Dr. Koschmann will have three key objectives:

1. Explain the mechanism of ONC201 in pediatric H3K27M-mutant diffuse midline glioma (DMG).
2. Detail the genetic factors from tumor sequencing that may predict response to ONC201.
3. Present the correlate assessments through liquid biopsies to track responses to therapy in H3K27M-DMG.

Dr. Carl Koschmann is an Associate Professor and the Chad Carr Pediatric Brain Tumor Center Research Professor in the Department of Pediatrics at the University of Michigan. He also serves as their Director and the Children’s Brain Tumor Foundation Co-Scientific Director. His lab investigates the molecular mechanisms by which mutations in pediatric high-grade glioma (HGG) and DMG promote tumor growth and therapeutic response. His longstanding NIH/NINDS-funded project on the role of ATRX in pHGG has uncovered the impact on cell cycle control and targeted therapeutics, resulting in publications in Science Translational Medicine and Cell Reports. Dr. Koschmann’s lab has also established an intrauterine electroporation (IUE) mouse model that is driven by pHGG drivers as detailed in JCI and Neuro Oncology publications. This model is the basis of translational studies supported by a U.S. Department of Defense Cancer Translational Team Science Award and an NIH/NINDS R01, studying the efficacy of ONC201 treatment in DMG as detailed in Cancer Discovery. He additionally serves as the PI/MPI for multiple early phase studies in pHGG/DMG, and his lab performs analysis of correlate tumor and CSF samples, as detailed in manuscripts in Clinical Cancer Research and Neuro-Oncology.

This lecture will have four objectives:

1. Appreciate the role of liquid biopsy in cancer and the emerging role of this approach in pediatric malignancies.
2. Learn about the unique nature and complexities of exosomes and other subtypes of extracellular vesicles and particles as biological mediators and liquid biopsy analytes.
3. Reflect on the link between oncogenic transformation and extracellular vesicle landscapes in cancer, including inroads made in some pediatric malignancies.
4. Touch on the emerging technologies aiming to analyse extracellular vesicle populations for the purpose of discovery and liquid biopsy applications.

Janusz Rak, MD, PhD is a Professor of Pediatrics, Experimental Medicine, Human Genetics and Biochemistry and Jack Cole Chair in Pediatric Hematology/Oncology at McGill University as well as Investigator at the Research Institute of the McGill University Health Centre (RIMUHC) in Montreal, QC, Canada. His laboratory investigates how oncogenic events deregulate vascular tumour microenvironment, orchestrate intercellular communications and impact processes mediated by the exchange of extracellular vesicles (EVs), including exosomes. He uncovered EVs carrying oncogenic cargo (oncosomes), an area in which his group is developing diagnostic applications, such as liquid biopsy, in cancers across the age spectrum. These studies increasingly concentrate on pediatric brain tumours. He was among founding members, Member of the Board and Treasurer of the International Society for Extracellular Vesicles (ISEV) and remains an Associate Editor of the Society’s Journal of Extracellular Vesicles and Journal of Extracellular Biology. He published over 220 research papers (37,000 citations, h-index 87) in Nature, Nature Cell Biology, Nature Communications, ACS Nano and other journals. He currently directs the Canada Foundation of Innovation program, Centre for Applied Nanomedicine (CAN), at the RIMUHC providing instrumental support for advanced EV research. He also leads the NET program sponsored by Fondation Charles Bruneau and Fondation CIBC to investigate EV-based liquid biopsy biomarkers in pediatric cancer. His research laboratory is also supported by Canadian Institutes of Health Research and other sources. He is a foreign Member of the Polish Academy of Sciences, Professor of the Masaryk University in Brno, Czech Republic, and Fellow of the Royal Society of Canada.

This lecture will have three objectives:

1. Frame the challenges of clinical drug development for children with cancer.
2. Discuss examples of successes and learnings in drug development.
3. Considerations for meeting the unmet needs of childhood cancer.

Dr. Fox leads regulatory and clinical trial operations as well clinical research prioritization and strategy at St Jude Children's Research Hospital. Nationally, she is the vice-chair of Developmental Therapeutics for the Children’s Oncology Group and the UM1 funded Pediatric Early Phase Clinical Trials Network. Dr. Fox has expertise in quantitative clinical and pre-clinical pharmacology, pediatric clinical oncology, and clinical research trial design, including response and toxicity biomarker endpoint development. Dr Fox received her Medical Degree from Pennsylvania State University and Masters of Clinical Research from Duke University.

Her fellowship training and early career in pediatric oncology and drug development was at the Pediatric Branch of the Intramural Program of the NCI, Bethesda, MD. Prior to coming to St Jude in 2020, she was a Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania, Director of Developmental Therapeutics in Oncology at the Children’s Hospital of Philadelphia, and PI of the ALSF Center of Excellence in Clinical Research and Pharmacology at the Children’s Hospital of Philadelphia. Dr Fox has contributed to the FDA approval of 7 of the 50 oncology drugs currently approved for children with cancer. Her professional mission is to utilize clinical research to develop the most promising therapies for children and mentor clinician-scientists and a diverse clinical research workforce to foster innovation to improve the outcome for children with catastrophic disease.

