although identified genes have revealed novel molecular pathways crucial for hematopoiesis and tumorigenesis, the precise mechanisms underlying these disorders remain incompletely understood.
This workshop will feature four strategic sessions: two overarching sessions exploring disease models/mechanisms and emerging discoveries across the field, and two focused sessions dedicated to DNA repair defects and ribosomopathies.
By attending this workshop, attendees will gain comprehensive insight into this increasingly captivating topic (which is minimally covered at the annual meeting), while engaging in stimulating debate.
Target Audience:
International investigators who are actively studying HMs and BMF, trainees, and clinicians.
Objectives:
- Discuss ongoing research efforts in germline predisposition disorders.
- Address disease models and mechanisms that bridge fundamental science with clinical applications, with two focused sessions on DNA repair defects and ribosomopathies.
- Strengthen existing and develop new international collaborations in these diseases..
Workshop Schedule
Session Description
Speaker:
Marcin W Wlodarski, MD, PhD
St. Jude Children's Research Hospital
Opening Remarks
Session Description
Speakers:
Gina Keiffer, MD
Sidney Kimmel Comprehensive Cancer Center, Thomas Jefferson University
Moderator Introduction
Simona Colla, PhD
The University of Texas MD Anderson Cancer Center
U2AF1S34 Mutations Activate Telomerase to Overcome Telomere Dysfunctions in Patients with Telomere Biology Disorders
Melvin Thomas III, PhD
St. Jude Children's Research Hospital
Characterizing the in Vivo Impact of Inflammation on Mutant Samd9l Hematopoietic Phenotypes
Stanley Lee, PhD
Fred Hutchinson Cancer Center,
Fred Hutch
Uncovering the Pathogenic Mechanisms of DDX41 Mutations in Myeloid Malignancies
Ayana Kon, MD, PhD
Univ.Tokyo Kyoto Univesity Dept.Pathology and Tumor Biology
Mechanistic Insights into Biallelic and Monoallelic DDX41 Mutations in Myeloid Neoplasms
Sharon Singh, MD
University Of Michigan
The Mechanism of Defective Erythropoiesis in DBA Syndrome
Elif Cenik, PhD
University of Texas
Differential Impacts of Ribosomal Protein Haploinsufficiency on Mitochondrial Function
Neha Prasad, PhD
Cleveland Clinic
Activation of Stress-Activated Protein Kinase in Bone Marrow Failure Ribosomopathies
Ribosomopathies: From Diamond-Blackfan Anemia to Shwachman-Diamond Syndrome and Beyond
3:49 p.m. - 4:36 p.m., OCCC - W307
Session Description
Speakers:
Alan Warren, PhD, FRCP, FRCPath
University of Cambridge
Moderator Introduction
Alexandra Russo Sr, MD
Universitätsmedizin der Johannes Gutenberg- Universität Mainz
Decoding Epitranscriptomic Drivers of Malignancy: RNA Modopathies Beyond the Genetic Code
Alyssa Cull, PhD
University of York
Emerging Genetic Technologies Inform Personalized Medicine Approaches in Shwachman-Diamond Syndrome
Sarada Ketharnathan, PhD
CHEO Research Institute
Leveraging Metabolic Alterations for the Development of Pre-Emptive Leukemia Therapies in Dyskeratosis Congenita and Shwachman-Diamond Syndrome
Felicia Andresen, MD, PhD
Boston Children's Hospital
Concordance Of Somatic Genetic Testing in Blood Vs. Bone Marrow in Shwachman-Diamond Syndrome
Jonathan Roelof Adriaan de Wilde
Activation Of Pyruvate Kinase by Mitapivat Potentially Rescues Ineffective Erythropoiesis in Models of Diamond Blackfan Anemia
Yirun Miao, PhD
St. Anna Children's Cancer Research Institute
Investigating The Role of a Ribosome Biogenesis Factor in A DBA-Like Congenital Bone Marrow Failure Syndrome
Session Description
Session Description
Speaker:
Francesca Fioredda
Longitudinal Evaluation of Clonal Hematopoiesis in Bone Marrow Failure and Primary Immunedysregulation Disorders: Single Center Preliminary Data
Friday, December 5, 2025, 3:05 p.m. - 6:00 p.m.
