Scientific Spotlight Sessions

Bridging Translational Science and Clinical Trials to Transform Burkitt Lymphoma Research Globally

Monday, December 8, 2025, 10:30 a.m. - 11:45 a.m.
OCCC - Tangerine Ballroom F2

The treatment of Burkitt’s lymphoma (BL) is a testament to the profound curative potential of combination chemotherapy, and most patients are cured with modern treatment approaches that utilize intensive chemotherapy along with aggressive supportive care. Still, gaps remain for patients unable to tolerate intensive chemotherapy, for those with disease that become resistant to chemotherapy, and for those who live in areas of the world with fewer supportive care resources.  This session will examine the current understanding of the molecular biology of BL, the tumor microenvironment and how this might influence clinical and translational research efforts.  It will also explore and highlight significant efforts in Sub-Saharan Africa that attempt to bridge the gap and improve outcomes globally.

Dr. Mark Roschewski will describe the history of BL and its emergence as one of the most sensitive subtypes of lymphoma to chemotherapy.  More recently, the field has come to understand molecular subtypes of BL that may have differential response to immunotherapy and/or targeted agents.  Dr. Roschewski will also address the role of the tumor microenvironment and its relevance in clinical outcomes. 

Dr. Clara Chamba will present progress from the Aggressive Infection-Related East African Lymphoma (AI-REAL) study, focusing on innovative approaches to diagnosing and managing BL in Sub-Saharan Africa. She will highlight minimally invasive diagnostic strategies, share key insights from recent validation efforts, and discuss lessons learned in building sustainable capacity for lymphoma care in resource-limited settings. Dr. Chamba will also reflect on the collaborative model that brought together international partners and local institutions to address diagnostic delays. Finally, she will outline the broader implications of this work for advancing equitable access to timely cancer diagnosis across low- and middle-income countries.

Speakers:

Mark Roschewski, MD
Center for Cancer Research, National Cancer Institute, NIH
Bethesda, MD, United States
Translating Knowledge about Burkitt Lymphoma Genomics and Microenvironment into Novel Clinical Trials

Mark Roschewski, MD
NIH/NIC
Bethesda, MD, United States
Translating Knowledge about Burkitt Lymphoma Genomics and Microenvironment into Novel Clinical Trials

Clara Chamba
Muhimbili University of Health and Allied Sciences
Dar-es-Salaam, Tanzania
Burkitt Lymphoma Research in Africa: Overcoming Challenges and Unlocking Translational Potential

Creative and Novel Statistical Techniques to Design and Analyze Data for Trials Focused on Rare Hematologic Diseases

Monday, December 8, 2025, 10:30 a.m. - 11:45 a.m.
Hyatt - Regency Ballroom R

This session will highlight novel statistical methods and unique design and analysis considerations for clinical trials focused on rare hematologic diseases. It will provide a unique platform for interactive discussion and knowledge sharing on statistical design/analysis of clinical studies, for the clinical community.

Chair:

Fangxin Hong
Pfizer Inc
Cambridge, MA, United States

Speakers:

Arzu Onar-Thomas
St Jude's Children Hospital
Memphis, TN, United States
Rational Compromises in Trial Design for Rare Diseases - Lessons from Pediatric Cancer

Ernest Amankwah
Johns Hopkins All Children's Hospital
St Petersburg, United States
Design and Application of RCT for Rare/Low-Frequency Diseases: From Traditional Parallel-Cohort to Novel Contemporaneous Control Recapture

Ernest Amankwah
Johns Hopkins University School of Medicine
Baltimore, United States
Design and Application of RCT for Rare/Low-Frequency Diseases: From Traditional Parallel-Cohort to Novel Contemporaneous Control Recapture

Epigenomic Frontiers in the Diagnosis of Hematological Malignancies

Monday, December 8, 2025, 2:45 p.m. - 4:00 p.m.
OCCC - W230

The rapid and accurate diagnosis of acute leukemias remains a significant challenge in hematopathology. Traditional diagnostic methods often fall short in providing timely and precise classification, which is crucial for effective treatment planning. Recent advancements in next-generation and third-generation sequencing technologies have revolutionized our ability to read out nucleotide sequences and epigenetic modifications, such as chromatin accessibility and DNA methylation. These technologies offer a promising avenue for improving the diagnostic accuracy and speed for hematological malignancies.

By leveraging long-read sequencing and chromatin analysis, researchers are now able to classify leukemias more accurately and rapidly than ever before. These innovative approaches enhance our understanding of the molecular underpinnings of these malignancies and are entering evaluation in clinical trials.

This session will highlight cutting-edge developments in epigenomic and chromatin-based diagnostics.

