Scientific Workshops

Session Description

Opening Remarks

Chair:

Paolo Ghia, MD, PhD

Session Description

This session will examine genetic and non-genetic mechanisms of resistance to targeted therapies in CLL, including non-covalent and next-generation BTK inhibitors and BCL2 inhibitors. The discussion will connect mechanistic findings with emerging translational strategies to anticipate and overcome resistance.

Moderator:

José Ángel Hernández-Rivas, MD, PhD
Hospital Universitario Infanta Leonor and Universidad Complutense de Madrid

Speakers:

José Ángel Hernández-Rivas, MD, PhD
Hospital Universitario Infanta Leonor and Universidad Complutense de Madrid
Moderator Introduction

Jennifer R. Brown, MD, PhD
Dana-Farber Cancer Institute and Harvard Medical School
Genetic Mechanisms of Resistance to Non-Covalent BTK Inhibitors

Justin Taylor, MD
Sylvester Comprehensive Cancer Center, University of Miami
Emerging Mechanisms of Resistance to Novel BTK-Targeted Therapies

Adalgisa Condoluci, MD, PhD
Institute of Oncology Research (IOR) and Oncology Institute of Southern Switzerland (IOSI)
Non-Genetic Mechanisms of Resistance to BTK Inhibitors

John F. Seymour, MBBS, PhD
Peter MacCallum Cancer Centre and Royal Melbourne Hospital
Mechanisms of Resistance to BCL2 Inhibitors

Session Description

This session will explore clonal evolution from monoclonal B-cell lymphocytosis to CLL and Richter transformation, including multiclonality and molecular models of transformation. It will examine how cell-intrinsic and cell-extrinsic forces shape evolutionary trajectories and disease progression.

Moderator:

Davide Rossi, MD
Laboratory of Experimental Hematology, Institute of Oncology Research

Speakers:

Davide Rossi, MD
Laboratory of Experimental Hematology, Institute of Oncology Research
Moderator Introduction

Susan L. Slager, PhD
Mayo Clinic
The Path from Monoclonal B-Cell Lymphocytosis to CLL

Ian Márquez-López, MSc, PhD Candidate
Fundació de Recerca Clínic Barcelona–Institut d’Investigacions Biomèdiques August Pi i Sunyer (FRCB-IDIBAPS)
Multiclonality in CLL: Early Clonal Trajectories in CLL and Richter Transformation

Elisa Ten Hacken, PhD
Weill Cornell Medical College, Weill Cornell Medicine
Modeling Richter Transformation at the Molecular Level

Adrian Weistner, MD, PhD
National Heart, Lung, and Blood Institute, National Institutes of Health
Clonal Evolution in CLL: Cell-Intrinsic or Cell-Extrinsic Mechanisms?

Session Description

This session will review current MRD assessment approaches, genomic correlates, multidimensional analysis, and MRD kinetics. It will evaluate how MRD can be used to detect and monitor clonal evolution, interpret treatment response, and guide translational monitoring strategies.

Moderator:

Ferran Nadeu, PhD
FRCB-IDIBAPS and CIBERONC

Speakers:

Ferran Nadeu, PhD
FRCB-IDIBAPS and CIBERONC
Moderator Introduction

Anton Langerak, PhD
Erasmus MC, University Medical Center
Approaches to MRD Assessment

Othman Al-Sawaf, MD, PhD
University Hospital Cologne, University of Cologne
The Genomic Background of Clonal Evolution: Evidence from Clinical Trials

Catherine Wu, MD
Dana-Farber Cancer Institute and Harvard Medical School
Multidimensional Analysis of MRD

Andy Rawstron, PhD
HMDS, Leeds Teaching Hospitals NHS Trust
MRD Kinetics and Clinical Outcomes

Session Description

Closing Remarks

Chair:

Martin Antonio Cabero Becerra, MD, PhD

Session Description

Opening Remarks

Chair:

Sanjay Patel, MD, PhD

Session Description

This session will discuss recent advances in spatial biology - technical and practical – that may bring such approaches into the clinical lab setting in the near future.

Moderator:

Sanjay Patel, MD, PhD
Weill Cornell Medical College/New York-Presbyterian Hospital

Speakers:

Sanjay Patel, MD, PhD
Weill Cornell Medical College/New York-Presbyterian Hospital
Moderator Introduction

Scott Rodig, MD, PhD
Beth Israel Deaconess Medical Center/Dana-Farber Cancer Institute
Spatial Proteomic and Transcriptomic Approaches for the Evaluation of Lymphoma

Andrea Radtke, PhD
Leica Microsystems
From Tissue Architecture to Risk Stratification in Follicular Lymphoma: What It Takes to Build a Trustworthy Panel

Sanjay Patel, MD, PhD
Weill Cornell Medical College/New York-Presbyterian Hospital
A Clinically-Deployable Low-Plex Spatial Phenotyping Approach for Dissecting Altered Marrow Microarchitecture in MDS

Session Description

This session will discuss the impact of full spectrum flow and novel analytic approaches on flow cytometry for the evaluation of hematologic malignancies.