Organized in collaboration with the Society for Immunotherapy of Cancer (SITC)
Co-Chairs:
Marina Cavazzana, MD, PhD
Hopital Necker - Enfants Malades
Paris, France
[email protected]
Daniel Powell, PhD
University of Pennsylvania
Philadelphia, PA
[email protected]
Marco Ruella, MD
University of Pennsylvania
Philadelphia, PA
[email protected]
Overview:
This ASH-SITC joint workshop will explore how host factors — such as metabolism, diet, microbiome, genetics, aging, sex, obesity, and immune status — shape the efficacy and toxicity of cell and gene therapies across hematologic malignancies, solid tumors, and
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" href="https://www.hematology.org/classical-hematology" target="_blank">classical hematology disorders. The sessions will examine how these factors influence treatment response, durability, and adverse effects, with a focus on chimeric antigen receptor T-cell therapy, bispecific antibodies, and gene therapies. Discussions will address resistance mechanisms, toxicity mitigation, and strategies to optimize patient selection and therapeutic outcomes.
Target Audience:
Researchers, clinicians, regulatory experts, and industry stakeholders committed to overcoming barriers in clinical cell and gene therapy development for non-malignant and malignant hematologic disease.
Objectives:
- Discuss mechanisms by which baseline host factors influence cell manufacturing, vector entry, immune activation, and therapeutic efficacy.
- Examine strategies to mitigate immunotoxicity and genotoxicity, and screening protocols for genetic predispositions.
- Share insights into next-generation approaches that improve therapeutic efficacy.
- Explore innovative approaches to address variability in gene-modified cell performance and optimize approaches and procedures for better clinical outcome
Friday, December 5, 2025, 3:00 p.m. - 6:00 p.m.
Co-Chairs:
Lisa Baumann Kreuziger, MD
Versiti Blood Research Institute
Milwaukee, WI
[email protected]
Jeffrey Zwicker, MD
Memorial Sloan Kettering Cancer Center
New York, NY
[email protected]
Venous thromboembolism (VTE) is the second leading cause of death in patients with cancer, and cancer patients are the highest risk group for developing VTE. Despite considerable epidemiologic research, the mechanism of thrombosis in cancer is poorly understood. This workshop will provide a unique forum to discuss the mechanisms of thrombosis in hematologic malignancies, a topic that bridges malignant and non-malignant hematology within ASH.
Target Audience:
Basic scientists and researchers interested in the mechanisms of thrombosis in cancer patients 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. Early career and senior investigators will be encouraged to facilitate mentoring 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 Schedule
Session Description
Speaker:
Lisa Baumann Kreuziger, MD, MS
Versiti Medical Sciences Institute
OPENING REMARKS
Session Description
Speakers:
Lisa Baumann Kreuziger, MD, MS
Versiti Medical Sciences Institute
Moderator Introductions
Matthew Flick, PhD
UNC Blood Research Center
uPA/uPAR and tPA to the Pathogenesis of Pancreatic Cancer
Anthony Sloan, PhD
Cleveland Clinic
Sex Differences in PAR4 Signaling Influencing Glioblastoma Growth
Robert Flaumenhaft, MD
Harvard University
Panel Discussion/Q&A
Pathophysiology and Prediction of Thrombosis in Patients with Cancer
4:04 p.m. - 5:45 p.m., OCCC - W304EFGH
Session Description
Speakers:
Marina Marchetti, PhD
Department of Immunohematology and Transfusion Medicine, Hospital Papa Giovanni XXIII
Moderator Introductions
Keith McCrae, MD
Cleveland Clinic
Mechanisms of Immune Checkpoint Inhibitor Causing Thrombosis
Serena Lucotti, PhD
Weill Cornell Medicine
Role of Extracellular Vesicles and Lung Macrophages in CAT
Carolyn Haller Jr
P-Selectin Inhibition Reduces Thromboinflammation in a Pancreatic Cancer Xenograft Model
Saran Lotfollahzadeh, MD, MMSc
Boston University School of Medicine
Tryptophan Upregulation of Coagulation Factors Promotion Cancer Associated Thrombosis
Craig Horbinski
Predictors of VTE in Glioma
Alan Aberdeen, MS
Ground Truth Labs
Prediction of Thrombosis From Bone Marrow Pathology
Friday, December 5, 2025, 3:00 p.m. - 6:00 p.m.