Chair:

Bert Van der Reijden, PhD
Radboudumc
Nijmegen, Netherlands

Speakers:

Volker Hovestadt, PhD
Dana-Farber Cancer Institute
Boston, MA, United States
Long-Read Epigenomic Classification of Acute Leukemia

Florence Nguyen-Khac
Centre de recherche des Cordeliers/INSERM1138
Paris, France
Chromatin-Based Diagnosis in Lymphocytic Leukemia

Florence Nguyen-Khac
Hopital Pitie-Salpetriere, Sorbonne Universite
Paris, France
Chromatin-Based Diagnosis in Lymphocytic Leukemia

The Issue of Rejection of Allogeneic Cellular Therapies: Evidence, Mechanisms, and Novel Strategies to Overcome It

Sunday, December 7, 2025, 4:30 p.m. - 5:45 p.m.
OCCC - Sunburst Room (W340)

Allogeneic cell therapies offer significant advantages over autologous approaches, including reduced production times and broader accessibility for patients with rapidly progressing malignancies. However, immune rejection remains a critical challenge, as the host immune system often identifies and eliminates donor-derived cells through both adaptive and innate mechanisms. To address these barriers, researchers are developing innovative strategies such as genetic engineering to delete major histocompatibility complex molecules, overexpress immune checkpoint ligands, and disrupt key signaling pathways in innate immune cells. Additionally, immune cloaking techniques, like expressing CD47 to inhibit macrophage-mediated phagocytosis, and advancements in genome editing technologies, including CRISPR-Cas9, are being explored to enhance the persistence and efficacy of allogeneic cells.

This session will explore the breakthroughs aimed at overcoming immune rejection in allogeneic cell therapies.

Speakers:

Marco Ruella Jr, MD
Abramson Cancer Center
Philadelphia, PA, United States
Current Clinical Evidence of Allogeneic Cellular Products Failure and Mechanisms

Marco Ruella Jr, MD
University of Pennsylvania
Philadelphia, PA, United States
Current Clinical Evidence of Allogeneic Cellular Products Failure and Mechanisms

May Daher, MD
MD Anderson Cancer Center
Houston, TX, United States
Strategies to Reduce Immune Rejection of Off-the-Shelf Cell Therapies

Transcending Boundaries: Study of Ambiguous Lineage Acute Leukemia Unlocks Mysteries for All Leukemias

Monday, December 8, 2025, 4:30 p.m. - 5:45 p.m.
OCCC - W224CDGH

The existence of acute leukemias of mixed or ambiguous lineage has long been recognized but the diagnostic criteria for mixed phenotype acute leukemia (MPAL) has been historically fluid. This fluidity reflects the heterogeneous nature of this entity, which unlike other acute leukemias, largely lacks defining genetic lesions and relies on immunophenotypic criteria for diagnosis, though more recent diagnostic criteria incorporate a limited number of genetic abnormalities. This lack of disease-defining genetics further underscores a dearth of understanding for molecular mechanisms driving pathophysiology. Furthermore, shifting diagnostic criteria and disease heterogeneity make clinical studies of this rare disease challenging, presenting major barriers to refining treatment to optimize clinical outcomes. This session will discuss the phenomenon of ambiguous lineage acute leukemia as a spectrum encompassing multiple leukemia subtypes that are characterized by retained differentiation potential and lineage plasticity.

Dr. Ruud Delwel will discuss how leukemias that were initially uncovered as acute myeloid leukemias (AML) appeared to be clearly distinct based on unique DNA methylation profiles. These leukemias exhibit hybrid myeloid/lymphoid epigenetic landscapes. DNA methylation reveals similarity with early T-cell precursor acute lymphoblastic leukemias, whereas histone acetylation signatures are more in line with profiles uncovered in AML, in particular with cases that carry CEBPA mutations. Dr. Delwel will discuss the role of epigenetic dysregulation as the basis of malignant development of these leukemias with ambiguous lineage.

Dr. Catherine Smith will discuss MPAL as a stem cell-like leukemia, focusing on multiomic single cell sequencing studies of MPAL which reveal a shared stem cell-like transcriptional profile indicative of high differentiation potential and independent of underlying genetic abnormalities. She will describe MPAL95, a gene set score established from genes highly enriched in the most stem-like MPAL cells, which is applicable to bulk RNA sequencing data and is predictive of survival in adult and pediatric MPAL patient cohorts.

Speakers:

Ruud Delwel
Erasmus MC Cancer Institute and Oncode Institute
Rotterdam, Netherlands
Epigenetic Dysregulation Drives Mixed Phenotype Acute Leukemia

Catherine Smith, MD
University of California at San Francisco
San Francisco, CA, United States
Multi-omic Analysis Reveals the Heterogeneity and Stem Cell Origin of Acute Leukemia of Ambiguous Lineage/Mixed Phenotype Acute Leukemia

Catherine Smith, MD
University of California, San Francisco
San Francisco, CA, United States
Multi-omic Analysis Reveals the Heterogeneity and Stem Cell Origin of Acute Leukemia of Ambiguous Lineage/Mixed Phenotype Acute Leukemia