Moderator:

Christopher Y Park, MD,PhD
NYU Grossman School of Medicine

Speakers:

Christopher Y Park, MD,PhD
NYU Grossman School of Medicine
Moderator Introduction

Mohamed Sharaf, MD
NYU Grossman School of Medicine
Value of Foundation Models in Clinical Flow Cytometry

Jean Oak, MD, PhD
Stanford Hospital and Clinics
Assessing Antigen Density By Flow Cytometry Predicts Clinical Outcomes in CAR-T Treated Patients

David Ng, MD
ARUP
Clinical Deployment of Machine Learning Approaches in Flow Cytometry

Session Description

Break

Session Description

This session will cover recent technological and clinical workflow advances that may impact the clinical molecular lab.

Moderator:

Gerlinde Wernig, MD
Stanford Hospital and Clinics

Speakers:

Gerlinde Wernig, MD
Stanford Hospital and Clinics
Moderator Introduction

Gabriel Griffin, MD
Dana Farber Cancer Institute
Rapid Long-Read Sequencing for Acute Leukemia Diagnosis

Russell Ryan, MD
University of Michigan
Next Generation Chromatin Structural Assessments: Ready for Primetime?

Anna Nam, MD
Weill-Cornell Medicine
Single-Cell Approaches on the Horizon in the Molecular Lab

Session Description

This session will cover recent advances in machine learning approaches to diagnose hematologic malignancies using digital images of tissue biopsy and blood/aspirate smear slides.

Moderator:

Daniel Royston, DPhil, FRCPath, MBBChir
University of Oxford

Speakers:

Daniel Royston, DPhil, FRCPath, MBBChir
University of Oxford
Moderator Introduction

Gerlinde Wernig, MD
Stanford Hospital and Clinics
Machine Learning–Integrated Spatial Transcriptomics and Proteomics in Lymphoma Diagnostics

Daniel Royston, DPhil, FRCPath, MBBChir
University of Oxford
Digital Pathology Bone Marrow Biopsy Evaluation for the Diagnosis of Myeloid Malignancies

Jan Moritz Middeke, MD
University Hospital Dresden
AI-Assisted Bone Marrow Diagnostics: From Automated Myelograms to Digital Biomarkers

Session Description

Closing Remarks

Chair:

Christopher Y Park, MD,PhD

Session Description

Opening Remarks

Chair:

Lucy C. Fox, MBBS BCom/BSc DMedSci FRACP FRCPA

Session Description

This overarching session will explore newly characterized germline predisposition disorders and the mechanisms by which deleterious germline variants drive abnormal hematopoiesis, bone marrow failure, and progression to malignancy. Talks will integrate genetic, biochemical, cellular, and model-organism approaches to illuminate disease pathophysiology and identify shared and disorder-specific vulnerabilities that may guide future therapeutic strategies.

Moderator:

Sushree Sahoo, PhD
Fralin Biomedical Research Institute at VTC, Virginia Tech

Speakers:

Sushree Sahoo, PhD
Fralin Biomedical Research Institute at VTC, Virginia Tech
Moderator Introduction

Roshina Thapa, PhD
St Jude Children's Research Hospital
Dissecting the Role of ETV6 in Human Blood Cell Differentiation and Transcriptional Regulation

Cheryl van de Wetering, PhD
St. Anna Children's Cancer Research Institute
POP5 Regulates Human T Cell Function and Differentiation Via Transfer RNA Processing and Telomere Maintenance

Patrycja Fryzik, MD, PhD
Princess Maxima Center for Pediatric Oncology
Spatially-Resolved Single-Cell Profiling Identifies Distinct Quiescent and Cycling Hematopoietic Niches in Healthy and Failing Pediatric Bone Marrow

Arantxa Carrasco-Leon, PhD
Barts Cancer Institute, Queen Mary University of London
Integrative Functional and Transcriptomic Profiling Identifies Biomarkers for the Interpretation of Germline DDX41 Variants

Francisco Beas, MD
Vall Hebron Institute of Oncology
Germline-Tumor Telomere Dynamics via Whole Genome Sequencing in Early-Onset myelodysplastic syndromes: Identifying a Subgroup with Paradoxical Somatic Elongation on a Short Germline Background

Inci Aydemir, MD
Northwestern University
Single-cell and Spatial Profiling of human iPSC-Derived Bone Marrow Organoids Reveals Genetic Epistasis Between DDX41 and CHEK2 in Niche Remodeling

Serine Avagyan, MD, PhD
University of California
Transcriptional Dynamics of Monosomy 7 Clonal Evolution in Germline SAMD9L Syndrome

Session Description

This focused session will examine how precision genomic approaches, including gene therapy, gene editing, and small molecules, are being developed and applied for germline HM/BMF disorders. Talks will cover preclinical studies and emerging clinical experience, and will highlight the unique considerations of delivering precision therapies to patients with germline disorders.