Co-Chairs:
Aaron Schimmer, MD, PhD
Princess Margaret Cancer Centre
Toronto, Canada
[email protected]
Sarah Skuli, MD, PhD
University of Pennsylvania
Philadelphia, PA
[email protected]
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, including research in model organisms, as they relate to blood cancer and normal hematopoiesis.
- Translational research – Preclinical studies of novel mitochondrial and metabolic targets for the treatment of hematologic malignancies.
- Clinical research – Clinical trials of novel 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:
- Highlight fundamental discoveries in metabolism that shed light on clinically relevant problems in blood cancer.
- Compare and contrast mitochondrial pathways and metabolism between normal hematopoiesis and blood cancer.
- Identify targets in metabolism whose inhibition or activation would preferentially eradicate malignant cells.
Workshop Schedule
Session Description
Speakers:
Sarah Skuli, MD, PhD
University of Pennsylvania
Opening Remarks
Aaron Schimmer, MD PhD
Princess Margaret Cancer Centre, University of Toronto
Opening Remarks
Session Description
Speakers:
Maria Laura Amaya, MD PhD
University of Colorado
Moderator Introduction
Nicola Vannini, PhD
Ludwig Institute for Cancer Research Lausanne
The Metabolism of Immune Aging
Jose A Cancelas, MD, PhD
Dana-Farber Cancer Institute
Mitochondrial Dependencies of Normal Hematopoiesis
Maria Laura Amaya, MD PhD
University of Colorado
Panel Discussion/Q&A
Translational Research - Targeting Mitochondria and Metabolism in Cancer
4:05 p.m. - 5:05 p.m., OCCC - W414AB
Session Description
Speakers:
Chris Halsey, PhD
University of Glasgow
Moderator Introduction
Michaela Reagan, PhD
Tufts University,
MaineHealth
Obesity and Adipocytes in Multiple Myeloma from Biology to Therapy
Jeevisha Bajaj, PhD
University of Rochester Medical Center
Metabolic Vulnerabilities of Leukemic Stem Cells
Chris Halsey, PhD
University of Glasgow
Panel Discussion/Q&A
Session Description
Speakers:
Marina Konopleva, MD, PhD
Montefiore Einstein Comprehensive Cancer Center
Moderator Introduction
Takeshi Fujino, MD, PhD
Sloan Kettering Institute
Mitochondrial Transplantation For The Treatment of MDS
Ashkan Emadi, MD, PhD
Department of Medical Oncology, WVU School of Medicine WVU Cancer Institute
Cutting Glutamine, Halting Translation: A Metabolic Therapy in Blood Cancer
Marina Konopleva, MD, PhD
Montefiore Einstein Comprehensive Cancer Center
Panel Discussion/Q&A
Friday, December 5, 2025, 3:00 p.m. - 7:00 p.m.
Co-Chairs:
Patricia Ernst, PhD
University of Colorado Denver
[email protected]
Ulrich Steidl, MD, PhD
Albert Einstein College of Medicine
[email protected]
The workshop will cover the basic science of myeloid development or pathophysiology. While individual talks can and will have more translational components, there will be no presentations primarily focused on clinical strategies. The presentations will highlight tools and concepts from molecular and cellular biology, biochemistry, bioinformatics, animal models, and study of human samples to address key topics in myeloid biology. Talks will feature emerging data from cutting-edge research groups at all stages expected to have a broad and lasting impact on the field.
Active discussion is encouraged, and participants are expected to present and discuss the latest unpublished advances in normal and malignant myeloid biology.
This workshop will only be available to people who attend the annual meeting in person and will not be live streamed to a virtual audience. A recording of the workshop will be available at the conclusion of the workshop on the virtual meeting platform until 11:59 p.m., Pacific time, on January 1.
Target Audience:
Laboratory-based and translational investigators, including trainees, with many interests surrounding normal and pathological myeloid/stem cell biology.