This lecture will have three objectives:

1. To examine how NF1 mouse low-grade brain tumor growth is controlled by immune system cell circuits.
2. To explain how these stromal circuits suggest potential pediatric low-grade glioma therapeutic targets.
3. To understand how risk factors converge on these stromal circuits to dictate pediatric low-grade glioma biology.

Dr. Gutmann received his undergraduate, graduate (PhD) and medical (MD) degrees from University of Michigan. He identified the neurofibromatosis type 1 (NF1) protein and began uncovering its function as a negative RAS regulator. He has been at Washington University since 1993, becoming a full professor in 2001. He established both the Children’s Hospital Neurofibromatosis Clinical Program and the Washington University Neurofibromatosis Center, and he is currently the Donald O. Schnuck Family Professor and the Director of the Neurofibromatosis Center.

Dr. Gutmann’s research focuses on understanding the genomic, molecular and cellular basis for nervous system problems affecting children and adults with NF1. As founder and director of one of the world’s largest centers for NF and related disorders, he has trained numerous NF clinicians and researchers in collaborative cross-disciplinary research and developed innovative community-based therapy programs to provide integrated medical care for this population. Dr. Gutmann has published over 500 peer-reviewed manuscripts, and his research lab has been funded by the National Institutes of Health since 1993, including a National Institute of Neurological Disorders and Stroke R35 Research Program (MERIT) Award.

Dr. Sayour’s discussion will have three key takeaways:

• Understand the necessity of type I interferons in sensitizing cancer immunogenicity.
• Understand the role of RNA-nanoparticles as an immunomodulating agent and a vaccine therapy.
• Understand the role of large animals in comparative oncology as an accelerant in bringing translational therapeutics to clinical trial.

Elias Sayour, MD, PhD is a board-certified pediatric oncologist who has served as sponsor of multiple FDA-IND submissions leading to first-in-human clinical trials (NCT05264974, NCT04573140, NCT05660408). He has spearheaded the creation of the UF Pediatric Cancer Immunotherapy Initiative (PCI2) and the Large Animal Comparative Oncology Initiative and serves as principal investigator of the RNA Engineering Laboratory where he has been the primary inventor on a number of pending patent applications concerning the immunologic treatment of cancer.

Dr. Davis’s discussion has three aims:

• Review current understanding of relapse in B ALL.
• Explain B cell developmental stages and decision-making.
• Describe the role of transcription factors and metabolic states in relapse to current therapies.

Kara L. Davis, D.O. is an Assistant Professor of Pediatrics at Stanford University in the Division of Hematology and Oncology. Dr. Davis is both the Anne T. Robert M. Bass Endowed Faculty Scholar and the Harriet and Mary Zelencik Endowed Faculty in Pediatric Cancer and Blood Diseases. Having received her B.A. from Pennsylvania State University and Doctor of Osteopathy from Philadelphia College of Osteopathic Medicine, Dr. Davis completed her pediatric training at Thomas Jefferson University and Oncology/Hematology Fellowship at Stanford.

Dr. Davis has been instrumental in leading clinical trials utilizing the immune system to treat relapsed leukemia and solid tumors. She served as the institutional PI for the pivotal trial which led to FDA approval of the first cellular therapy for cancer, chimeric antigen T-cells. She is a co-investigator on the Stanford institutional clinical trials for CD19-CD22 bispecific CAR and CD22 CAR for relapsed or refractory leukemia and the GD2 CAR for childhood brain tumors and leads the clinical program for cell therapies in pediatrics. Dr. Davis is active in the Children’s Oncology Group in the acute lymphoblastic leukemia committee and co-lead in the phase I-II study of immune checkpoint inhibition in children and young adults through the Children’s Oncology Group.

Dr. Harbour’s talk will have two main objectives:

• Explain the current challenges in treating retinoblastoma.
• Discuss how recent progress in genomic research is uncovering new potential therapies in retinoblastoma.

J. William Harbour, MD, is a pioneering retina specialist, ocular oncologist and cancer researcher. He attended Johns Hopkins for medical school, Wills Eye Hospital for residency, Bascom Palmer Eye Institute for retina fellowship, University of California San Francisco for ocular oncology fellowship, and Washington University in St. Louis for postdoctoral cancer research training.

Dr. Harbour was a distinguished professor at Washington University and the Mark J. Daily Endowed Professor and Vice Chairman for Translational Research at Bascom Palmer Eye Institute, and Associate Director for Basic Science at Sylvester Comprehensive Cancer Center at University of Miami. Today, Dr. Harbour serves as Chair of the Department of Ophthalmology at the University of Texas Southwestern Medical Center. His research has been continuously funded for over 25 years by the National Institutes of Health, Research to Prevent Blindness, Alcon Research Institute, and numerous other sources. Among his many accomplishments, Dr. Harbour invented a multi-gene AI-driven prognostic test for uveal melanoma that is standard of care in North America. He discovered that RB1 loss in retinoblastoma initiates an oncogenic dependency on ESRRG, which may represent a novel therapeutic target in this cancer.