Moderators:

Deena Iskander, MD, PhD
Imperial College London

Kristen Schratz, MD

Speakers:

Deena Iskander, MD, PhD
Imperial College London
Moderator Introduction

Noah Saito, BS
Cleveland Clinic and Case Western Reserve University
From Yeast to Human: Conserved p38/ISR Dysregulation as a TP53-Independent Route to Leukemia in SDS

Jason N Berman, MD
CHEO Research Institute
Repurposing Cholesterol and MEK Inhibitors for the Treatment of Bone Marrow Failure in Dyskeratosis Congenita: Preclinical Evidence from a Zebrafish Model of Parn Deficiency

Robert Rowe, MD, PhD
Boston Children's Hospital
Survey of Clonal Hematopoiesis Reveals Augmentation of ALDH9A1 as a Therapeutic Strategy in Fanconi Anemia

Shruthi Suryaprakash, MD
St. Jude Children's Research Hospital
Preclinical Development of Lentiviral Gene Therapy for RPS19-mutated Diamond-Blackfan Anemia Syndrome

Brittany Stewart, CGC, PhD
Cleveland Clinic
Building Consensus for the Management of RUNX1 Familial Platelet Disorder: Results from an International Modified-Delphi Survey

Karishma Rupani, MD, MMSc
Icahn School of Medicine at Mount Sinai
Severe Post-Transplant Bone Marrow Failure Driven by a Novel TERT TRBD Domain Variant (p.Gln340His) in Short Telomere Syndrome

Session Description

Break

Session Description

This focused session will discuss recent advances in the molecular surveillance of individuals carrying germline predisposition variants. Talks will address the application of high-sensitivity sequencing and clonal-tracking technologies, the natural history and clinical significance of clonal hematopoiesis arising on a germline-predisposed background, and emerging frameworks for using molecular data to guide pre-emptive intervention and stratify malignancy risk.

Moderators:

Felicia Andresen, MD
Boston Children's Hospital

Ing-Soo Tiong, MBBS, FRACP, FRCPA, PhD
Peter MacCallum Cancer Centre

Speakers:

Felicia Andresen, MD
Boston Children’s Hospital
Moderator Introduction

Olisaemeka Ogbue, MBBS
Mayo Clinic
Genome-First Characterization of Germline Telomere Biology Disorder Variants Identifies Gene-Specific Clonal Hematopoiesis and Malignancy Risk to Inform Surveillance

Abhishek Avinash Mangaonkar, MBBS
Mayo Clinic
PTBP1 Upregulation and Selective ATM-ATR Signaling Support SF3B1-Mutant Clonal Fitness in a Short Telomere Syndrome microenvironment

Ira Lignugaris Kraft, MD
National Institutes of Health
A Droplet Digital PCR Assay for DDX41 c.1574G>A (p.R525H) Enables Early Detection and Measurable Residual Disease Assessment in DDX41-Associated Neoplasms

Aaron N. Cheng, MD
Hospital of the University of Pennsylvania
Natural History of Myeloid Neoplasms in Telomere Biology Disorders Guides Optimal Bone Marrow Surveillance Strategies: The SAFE-TBD Study

Nandini Rajaram Siva, MD
Cleveland Clinic Foundation
Biological Aging Licenses Biallelic Transformation in Germline DDX41 Predisposition: Telomere Content and Epigenetic Clocks as Determinants of Myeloid Progression

Eunice Sindhuvi Edison, PhD
Christian Medical College
Next Generation Sequencing in Bone Marrow Failure Cases have a high Diagnostic Yield but Limited Prognostic Value for Treatment Outcomes in India

Ferran Balbastre-Úbeda, MS
Vall d'Hebron Institute of Oncology
Hair Bulb and Oral Mucosa DNA for Prompt Germline Testing in Hematologic Neoplasms

Session Description

This overarching session will highlight new scientific discoveries in germline predisposition to HMs and BMF, including newly recognized disease genes and syndromes, novel insights in established disorders, and new knowledge from variant curation efforts and guideline groups.