Objectives:
- Provide a venue for early-career and established investigators to present their newest cutting-edge science to the myeloid community.
- Offer an opportunity for informal discussion and feature late-breaking science. Provide trainees with a chance to hear new scientific concepts and to meet leaders in our field.
- Highlight emerging new concepts in myeloid biology that have the potential to broadly impact hematology.
Workshop Schedule
Session Description
Speaker:
Patricia Ernst, PhD
University of Colorado Medical School
OPENING REMARKS
Session Description
Speakers:
Eric Pietras, PhD
University of Colorado
Moderator Introduction
Sandra Pinho, PhD
University of Illinois Chicago
Megakaryocyte-Derived PF4 as a Dual Regulator of Hematopoietic and Leukemic Stem Cells
Session Description
Speaker:
Kathrin Bernt
Moderator Introduction
Session Description
Speakers:
Olga A Guryanova, MD, PhD
University of Florida Health Cancer Institute
Clonal Hematopoiesis-Related Loss of Dnmt3a Deregulates Trained Immunity to Alter Cancer Control
John Crispino Sr, PhD
St. Jude Children's Research Hospital
Cohesin mutations suppress HLA Class II gene expression in the myeloid leukemia of Down syndrome
Friday, December 5, 2025, 3:00 p.m. - 6:00 p.m.
Co-Chairs:
Pamela S. Becker, MD, PhD
City of Hope
Duarte, CA
[email protected]
Jun J. Yang, PhD
St. Jude Children’s Research Hospital
Memphis, TN
[email protected]
Initially, personalized medicine was solely focused on genomics, and patients in large trials were treated based on individual mutations. However, success was limited, likely because of the presence of multiple mutations and innate or adaptive drug resistance. In a global effort, investigators have developed sophisticated functional assays for testing drug sensitivity, and these advances have led to predictive algorithms for patient response. Combining the data from extensive genomic and functional analysis holds great promise for expanding the reach of precision medicine for blood cancers. This approach has been particularly useful for patients with primary refractory or multiply relapsed disease, or rare conditions for which there are less data to drive treatment selection. The workshop will cover aspects of resistance to therapy in blood disorders, focusing on non-genetic molecular mechanisms such as epigenetic, epitranscriptomic, and metabolic aspects and their potential interaction with genetic alteration. Platforms allowing the comparison between diagnosis and relapsed samples, or relapse-promoting mechanisms existing at diagnosis and potential therapeutic intervention, will be discussed.
Given these rapid advances in genomic and functional precision medicine, with remarkable recent achievement of clinical predictability demonstrated in ongoing clinical trials, this workshop will address the promise and continued challenges of a fast-growing field, both technically and scientifically.
Target Audience:
Laboratory scientists, translational researchers and clinical investigators studying drug therapy and target identification in hematologic malignancies and interested in novel approaches to personalized medicine.
Objectives:
- Educate participants on the rationale for combined genomic and functional precision medicine (especially in contrast to the genomics-centric paradigm), the methodology for drug sensitivity phenotyping for both inter- and intra-leukemia heterogeneity, and the clinical interpretation of ex vivo drug response.
- Describe computational biology and systems biology approaches used for data analysis and correlations of functional data with the results of multi-omic profiles to predict response.
- Discuss clonal evolution of cancers and how it impacts and informs drug sequencing, dosing, and resistance.
- Provide a platform for robust discussion on the value of the combination of genomic and functional precision medicine in blood cancers.
Workshop Schedule
Session Description
Speaker:
Pamela Sue Becker, MD,PhD
City of Hope
OPENING REMARKS
Session Description
Speakers:
Jun J Yang, PhD
St. Jude Campus
Moderator Introduction
Tea Pemovska, PhD
Medical University of Vienna
Rethinking How We Treat Disease Through High-Throughput Drug Response Phenotyping
Heikki Kuusanmäki, PhD
University of Helsinki
High Throughput Multiparameter Flow Cytometry and Intra-Leukemia Heterogeneity in Drug Response
Patrick Bhola, PhD
Dana-Farber Cancer Institute
Apoptosis-Priming as a Measurement of Drug Sensitivity
Tea Pemovska, PhD
Medical University of Vienna
Panel Discussion: Can We Harmonize Drug Response Criteria
Session Description
Speakers:
Mika Kontro, MD, PhD
Helsinki University Hospital
Moderator Introduction
Guangrong Qin, PhD
Institute for Systems Biology
Systems Biology and Machine Learning to Predict Response
Timothy Ley, MD
Washington University in St. Louis
AML Mutation Clearance after Induction: an Integrated Genomic Approach to Define Subclonal Responses to Therapy
Mika Kontro, MD, PhD
Helsinki University Hospital
Panel Discussion: Integration of Functional and Genomic Precision Medicine, are we there yet?