This talk will cover three main areas of focus:

• Reinforce awareness of the contributions of germline variants to childhood cancer.
• Understand the impact of cancer predisposition in guiding care for children and their families.
• Awareness of guidelines and tools for patient identification and tumor surveillance.

Dr. Porter is the Director of the Cancer Predisposition Program at the Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta and Professor of Pediatrics at Emory University. In addition, he is the Co-Chair of the Consortium for Childhood Cancer Predisposition and Co-Chair of the Cancer Predisposition Working Group of the Children's Oncology Group.

In this talk, you’ll learn more about:

1. The ins and outs of tumor organoids.
2. What tumor organoids can teach us about sarcoma biology, drug sensitivity and resistance.
3. The advantages and limitations of using organoids for personalized medicine.

Alice Soragni, PhD, is an Assistant Professor in the David Geffen School of Medicine at UCLA and a member of the Jonsson Comprehensive Cancer Center as well as the UCLA Molecular Biology Institute. She has a Master of Science cum laude from the University of Bologna, Italy and a PhD from the ETH of Zuerich, Switzerland. Her laboratory in the Department of Orthopaedic Surgery at UCLA couples basic research into mechanisms of disease to the development of novel therapeutic strategies. Dr. Soragni’s expertise is in the development of tumor organoid models to investigate the biology of rare tumors and perform screenings for functional precision medicine applications.

Learning objectives: In this lecture you will develop an understanding of the...

1. Tumor target landscape
2. Process for targeting of peptide-MHC (pMHC) complexes tumor antigens
3. CAR T cell promises and challenges
4. Immunotherapy target discovery
5. And finally gain an introduction to Peptide-Centric (PC)-CAR T cells

Dr. Mark Yarmarkovich is an Assistant Professor at NYU Grossman School of Medicine focused on developing safe and effective cancer immunotherapies through innovations in biotechnology. He has worked in industry at Genentech and completed his PhD at the University of Pennsylvania and postdoctoral work with John Maris at the Children's Hospital of Philadelphia, working across a number of disciplines including oncology, protein chemistry, immunology, and computational biology, applying these experiences to addressing the major challenges in developing the next generation of immunotherapies. He has led the development of new technologies for the discovery of tumor-specific targets and the engineering of target-specific receptors. He has discovered novel immunotherapy targets derived from previously undruggable oncoproteins and engineered CAR T cells capable of specifically killing tumor cells. He led the discovery of cross-HLA recognition of Peptide-Centric (PC)-CAR T cells, allowing for significantly expanded application of immunotherapies. Collectively, the application of his efforts has resulted in a PC-CAR T cells entering first-in-class clinical trial in pediatric neuroblastoma in 2023 and a pipeline of PC-CARs being additional cancers. His lab is focused on developing the next generation of immunotherapies to help cancer patients in need.

Participants can expect three key takeaways:

1. Recognize the high failure rate of pediatric oncology clinical trials.
2. Understand the need for rigor and reproducibility in pediatric pre-clinical data before advancing drugs for testing in children.
3. Appreciate the opportunities available for developing new agents for advancement to clinical testing through PNOC.

Dr. Eric Raabe received his MD and PhD from the University of Cincinnati College of Medicine. He completed his general pediatrics residency at Children’s Hospital of Philadelphia and then worked in Africa as part of the Baylor International Pediatric AIDS Corps. He received fellowship training at Johns Hopkins in Pediatric Hematology/Oncology and Pediatric Neuro-oncology. He joined the faculty at Johns Hopkins in 2011, where he leads a basic and translational research group targeting stem cell factors and their downstream effectors in high-risk pediatric brain tumors.

Dr. Qasim’s lecture will:

• Summarize two decades of development and gene therapy trial experience at GOS.
• Provide updates on TALEN and CRISPPR developments to provide “universal” cell therapies using engineered CAR T cells.
• Discuss how emerging base-editing has enabled T cells to be directed against T cell malignancies.

Dr. Qasim is a Pediatric Immunologist and Professor of Cell and Gene Therapy at Great Ormond Street Hospital and UCL Institute of Child Health in London. He trained in Newcastle and qualified in Medicine with a specialization in Pediatrics and Immunology. After conducting PhD research into T cell engineering, he delivered gene therapies from bench-to-bedside. His first-in-human applications of viral vectors were followed by trials in genome editing with TALENs, CRISPR/Cas9 and Base Editors. Now, clinical trials are actively exploiting engineered CAR-T cells against acute leukemia and overcoming allogeneic barriers to provide “universal” cell therapies.

During this lecture, participants will:

• Learn about the limitations and potential of genomic testing for the analysis of pediatric cancers.
• Understand how genomic tumor and blood testing has become a fundamental aspect of pediatric cancer care.
• Gain insight into key observations from the NCI-COG Pediatric MATCH trial for children and young adults with refractory cancers.