Moderators:

Harry Layton Lesmana, MD
Cleveland Clinic

Sara Torres-Esquius, CGC
Vall d’Hebron Hospital Universitari

Speakers:

Harry Layton Lesmana, MD
Cleveland Clinic
Moderator Introduction

Yirun Miao, PhD
St. Anna Children's Cancer Research Institute
Identification of a Novel DBAS-Like Congenital Bone Marrow Failure Syndrome Caused By Germline Biallelic Mutations in GRWD1

William Stevenson, MBBS, PhD
Royal North Shore Hospital
Structural Variant Involving ANKRD26 Causes Leukaemia Predisposition in a Family with Severe Inherited Thrombocytopenia and is Associated with Expression of Alternate mRNA and Protein Isoforms

Wolfgang Novak, MD
St. Anna Children’s Hospital
A Recurrent Biallelic POLE Genotype: Dyserythropoiesis, Stress-Induced Marrow Hypocellularity and EBV Susceptibility.

Patrycja Dubielecka, PhD
Brown University
Stem/Progenitor Cell Compartment-Specific Enrichment of Recurrent Rare Genome-Maintenance Variants in Myeloproliferative Neoplasms

Panteha Behboodi, PhD
Hackensack Meridian Health
Directionality of Gains of Allele-Specific DNA Methylation at Germline Risk Loci for Multiple Myeloma

Xin Jiang, PhD
Boston Children's Hospital, Harvard Medical School
Patient-Derived Neomorphic SEPTIN6 Mutations Induce Hematopoietic Stem Cell Senescence and Disrupt Nuclear Actin–Chromatin Organization in Myelodysplastic Syndrome

James Stewart Blachly, MD
The Ohio State University
Germline Balanced Translocation t(6;21) as a Novel Mediator of RUNX1-Familial Platelet Disorder

Session Description

Closing Remarks

Chair:

Marcin W Wlodarski, MD, PhD

Session Description

Chair:

Jeffrey Zwicker, MD

Session Description

This session examines the multifaceted biology of cancer-associated thrombosis, spanning immune checkpoint blockade, extracellular vesicles, tumor–vascular interactions, and metabolic drivers of coagulation. Speakers will highlight experimental and clinical insights into how the tumor primes a prothrombotic state in cancer.

Moderators:

Marc Carrier, MD, MSc, FRCPC
Ottawa Health Research Institute

Lisa Baumann Kreuziger, MD, MS

Speakers:

Marc Carrier, MD, MSc, FRCPC
Ottawa Health Research Institute
Moderator Introduction

Rushad Patell, MD
Beth Israel Deaconess Medical Center
Targeting Extracellular Vesicles with Rosuvastatin

Thomas Francis Fusillo, DO, MS, MHA
Memorial Sloan Kettering Cancer Center
Non-Driver Mutations and Thrombosis in MPN

Danielle Roy, PhD
University of Ottawa
Proteomic and Genomic Predictors of Thrombosis or Bleeding in Cancer

Puneeth Iyengar, MD, PhD
Louisiana State University
Linking Cachexia and Thrombosis in Cancer

Florian Moik, MD, PhD
Medical University of Graz
Inflammatory Markers to Predict ICI-Mediated Thrombosis

Session Description

Break

Session Description

Summary: This session explores how coagulation and fibrinolytic pathways drive cancer biology beyond hemostasis, shaping tumor progression, immune responses, and the tumor microenvironment. By integrating insights from pancreatic cancer, the presentations highlight emerging mechanisms and therapeutic opportunities at the intersection of coagulation and oncology.

Moderator:

Jeffrey Zwicker, MD
Memorial Sloan Kettering Cancer Center

Speakers:

Jeffrey Zwicker, MD
Memorial Sloan Kettering Cancer Center
Moderator Introduction

Betül Ünlü, PhD
Royal College of Surgeons in Ireland
VWF Mediates Pancreatic Cancer Progression

Melissa Fishel, PhD
University of Indiana
Targeting Fibrinogen to Suppress Pancreatic Cancer

Taslim Al-Hilal, PhD
University of Utah
Stromal Fibrin and PDA infiltration

Grigorios Gerotziafas, MD, PhD
Sorbonne Université
Fibrin Shields in Cancer-Associated Thrombosis

Session Description

Closing Remarks

Chair:

Lisa Baumann Kreuziger, MD, MS

Session Description

Sickle Cell Disease (SCD) is a complex monogenic disorder characterized by vast clinical heterogeneity. The diversity of clinical phenotypes is likely due to underlying heterogeneity in the underlying pathophysiological cascades downstream of sickle Hb polymerization, such as chronic hemolysis, vaso-occlusion, and systemic inflammation. Traditional hematological markers often fail to capture the full spectrum of individual disease trajectories.?Mechanistic biomarkers derived from the core biology of SCD are essential for diagnostic, prognostic, and therapeutic insights. Embedded in disease pathogenesis, they reflect precise, target-driven interactions rather than just correlations. By monitoring these mechanistic markers, the clinical evolution and progression of specific complications can be anticipated, guiding patient stratification, clinical decisions, drug development, and predicting response to therapies. In this workshop, we aim to build on the 2025 workshop on disease severity scoring and clinical trial endpoints by focusing on recent advances in mechanism-based biomarker development. Participants will gain insights into future research directions in this mechanistically complex disease and establish opportunities for collaboration that will drive forward ASH 2025 SCD Research Priorities agenda on biomarker identification for individualized medicine.