Friday, December 5, 2025, 3:00 p.m. - 6:00 p.m.
Co-Chairs:
Panagiotis Ntziachristos, PhD
Ghent University
Ghent, Belgium
[email protected]
Christina Glytsou, PhD
Rutgers University
Piscataway, NJ
[email protected]
Daniel Starczynowski, PhD
Cincinnati Children’s Hospital Medical Center
Cincinnati, OH
[email protected]
Resistance to therapy poses a significant obstacle to the successful treatment of blood disorders. Despite advances in targeted therapies, the underlying mechanisms driving therapy resistance — both preexisting and acquired — are not fully understood. This workshop will address this critical challenge by exploring the complex molecular landscape that contributes to drug resistance in hematological malignancies. This workshop will bring together leading scientists and clinicians to discuss the latest updates in both genetic and non-genetic molecular mechanisms, including epigenetic, epitranscriptomic, translational, post-translation, and metabolic aspects, as well as their potential interaction. Cellautonomous or non-cell-autonomous mechanisms, as well as the dynamic interplay between tumor cells and their microenvironment, will be explored.
The workshop will also have a special focus on emerging model systems and machine-learning-based computational technologies that are revolutionizing our ability to monitor, predict, and reverse therapy resistance. Participants will gain a comprehensive understanding of the complexities of drug 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.
- Integrate various mechanisms of resistance.
- Provide a forum of open discussion and exchange of ideas that will identify future research priorities towards the development of solutions to overcome therapy relapse.
- Promote interdisciplinary collaboration and exchange of technologies.
Workshop Schedule
Session Description
Speaker:
Panagiotis Ntziachristos, PhD
Ghent University
OPENING REMARKS
Session Description
Speakers:
Panagiotis Ntziachristos, PhD
Ghent University
Moderator Introduction
Kimberly Stegmaier, MD
Dana-Farber Cancer Institute
Identification of New Vulnerabilities in Therapy Resistance AML
Taishi Yonezawa, PhD
Baylor College of Medicine (BCM)
Targeting Post-Translational Regulation of DNMT3A Offers a Promising Therapy for Clonal Hematopoiesis and Hematologic Malignancies
Aristotelis Tsirigos, PhD
NYU School of Medicine
Dissecting the Regulatory Landscape of Leukemias with Multi-Modal AI Models of Gene Regulation
Panagiotis Ntziachristos, PhD
Ghent University
Panel Discussion/Q&A
Session Description
Speakers:
Lili Wang
Moderator Introduction
Benson M. George
Identification and Therapeutic Targeting of Cell Surface RNA-Binding Proteins
Lili Wang
Splicing Control in Leukemia and Beyond
Lili Wang
Panel Discussion/Q&A
Session Description
Speakers:
Daniel Starczynowski, PhD
Cincinnati Children's Hospital
Moderator Introduction
Anastasia Tikhonova, PhD
University Health Network
Immune Microenvironment Defining Responses of Lymphoblastic Leukemia
Theodoros Karantanos, MD, PhD
Johns Hopkins University
Inflammatory Pathways Foster Treatment Resistance in Myeloid Neoplasms
Quinlan Sievers, MD, PhD
Memorial Sloan Kettering
Molecular and Structural Basis of Pan-Resistance to BTK Targeting Therapies via BTK A428D Mutation
Daniel Starczynowski, PhD
Cincinnati Children's Hospital
Panel Discussion/Q&A
Session Description
Speakers:
Shruti Bhatt, PhD
National University of Singapore, Department of Pharmacy and Pharmaceutical Sciences
Moderator Introduction
Quinlan Sievers, MD, PhD
Memorial Sloan Kettering
Targeting Macrophage Metabolism to Overcome Chemoresistance in Myeloid Leukemia
Christina Mayerhofer
OGFOD1 enables AML chemo- and nutrient stress resistance by regulating protein synthesis
Paolo Gallipoli, MD
Queen Mary University of London
Adipocytic niche protects AML cells from FLT3 inhibitor therapy by activating the PI3 kinase pathway and rescuing their central carbon metabolism
Shruti Bhatt, PhD
National University of Singapore, Department of Pharmacy and Pharmaceutical Sciences
Panel Discussion/Q&A
Friday, December 5, 2025, 3:00 p.m. - 6:00 p.m.