Dr. Parsons is a board-certified pediatric oncologist and Deputy Director of Texas Children’s Cancer and Hematology Center with a particular interest in the development and evaluation of molecularly targeted therapies. Plus, his work has been instrumental in characterizing the genetic landscapes of a variety of pediatric and adult cancers. His leadership roles include serving as the Children’s Oncology Group Study Chair for the NCI-Cog Pediatric MATCH trial and Steering Committee member for the NIH Pediatric Early Phase Clinical Trials Network. He received his B.A. in chemistry from Princeton University and his MD and PhD from The Ohio State University College of Medicine. His pediatric residency was completed at Johns Hopkins University with oncology/neuro-oncology fellowship training at Johns Hopkins and the Pediatric Oncology Branch of the National Cancer Institute.

Driver mutations in childhood brain tumors often show a remarkable spatiotemporal distribution, tightly linked with brain development. Childhood tumors originate in developing tissues intrinsically different from those in adults, and thus likely possess different vulnerabilities. Here, I will present our work towards systematic identification of tumor origins, by generating single-cell resolution maps of the developing brain to catalogue cell populations in regions where these tumors arise, their dynamics over time, and their distinct cellular states. I will discuss our computational strategies for analyzing multimodal tumor datasets, and how integration with normal developing references points to the lineage of origin, the differentiation state, and the chromatin architecture and associated vulnerabilities of several subtypes of high-grade gliomas.

Three key takeaways participants will learn from the presentation.

After this lecture, participants:

• Understand mechanisms by which the lineage of origin can dictate mutational patterns observed in patients. 
• Learn about integrative profiling methods to characterize the transcriptomic and epigenomic landscape of tumors. 
• Understand how to leverage large single-cell resolution references of normal developing tissues to interpret tumor data. 

Dr Claudia Kleinman is an Associate Professor in the Department of Human Genetics, Faculty of Medicine, at McGill University, Montreal, Canada. She is a fulltime Investigator at the Lady Davis Institute for Medical Research, an Associate Member of the McGill Centre for Translational Research in Cancer, and a researcher at the Ludmer Centre for Neuroinformatics & Mental Health. Her research exploits genome-wide technologies and data science to understand mechanisms of gene expression. She has an interdisciplinary training that combines molecular biology, computer science, statistics and evolutionary biology, which she applies to the study of pathological transcriptional and RNA processing events, focusing particularly on pediatric brain tumors. She has received awards from the Natural Sciences and Engineering Research Council of Canada (NSERC), Fond de Recherche du Québec – Santé (FRQS), Canadian Institutes of Health Research (CIHR) and currently holds a career award from the Fond de Recherche du Québec – Santé (FRQS). She is the recipient of the Bernard and Francine Dorval Prize, for her work mapping the development of the human brain at the single cell level to define the origins of pediatric brain tumors.

During this lecture, participants will learn about:

1. How immune components of pediatric tumors can be characterized by various complementary genomic techniques.
2. Which pediatric cancers are informative of immunotherapy outcomes based on distinct classes of immune infiltration.
3. How cell-free DNA sequencing may be an indirect approach to infer shifts in a tumor’s immune cell repertoire during treatment.

Dr. Trevor Pugh, PhD, FACMG is a cancer genomics researcher board-certified in molecular genetics, holding the title of Canada Research Chair in Translational Genomics. He is Director of the Joint Genomics Program of the University Health Network and Ontario Institute for Cancer Research – which delivers basic, translational and clinically-accredited genomics services.

Dr. Pugh is also Associate Professor in the University of Toronto Department of Medical Biophysics, Senior Scientist at the Princess Margaret Cancer Centre, and Senior Investigator at the Ontario Institute for Cancer Research. His work focuses on understanding clinical implications of clonal shifts in cancer and non-cancerous cells during treatment. Most recently, he was recognized by Canada's Top 40 Under 40, accepted the Canadian Cancer Society Bernard and Francine Dorval Prize, received a Terry Fox New Investigator Award, and was included in the Web of Science Highly Cited Researchers List (their top 1% of citations by field internationally).

During this lecture, participants will learn about:

1. To understand issues related to accessing novel agents for children with cancer.
2. To learn about new approaches to cancer drug development relevant to children.
3. To understand implications of new US regulatory measures to hasten access to new agents for children with cancer

Dr. DuBois completed medical school and pediatric training at the University of California, San Francisco (UCSF).  He completed pediatric oncology training at Dana-Farber / Boston Children’s Hospital and obtained a Master of Science in Epidemiology from the Harvard School of Public Health.  He is currently an Associate Professor of Pediatrics at Harvard Medical School.  He is the Director of Experimental Therapeutics at Dana-Farber / Boston Children’s Cancer and Blood Disorders Center where he leads a program designed to bring new targeted therapies to children with cancer.