Chair:

Karina Yazdanbakhsh, PhD

Session Description

This session will focus on cell-based alterations contributing to SCD pathophysiology, specifically role of autonomic vascular dysfunction along with the basis of mechanistic drivers in red cells, white cells and vascular endothelium that offer a high-resolution view of disease pathogenesis may predict vaso-occlusion and organ damage, guide personalized clinical interventions, and monitor therapeutic responses.

Moderator:

Thomas D. Coates, MD
Children's Hospital Los Angeles

Speakers:

Thomas D. Coates, MD
Children’s Hospital Los Angeles
Moderator Introduction

Saranya Veluswamy, MD
Keck School of Medicine of USC
Neuromediated Vasoconstriction

Karina Yazdanbakhsh, PhD
New York Blood Center Enterprises
Patrolling Monocytes and VOCs

Thomas D'Humières, MD
Henri Mondor Hospital, Assistance Publique Hôpitaux de Paris (AP-HP)
Cardiac Remodeling, Diastolic Dysfunction and Arrhythmia

Session Description

Break

Session Description

The second session will focus on the mechanisms driving complement dysregulation and thromboinflammation in SCD, in predicting vaso-occlusion and organ damage, and also in guiding clinical interventions, and monitoring therapeutic responses.

Moderator:

Arun Shet, MD, PhD
NHLBI

Speakers:

Arun Shet, MD, PhD
National Heart, Lung, and Blood Institute
Moderator Introduction

Lucia De Franceschi, MD
University of Verona
Complement Activation in Sickle Cell Disease

Erica Sparkenbaugh, PhD
UNC Chapel Hill
PAR1 and Thromboinflammation in Sickle Cell Disease

Wilbur A. Lam, MD, PhD
Emory University
Microvascular Occlusion, Thrombosis and Clot Resolution in Sickle Cell Disease

Session Description

Break

Session Description

The third area of discussion will address genetic modifiers?that?delineate?the?mechanistic pathways?underlying specific clinical phenotypes facilitates the identification of risk-conferring genotypes, enabling?stratified clinical interventions?that match specific pathophysiological profiles with optimal?disease-modifying treatments. It will also investigate the emerging landscape of multi-omic biomarkers, including proteomics and metabolomics that can provide a potentially interactive view of the real-time molecular mechanisms driving SCD severity, and the dynamic -omic signatures that can monitor disease activity and assessment of novel therapies.

Moderator:

Swee Lay Thein, MD
National Heart, Lung and Blood Institutes

Speakers:

Swee Lay Thein, MD
National Heart, Lung and Blood Institutes
Moderator Introduction

Julie Makani, MD, PhD
Muhimbili University
Genetic Modifiers of HbF and F Cells

Francesca Cendali, PhD, BS
University of Colorado
Cell-Based Metabolomic Signatures of Pulmonary Hypertension

Guillaume Lettre, PhD
University of Montreal
Plasma Proteomics and Disease Severity

Session Description

Break

Session Description

Moderator:

Karina Yazdanbakhsh, PhD

Session Description

This workshop will end with an introduction to Part 2 of this session, SCD Special Interest Session occurring on Monday, December 14, 2026, 4:30-6:00 p.m. Central Time., to examine gaps in endpoint development, trial design, and precision medicine, and to identify areas where greater alignment across researchers, clinicians, regulators, and patient communities may accelerate progress in SCD clinical research.

Chair:

Thomas D. Coates, MD

Session Description

Opening Remarks

Chair:

Marina Konopleva, MD, PhD

Session Description

Moderator:

Kelsey Fisher-Wellman, PhD
Wake Forest University

Speakers:

Kelsey Fisher-Wellman, PhD
Wake Forest University
Moderator Introduction

Tobias Berg, PhD
McMaster University
Epigenetic Regulation of Metabolism

Luisa Cimmino, PhD
University of Miami
Micronutrient Regulation of Mitochondrial Metabolism and Cell Fate in Myeloid Malignancies

Dorian Forte, PhD
University of Bologna
Extracellular Vesicles Reprogram Metabolism in the Bone Marrow Tumor Microenvironment

Session Description

This session will highlight new mitochondrial and metabolic vulnerabilities in blood cancer and how they vulnerabilities might be targeted with new therapies.