Piers Blombery, PhD, MBBS
Peter MacCallum Cancer Centre
Melbourne, VIC, Australia
[email protected]
Torsten Haferlach, MD, PhD
Munich Leukemia Laboratory
Munich, Germany
[email protected]
Ella Thompson, PhD
Peter MacCallum Cancer Centre
Melbourne, VIC, Australia
[email protected]
Wencke Walter, PhD
Munich Leukemia Laboratory
Munich, Germany
[email protected]
Measurable residual disease (MRD) detection is rapidly becoming a central paradigm to direct the management of patients with hematological malignancy as evidenced by (i) clinical trials explicitly using MRD to direct therapeutic arms, (ii) international groups discussing the standardization and measurement of MRD, and (iii) the recognition of MRD as a surrogate endpoint.
This workshop will focus on technological advancements, development, and delivery of MRD in the clinical diagnostic setting.
Target Audience:
Hematologists, oncologists, hematopathologists, clinical investigators (particularly those involved in clinical trial design), computational biologists/bioinformaticians, and medical/laboratory scientists.
Objectives:
- Present recent advancements in MRD/circulating tumor DNA (ctDNA) detection platforms, including novel approaches with a focus on diagnostic and practical implementation as well as their use in clinical trials.
- Foster collaboration between pathologists, clinicians, and laboratory scientists to advance the translational implementation of novel MRD/ctDNA diagnostics.
- Support current standardization efforts by facilitating discussion around ongoing efforts by international consortia and regulatory bodies to standardize MRD/ctDNA measurement, reporting, and clinical thresholds.
Workshop Schedule
Session Description
Speaker:
Torsten Haferlach, PhD
MLL Munich Leukemia Laboratory
OPENING REMARKS
Session Description
Speakers:
Wencke Walter, PhD
MLL Munich Leukemia Laboratory
Moderator Introduction
Reid W Merryman, MD
Dana-Farber Cancer Institute
Personalized Whole-Genome Sequencing (WGS)-Based ctDNA Testing for B-cell Lymphoma
Daniel Hodson, MD, PhD
Cambridge Stem Cell Institute
The UK DIRECT Study; a Roadmap for ctDNA in Aggressive B-Cell Lymphoma
Torsten Haferlach, PhD
MLL Munich Leukemia Laboratory
Panel - Interactive ctDNA MRD Case Studies in B-Cell Lymphoma
New Developments in Measurable Residual Disease for Patients with Acute Myeloid Leukemia
4:38 p.m. - 5:57 p.m., OCCC - W311ABCD
Session Description
Speakers:
Ella Thompson, PhD
Peter MacCallum Cancer Centre
Moderator Introduction
Laura Dillon, PhD
Virginia Tech FBRI Cancer Research Center
Measurable Residual FLT3 Internal Tandem Duplication in Acute Myeloid Leukemia
Linde A Miles, PhD
Cincinnati Children's Hospital Medical Center
Single Cell Proteogenomics for AML MRD?
R. Coleman Lindsley, MD, PhD
Dana-Farber Cancer Institute
Integrating Serial Molecular Quantification in MDS/AML
Sun Loo, MD
Peter MacCallum Cancer Centre and Royal Melbourne Hospital
Clinical Application of MRD-Directed Therapy
Ella Thompson, PhD
Peter MacCallum Cancer Centre
Panel - Interactive Case Studies in AML MRD