Dr. DuBois leads an active clinical and translational research program focused on patients with advanced neuroblastoma and Ewing sarcoma.  He conducts clinical trials of novel targeted agents relevant to these diseases, including national phase 1, 2, and 3 clinical trials.  He also studies new biomarkers that improve our understanding of the biology of pediatric solid tumors and of the pharmacodynamic effects of targeted therapies.

Dr. DuBois has served on a number of national committees, including the Children’s Oncology Group (COG) Neuroblastoma Steering Committee, COG Bone Tumor Committee, COG Developmental Therapeutics Executive Committee, the American Society of Clinical Oncology (ASCO) Scientific Program Committee, and the US FDA Pediatric Oncology Drugs Advisory Committee (ODAC).

During this lecture, participants will learn about:

1. Clinical implementation of multiplex characterization in the practice of cancer medicine is possible and is impactful. 
2. Specific case studies illustrate the value added from genomics-based characterization. 
3. There is a virtuous cycle of patient-centric research that informs hypothesis driven research. 

Dr. Mardis, is co-Executive Director of the Steve and Cindy Rasmussen Institute for Genomic Medicine at Nationwide Children’s Hospital and holds the Rasmussen Nationwide Foundation Endowed Chair of Genomic Medicine. She also is Professor of Pediatrics at The Ohio State University College of Medicine.  Dr. Mardis is an internationally recognized expert in cancer genomics and immunogenomics, with ongoing interests in the integrated characterization of cancer genomes, defining DNA-based somatic and germline interactions and RNA-based pathways and immune microenvironments that lead to cancer onset and progression, with a focus on pediatric cancers and precision oncology.

During this lecture, participants will learn:

1. The role preclinical models play in the development of drugs for childhood cancer.
2. The major limitations that impact the effectiveness of preclinical models.
3. How alternative experimental designs for preclinical testing may lead to more accurate translation for clinical trials.

Dr. Houghton, is the Distinguished Chair of Pediatric and Hematologic malignancies at the Children’s Cancer Research Institute. His research is focused on understanding the biology of childhood solid tumors with a primary interest on the biology and therapy of childhood sarcoma. Dr. Houghton received his Ph.D. from the University of London, Institute of Cancer Research. He moved to St. Jude Children's Research Hospital as a postdoc and chaired the Department of Molecular Pharmacology from 1992- 2009, and co-led the Solid Tumor Research Program from 1988, till he moved to become the Director of the Childhood Cancer and Blood Diseases Institute at Nationwide Children's Hospital. From 2015 - 2021 he was Director at the Greehey Children's Cancer Research Institute, UT Health San Antonio. Dr. Houghton has contributed over 400 publications focused on development of preclinical models of childhood cancer, and drug development for pediatric cancer. Dr. Houghton's lab has been continually funded by NIH for four decades and is currently supported through NIH and CPRIT funding.

During this lecture, participants will learn:

• How the use of different liquid biopsy technologies can yield answers for different research questions.
• The current clinical rationale for ordering liquid biopsy tests -- plus, what indications have been validated by ongoing studies and what this might mean for the future.
• What the pediatric oncology community can do to help accelerate the validation of liquid biopsy technologies to improve outcomes for patients.

Dr. Crompton is a pediatric oncologist and physician scientist focusing on utilizing genomic and proteomic technologies to identify and validate new therapeutic targets for pediatric solid tumors. His aim is to develop non-invasive biomarkers of treatment response and resistance in these diseases. Dr. Crompton’s laboratory has developed new approaches for estimating circulating tumor DNA (ctDNA) levels from liquid biopsy samples in patients with pediatric solid tumors. These assays help track ctDNA levels over time and allow correlation between ctDNA levels, therapeutic response and outcome. His team is also developing strategies for identification, quantification, and interrogation of circulating tumor cells (CTCs) from liquid biopsy samples, including the application of single-cell profiling technologies.

During this lecture, participants will:

• The current efforts to incorporate mutational profiling into the risk stratification of pediatric rhabdomyosarcoma.
• Understanding the assays designed to evaluate cell free DNA and discuss the potential of these tools in the pediatric oncology clinic.
• The NCI-COG partnership known as the Childhood Cancer Data Initiative Molecular Characterization Initiative (MCI).

Dr. Jack Shern is a pediatric oncologist with primary interest in developing effective therapies for children and adults with genetic tumor predisposition syndromes and rare solid tumors. After graduating from the Medical College of Georgia, he completed his pediatric residency at the University of Chicago. He then moved to a combined Pediatric Hematology and Oncology Fellowship training program at the National Cancer Institute, Pediatric Oncology Branch (POB) and Johns Hopkins University.

Following his fellowship he was an assistant clinical investigator and now a Lasker Clinical Research Scholar leading the Tumor Evolution and Genomics Section. Dr. Shern established a translational research program within NCI’s Pediatric Oncology Branch focused on pediatric soft tissue sarcomas. Using cutting edge genomic and molecular profiling techniques, his research focuses on defining and developing precision therapies targeting the genetic mutations that drive tumorigenesis.