Moderator:

Natalia Baran, MD, PhD, MSc
University of Bern

Speakers:

Natalia Baran, MD, PhD, MSc
University of Bern
Moderator Introduction

Robert Orlowski, MD, PhD
MD Anderson Cancer Center
Novel Metabolic Dependencies as Therapeutic Targets in Multiple Myeloma

Versha Banerji, MD
University of Manitoba
Exploiting NAD Metabolism in CLL: Preclinical Advances in NAMPT Inhibition

Tim Triche, PhD
Van Andel Institute
Single-Cell Metabolic Profiling of MRD and Disease Progression in Multiple Myeloma

Session Description

Break/Networking/Interaction

Session Description

This session will discuss mitochondrial and metabolic pathways the tumor microenvironment and the subsequent impact on blood cancer cells.

Moderator:

Sarah Skuli, MD, PhD
University of Cincinnati

Speakers:

Sarah Skuli, MD, PhD
University of Cincinnati
Moderator Introduction

Stavroula Kousteni, PhD
Columbia University
Metabolic Rewiring of the Pre-Leukemic Bone Marrow Niche Promotes AML Progression

Marco Ruella, MD
University of Pennsylvania
Ketogenic Modulation of the Lymphoma Microenvironment to Improve CAR-T Cell Efficacy

Johanna Chiche, PhD
INSERM, Mediterranean Center for Molecular Medicine Research
Targeting Host and Tumoral Arginine Metabolism in B-Cell Lymphomas

Kai Tan, PhD
Children’s Hospital of Philadelphia
Optimizing Single Cell Spatial Transcriptomics to Evaluate the Bone Marrow Tumor Microenvironment

Session Description

This session will discuss early phase clinical trials of novel agents targeting mitochondrial and metabolic pathways in patients with blood cancer.

Moderator:

Michael Andreeff, MD, PhD
MD Anderson Cancer Center

Speakers:

Michael Andreeff, MD, PhD
MD Anderson Cancer Center
Moderator Introduction

John Byrd, MD
University of Pittsburgh Medical Center, Hillman Cancer Center
Navigating Academic Drug Development: Lessons From a Novel DHODH Inhibitor for AML

Alexandra McLean Stevens, MD PhD
Texas Children's Hospital
Anti-Infective by Indication, Anti-Leukemic by Design: Repurposing Atovaquone to Target OXPHOS in Pediatric AML

Session Description

Closing Remarks

Chair:

Marina Konopleva, MD, PhD

Session Description

Opening Remarks

Chair:

Ksenia Matlawska-Wasowska, PhD

Session Description

This session will highlight recent advances in defining the genomic architecture of T-cell acute lymphoblastic leukemia (T-ALL), including newly recognized molecular subtypes, oncogenic drivers and inherited susceptibility. Speakers will discuss how genomic alterations shape disease biology, contribute to relapse and treatment resistance, and inform emerging approaches to risk stratification and precision therapy.

Moderator:

Joao Barata, PhD
Universidade de Lisboa

Speakers:

Joao Barata, PhD
Universidade de Lisboa
Moderator Introduction

Charles G. Mullighan, MBBS (Hons), MSc, MD
St Jude Children's Research Hospital
Emerging Insights into the Genomic Landscape of T-ALL

David Teachey, MD
Children's Hospital of Philadelphia
Genomic Drivers of Relapse and Therapeutic Resistance in T-ALL

Jun J. Yang, PhD
St. Jude Children's Research Hospital
Inherited Susceptibility and Germline Genetics of T-ALL

Session Description

This session will explore how transcriptional and epigenetic programs establish and maintain distinct T-ALL cell states and create therapeutically actionable dependencies. Presentations will address transcriptional networks, signaling pathways, and chemical-genomic approaches that uncover vulnerabilities across biologically and clinically relevant T-ALL subtypes.

Moderator:

Mark Chiang, MD, PhD
University of Michigan

Speakers:

Mark Chiang, MD, PhD
University of Michigan
Moderator Introduction

Mark Chiang, MD, PhD
University of Michigan
Transcriptional Network Profiling Reveals Cell State Vulnerabilities in ETP leukemia

Giovanni Roti, MD, PhD
University of Parma
From Chemical Genomics to Vulnerability in T-ALL

Joao Barata, PhD
Universidade de Lisboa
IL7R Signaling Networks in T-ALL

Session Description

BREAK

Session Description

This session will examine how metabolic adaptation supports T-ALL initiation, maintenance and response to therapy. Speakers will discuss the interplay between enhancer and transcriptional regulation, RNA modification, nutrient availability, and metabolic rewiring, with an emphasis on identifying metabolic dependencies that can be exploited therapeutically.

Moderator:

Daniel Herranz, PharmD, PhD
Rutgers Cancer Institute

Speakers:

Daniel Herranz, PharmD, PhD
Rutgers Cancer Institute
Moderator Introduction

Rahul S Bhansali, MD
University of Pennsylvania
LDB1-Dependent Enhancer Connectivity Defines T-ALL Identities and Masks Metabolic Vulnerabilities

Hudan Liu, PhD
Wuhan University
FTO-Driven Metabolic Reprogramming as a Therapeutic Vulnerability in T-ALL

Daniel Herranz, PharmD, PhD
Rutgers Cancer Institute
Dietary Interventions and Metabolic Rewiring in T-ALL

Session Description

This session will focus on how interactions between T-ALL cells and their surrounding microenvironments influence leukemia progression, cellular plasticity, stress adaptation and treatment response. Particular emphasis will be placed on inflammatory and immune-regulatory programs, adaptive responses to niche-derived stress, and the biological mechanisms underlying central nervous system involvement, with discussion of opportunities for therapeutic targeting.

Moderator:

Ksenia Matlawska-Wasowska, PhD
University of Alabama at Birmingham

Speakers:

Ksenia Matlawska-Wasowska, PhD
University of Alabama at Birmingham
Moderator Introduction

Anastasia Tikhonova, PhD
Princess Margaret Cancer Centre, University Health Network
Inflammatory Programs and Immune Regulation in T-ALL

Birgit Knoechel, MD, PhD
Huntsman Cancer Institute, University of Utah
Adaptive Stress Responses and T-ALL Cell Plasticity

Ksenia Matlawska-Wasowska, PhD
University of Alabama at Birmingham
CNS Involvement in T-ALL: Biology and Translational Opportunities

Session Description

Closing Remarks

Chair:

Daniel Herranz, PharmD, PhD

Session Description

Opening Remarks

Chair:

Panagiotis Ntziachristos, PhD

Session Description

This session integrates computational tools with chromatin biology and clonal evolution as drivers of therapy resistance in hematologic malignancies. Geometry-informed deep learning frames single-cell chromatin trajectories; complementary single-cell genomics and DNA-methylation approaches resolve clonal evolution in CLL, MDS, and AML; chemical-genetic dissection identifies actionable chromatin and transcriptional vulnerabilities; and epigenetic studies of clonal hematopoiesis trace the origins of therapy-resistant leukemia. Together the talks foreground computational tools as a methodological backbone connecting chromatin states, clonal architecture, and the broader microenvironmental context. The session offers clinicians, researchers, and other health professionals a framework for risk stratification of clonal hematopoiesis, together with deployable single-cell platforms and tractable chromatin and metabolic targets.

Moderator:

Aristotelis Tsirigos, PhD
NYU Grossman School of Medicine

Speakers:

Aristotelis Tsirigos, PhD
NYU Grossman School of Medicine
Moderator Introduction

Maria Figueroa, MD
University of Miami, Miller School of Medicine
Epigenetics of HMA Resistance in MDS and CMML

Andrew Volk, PhD
Cincinnati Children's Hospital Medical Center
A Transient Differentiation-Permissive State Underlies Adaptive Lineage Redistribution during Venetoclax-Based Therapy in Acute Myeloid Leukemia

Marek Mraz, MD, PhD
CEITEC, Masaryk University
Interferon-?–STAT1 Transcriptional Rewiring as a Resistance Mechanism to Venetoclax in CLL

Zhe Yang, PhD
Stowers Institute for Medical Research
AI-Designed, mRNA-Delivered Peptides Deplete Leukemia Stem Cells by Intracellular Targeting of AKT–Wnt/ß-catenin Signaling

Session Description

This session focuses on RNA-level mechanisms that sustain therapy resistance in MDS, AML, and lymphoid malignancies. Coverage spans RNA-regulatory networks governing human hematopoiesis and disease vulnerability;splicing dysregulation as a driver of leukemia therapy resistance; RNA editing as a node linking inflammation to leukemic stem-cell adaptation; and post-transcriptional control of self-renewal. Computational tools for transcript-isoform and modification analysis recur as a cross-cutting methodological thread, and the inflammation–microenvironment axis surfaces where RNA-level circuits intersect with niche-derived signals. The session is positioned to inform clinicians, researchers, and other health professionals on emerging RNA-targeted therapeutics and on tractable post-transcriptional vulnerabilities.

Moderator:

Daniel Starczynowski, PhD
Cincinnati Children's Hospital

Speakers:

Daniel Starczynowski, PhD
Cincinnati Children's Hospital
Moderator Introduction

Vijay Sankaran, MD, PhD
Boston Children's Hospital and Dana-Farber Cancer Institute, Harvard Medical School
Genetic Variation Impacting RNA Regulation in Therapy Resistance

Rui Su, PhD
City of Hope
DDX2A Restrains RNA Granule Solidification to Modulate Leukemia Metabolism and Drug Resistance

Catriona Jamieson, MD, PhD
University of California San Diego
Inflammation-Driven RNA Editing in Leukemia Stem Cell Persistence

Florian Kuchenbauer, MD, PhD
BC Cancer
HLA Dysregulation Defines a Novel Subgroup of NPM1-Mutated AML Linking Immune Escape and Chemotherapy Resistance