Dr. Shern is involved in the Children's Oncology Group, the Cancer Moonshot funded Rare Tumor Initiative, the My Pediatric and Adult Rare Tumor Network (MyPART), and NCI’s Childhood Cancer Data Initiative (CCDI).

During this lecture, participants will:

• Learn how functional genomics has led to the definition of transcriptional circuitries that drive malignant cell identity.
• Understand how these circuitries can be perturbed in cancer cells to force malignant cells to acquire new cell states.
• Learn about new preclinical technologies used to target transcription.

Adam Durbin, MD, PhD is a member of the faculty at St. Jude Children’s Research Hospital in the Division of Molecular Oncology, Department of Oncology and the Developmental Biology and Solid Tumor Program. His team works to understand how transcriptional regulatory circuitries establish malignant cell identity, and how these circuitries can be perturbed for clinical benefit using conventional and novel small molecules.

During this lecture, participants will:

• Learn about the differences between the landscapes of pediatric and adult cancers.
• Learn about the impact of pediatric cancer clinical oncology testing using whole-genome, whole-exome and RNA-seq.
• Gain insight on discovery of non-coding driver variants using a novel algorithm cis-X.
• Get familiar with St. Jude Cloud, the largest pediatric cancer genomic data resource, and the ecosystem of Genomics Platform, Knowledgebase and Visualization.

Jinghui Zhang, PhD, chairs the department of Computational Biology at St. Jude Children’s Research Hospital and holds the St. Jude Endowed Chair in Bioinformatics. Her research in the genomic landscape of pediatric cancer has led to new directions in research involving high-risk leukemia, brain and solid tumors.

After this lecture, participants will:

• Know the features that identify a child/adolescent who is genetically disposed to get cancer (with or without a cancer at the time of evaluation).
• Understand the importance of cancer surveillance in individuals who are genetically predisposed to get cancer.
• Know the 5 most common cancers that occur in individuals with Li-Fraumeni syndrome under the age of 20.

Dr. Brodeur is the Director of the Cancer Predisposition Program at CHOP and Associate Director of the Abramson Cancer Center at Penn. He has a special interest in the molecular biology, genetics and targeted therapy of neuroblastoma. Dr. Brodeur is also a member of ALSF’s Scientific Advisory Board.

After this lecture, participants will:

• Understand strategies for immunotherapy target discovery and validation.
• Understand what makes an “optimal” immunotherapeutic target.
• Understand how one chooses an immunotherapeutic platform to move forward to preclinical and clinical development.

John Maris, MD is the Giulio D’Angio Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania and Children’s Hospital of Philadelphia. He is a physician-scientist who has focused for over three decades on the childhood cancer, neuroblastoma, with dual goals of improving patient outcomes and using the disease as a model to understand cancer in general. His group has discovered all known neuroblastoma susceptibility genes and has also identified many of the oncogenic drivers of the disease. Dr. Maris has steadfastly sought to translate these discoveries to the clinic using precision medicine approaches.

During this lecture, participants will:

• Learn about the contribution of chromatin biology to pediatric brain tumorigenesis.
• Understand therapeutic strategies in epigenetically altered cancers.
• Learn about new technologies in mapping epigenetic landscapes of cancer.

Stephen C. Mack, PhD is an Assistant Professor at Texas Children's Hospital and Baylor College of Medicine. His team studies a variety of high-risk brain tumors to understand the role of deregulated epigenomes as drivers of cancer.

After this lecture, participants will:

• Understand advances made in generating new animal models of fusion-oncogene driven pediatric sarcomas and their potential for therapeutic applications.
• Summarize the value of applying zebrafish rhabdomyosarcoma models for drug discovery, functional genomics, and personalized medicine.
• Understand how fusion-oncogenes co-opt developmental programs for tumorigenesis.

Genevieve C. Kendall, PhD, is a principal investigator in the Center for Childhood Cancer and Blood Diseases and an assistant professor in the Department of Pediatrics at The Ohio State University College of Medicine. Dr. Kendall received her bachelor’s degree in Cell and Molecular Biology at Texas Tech University where she was a Howard Hughes Medical Institute Undergraduate Research Fellow. She obtained her Ph.D. in Molecular Biology at the University of California, Los Angeles (UCLA), before performing her postdoctoral studies focused on pediatric oncology with Dr. James Amatruda at UT Southwestern Medical Center. Dr. Kendall’s research is focused on developing translational models of pediatric sarcomas that delineate the biology of the disease with the goal of identifying new therapeutic opportunities.

After this lecture, participants will:

• Understand what models are currently in the ALSF/COG repository and examples of studies that have been done with those models.
• Learn about efforts to establish and characterize additional models.
• Understand the advantages and disadvantages of various models and the potential for negative impact of poor model choice on experimental results.