Session Description

Break

Session Description

This session integrates protein-level therapeutic strategies with microenvironment and inflammatory drivers of therapy resistance. Targeted protein degradation and molecular glues frame post-translational vulnerabilities; JAK-STAT signaling and inflammation connect MPN and AML resistance to systemic and niche-derived signals; intravital imaging of the bone-marrow niche reveals stem cell–niche dynamics during leukemia treatment and relapse; and bone-marrow niche remodeling, encompassing stromal and innate-immune contributions,completes the picture of microenvironmental drivers of chemoresistance. Computational image analysis underpins the niche-imaging work, reinforcing the cross-cutting role of computational tools across the workshop. The session provides clinicians, researchers, and other health professionals a translational framework for combining JAK and degrader-based therapies with niche-directed strategies and orients the audience toward emerging targets at the protein-microenvironment interface.

Moderator:

Christina Glytsou, PhD
Rutgers University

Speakers:

Christina Glytsou, PhD
Rutgers University
Moderator Introduction

Daniel Lucas-Alcaraz, PhD
Cincinnati Children's Hospital Medical Center
Micro-Anatomical Control of Hematopoiesis

Marc Diederich, PhD
Seoul National University
Convergent OPA1 Targeting in AML: Isoform-Resolved Kinetics of Mitochondrial Collapse

Oliver Van Oekelen, MD
Icahn School of Medicine at Mount Sinai
S100A8/A9 Drives T Cell Dysfunction and Resistance to T Cell-Engaging Therapies in Multiple Myeloma

Eirini Papapetrou, MD, PhD
Icahn School of Medicine at Mount Sinai
RAS-Mutant Leukemia Stem Cells Promote AML Growth and Venetoclax Resistance through Non-Cell-Autonomous Mechanisms

Session Description

Closing Remarks

Chair:

Panagiotis Ntziachristos, PhD

Session Description

Opening Remarks

Chair:

Ann-Kathrin Eisfeld, MD

Session Description

In this section speakers will set the stage about CH and the various risk factors that can lead to TP53 and Clonal Hematopoiesis (which will be contextualized in the introduction as the full spectrum from first mutation to CH to MDS to AML). Speakers will discuss the multitude of factors at play in this malignancy which will include germline predisposition, Therapy-related CH, Age-related CH, external stressors, and potential other factors.

Moderator:

Annette Kim, MD, PhD
University of Michigan

Speakers:

Annette Kim, MD, PhD
University of Michigan
Moderator Introduction

Sanam Loghavi, MD
MD Anderson Cancer Center
Introduction/Background

Kelly Bolton, MD, PhD
Washington University/Siteman Cancer Center
Therapy-Related CH

Christopher R. Reilly, MD
Dana Farber Cancer Institute
Age-Related CH

Irum Khan, MD
Northwestern Feinberg School of Medicine
Stress and Other Factors

Session Description

BREAK

Session Description

This section will define TP53-related biological vulnerabilities by dissecting allelic states, co-mutation patterns, microenvironmental interactions, and pathway-level mimics, while addressing technical challenges in detection and variant interpretation using advanced genomic and functional tools.

Moderator:

Lachelle Weeks, MD, PhD
Dana Faber Cancer Institute

Speakers:

Lachelle Weeks, MD, PhD
Dana Faber Cancer Institute
Moderator Introduction: Potential Targets and Biomarkers: Unique Aspects of TP53

Elli Papaemmanuil, PhD
Genentech
Prognostic and Predictive Factors (Mono vs Biallelic, Complex Karyotype, Biomarkers, and Measuring TP53)

Torsten Haferlach, MD, PhD
MLL Munich Leukemia Laboratory
TP53 Variants that Frequently Beguile Existing Testing Modalities AND? Limitations of Existing Testing Modalities as They Pertain to TP53

Sergio Rutella, MD
University of Sheffield
Microenvironment and Geographic Distribution

Session Description

BREAK

Session Description

Leveraging the mechanistic insights from session 2, speakers will explore current and emerging therapeutic strategies, including immune-mediated control, rational targeting of TP53-driven networks, resistance mechanisms, and features of responders versus non-responders in preclinical and clinical studies.

Moderator:

Ann-Kathrin Eisfeld, MD
Ohio State University Medical Center

Speakers:

Ann-Kathrin Eisfeld, MD
The Ohio State University Wexner Medical Center
Moderator Introduction

Shruti Bhatt, PhD
Emory University
Features of Responders / Non-Responders

Alice Scott Mims I, MD
Ohio State University
Current and Recent Past Clinical Trials

Charles Craddock, MD
Queen Elizabeth Hospital
Planned/Ongoing Clinical Trials

Session Description

Closing Remarks

Chair:

Jonathan Gerber, MD