Dr. Reynolds grew up in El Paso, TX, received his BA in Biology from The University of Texas at Austin, his MD from UT Southwestern Medical School in Dallas, TX, his PhD (Cell Biology) from UT Austin, and his pediatrics training at the National Naval Medical Center. His postdoctoral fellowship was in cancer immunology at UT Southwestern Medical School, Dallas, TX. He holds the rank of Commander, US Navy (retired). He is currently the Cancer Center Director for the School of Medicine, Texas Tech University Health Sciences Center, and is Director of the South Plains Oncology Consortium and the ALSF/COG Childhood Cancer Repository.

After this lecture, participants will:

• Be able to summarize what is known and unknown about the social difficulties of pediatric brain tumor survivors.
• Understand the role of two key social information processes in the social functioning in pediatric brain tumor survivors.
• Identify areas of diminished neural activity in the social brain of pediatric brain tumor survivors during social processing.

Matthew C. Hocking, PhD is a pediatric psychologist who works with the Division of Oncology at CHOP and is an Assistant Professor of Clinical Psychology at the Perelman School of Medicine at the University of Pennsylvania. Dr. Hocking’s research aims to better understand the neurodevelopmental consequences of having survived childhood cancer or having neurofibromatosis type 1, to identify those who are most at risk for poor outcomes, and to intervene in some way in order to improve quality of life.

During this lecture, participants will:

• Learn about the overall proportion of childhood cancer patients that have germline susceptibility
• Understand the specific relationships between tumor histopathology and the likelihood of identifying a variant in cancer susceptibility genes
• Learn about the unexpected relationship between previously identified adult cancer susceptibility genes and childhood cancer

Sharon E. Plon, MD, PhD is a Professor at Texas Children's Hospital and Baylor College of Medicine. She has worked on several collaborative clinical studies that investigated the development and implementation of clinical genomic tests (germline and tumor) for pediatric cancer projects. Of note, her work in germline cancer genomics was sparked by being awarded an ALSF Innovation Award.

During this lecture, participants will:

• Understand clonal dynamics in blood stem cells
• Define new techniques for cellular barcoding
• Examine preleukemic states for clonal dynamics

Dr. Zon is the Grousbeck Professor of Pediatric Medicine at Harvard Medical School, Investigator at Howard Hughes Medical Institute, and Director of the Stem Cell Program at Boston Children’s Hospital.  Dr. Zon received his B.S. in chemistry and natural sciences from Muhlenberg College (1979) and his M.D. from Jefferson Medical College (1983).  He subsequently did an internal medicine residency at New England Deaconess Hospital (1986) and a fellowship in medical oncology at Dana-Farber Cancer Institute (1989). His postdoctoral research was in Stuart Orkin’s laboratory (1990).  Dr. Zon is internationally recognized for his pioneering work in stem cell biology and cancer genetics. He has been the preeminent figure in establishing zebrafish as an invaluable genetic model for the study of the blood and hematopoietic development.  He is founder and former president of the International Society for Stem Cell Research and chair of the Executive Committee of the Harvard Stem Cell Institute.  In 2005, he completed a term as President of the American Society for Clinical Investigation.  That same year, Dr. Zon was elected to the Institute of Medicine of the National Academies.   In 2008, Dr. Zon was elected to the American Academy of Arts & Sciences.  In 2010, Dr. Zon was awarded the E. Donnall Thomas Lecture and Prize from American Society of Hematology.  In 2013, Dr. Zon received the ISEH Donald Metcalf Lecture Award.  Other recent awards include the 2014 Boston Children’s Hospital Post-Doctoral Association Mentoring Award and the National Cancer Institute’s Alfred G. Knudson Award (2015).

During this lecture, participants will:

• Describe a conceptual difference between classical machine learning techniques and deep learning.
• Describe the difference between supervised and unsupervised machine learning.
• Identify at least two major challenges that biomedical data, and in particular pediatric cancer data, pose for the successful application of deep learning.

Casey S. Greene, PhD is an Associate Professor at the Perelman School of Medicine at the University of Pennsylvania and the Director of the Childhood Cancer Data Lab for Alex's Lemonade Stand Foundation. His research focuses on harnessing large collections of biomedical data to discover new, physiologically-relevant patterns.

During this lecture, participants will:

• Learn about extracting biologically relevant patterns from high-throughput biomedical data
• Understand obstacles to using data-intensive machine learning techniques in pediatric cancer research
• Learn about leveraging large, arbitrary collections of transcriptomic data to study rare diseases such as pediatric cancers

Jaclyn N. Taroni, PhD is the Principal Data Scientist at the Childhood Cancer Data Lab of Alex's Lemonade Stand. Her team develops robust and reusable workflows to study pediatric cancer and trains pediatric cancer researchers to better analyze their own data.

Upon completion of this lecture, participants will:

• Understand the current molecular subtypes of ALL.
• Understand the diversity of genetic alterations that drive leukemogenesis.
• Learn diagnostic and therapeutic implications of the revised taxonomy of ALL.

Charles Mullighan, MD is a member of the Department of Pathology, co-leader of the Hematologic Malignancies Program, Deputy Director of the Comprehensive Cancer Center and Director of the Biorepository at St. Jude Children’s Research Hospital. He gained his medical degree from the University of Adelaide, Australia, undertook doctoral studies in immunogenetics in Oxford, and specialist training in hematology and hematopathology at the Institute of Medical and Veterinary Science in Adelaide. He joined St. Jude as a postdoctoral fellow in 2004, and joined faculty in 2008.
 
His research examines the genetic determinants of leukemogenesis and treatment response in acute lymphoblastic leukaemia (ALL) and high risk leukemias. His work has defined the genetic pathogenesis of multiple new subtypes of ALL, and several genetic alterations that have entered the clinic as new diagnostic and therapeutic targets. He is the recipient of numerous awards including a National Cancer Institute Outstanding Investigator Award, the Meyenburg Prize for Cancer Research and the American Society of Hematology Dameshek Prize.

During this lecture, participants will:

• Learn about the Pediatric Brain Tumor Atlas Initiative of the Children’s Brain Tumor Tissue Consortium approach to accelerated discovery via open science.
• Learn about the available data resources and analyses associated with the Pediatric Brain Tumor Atlas Initiative
• Learn about how such approaches can provide for new clinical translation and biologically based therapies for pediatric brain tumors.

Adam Resnick is the Director of Data Driven Discovery in Biomedicine (D3b) at Children’s Hospital of Philadelphia (CHOP), responsible for leading a multidisciplinary team to build and support a scalable, patient-focused healthcare and educational discovery ecosystem on behalf of all children. He is also responsible for all scientific and fiscal responsibility for projects within the Center, including center leadership, supervision of bioinformatics, genomics, visualization tools and software development for the data resource portal shared by various center initiatives.
 
Adam’s research is focused on defining the cell signaling mechanisms of oncogenesis and tumor progression in brain tumors. His research lab studies cell signaling cascades and their alterations in pediatric brain tumors to elucidate the molecular and genetic underpinnings of each tumor to identify and develop targeted therapies. Adam serves as Scientific Chair for several consortia-based efforts, including the Children’s Brain Tumor Tissue Consortium (CBTTC) and Pacific Pediatric Neuro-Oncology Consortium (PNOC), which include more than 20 pediatric hospitals across the globe.
 
Adam earned a dual bachelor’s degree in Neuroscience and English & Literature from the University of Florida before completing a PhD in Neuroscience from Johns Hopkins University in Baltimore, MD.

During this lecture, participants will:

• Learn about the current state of pediatric cancer research that inspired the creation of the Pediatric Cancer Data Commons (PCDC).
• Understand how the PCDC fosters consensus-based decision-making and data sharing through its structure and governance.
• Gain insights into how the PCDC fits into the Cancer Research Data Commons ecosystem and WHO Initiative for Childhood Cancer.

Samuel Volchenboum, MD, PhD is a pediatric oncologist and Associate Professor of Pediatrics at the University of Chicago. He is Dean for Master’s Education and the Director of the Pediatric Cancer Data Commons. His group develops ways to collect, harmonize, and share data, focusing on building international consensus for data standards. His team also develops novel ways to present data to researchers, allowing users to search and visualize data and make connections to other data sources.

During this lecture, participants will:

• Review causes of somatic mutations in developing tissues.
• Define domesticated DNA transposases and their evolutionary origins and physiologic functions.
• Outline current and future research to translate these insights into improved therapies for patients.

Alex Kentsis, MD, PhD is Associate Member at the Sloan Kettering Institute and Associate Professor of Pediatrics, Pharmacology, and Physiology & Biophysics at the Weill Medical College of Cornell University, and pediatric oncologist at the Memorial Sloan Kettering Cancer Center. His research, funded by the ALSF, has led to new clinical trials for children and adults with high-risk leukemias and solid tumors.

During this lecture, participants will:

• How T cells recognize and attack brain tumors.
• The tricks tumors use to avoid being recognized.
• Strategies we can use to circumvent these tricks and promote tumor rejection.

Robert J. Wechsler-Reya, Ph.D., is Professor and Director of the Tumor Initiation and Maintenance Program at the Sanford Burnham Prebys Medical Discovery Institute in La Jolla, CA, and Director of the Clayes Center for Neuro-Oncology and Genomics at Rady Children’s Institute for Genomic Medicine.  His research focuses on understanding the origins and pathogenesis of the pediatric brain tumor medulloblastoma, and on developing safer and more effective therapies for the disease.

During this lecture, participants will:

• Describe epigenetic changes as a hallmark of cancer.
• Understand how hijacking the epigenome such as changes in enhancers alter the landscape of tumors.
• Explain how mutations in oncohistones and chromatin modifiers promote tumorigenesis.
• Understand the limitations of current technologies and explore the possibility of novel mechanisms.

Nada Jabado, MD, PhD is a Professor of Pediatrics and Human Genetics at the Montreal Children's Hospital and McGill University. Her team studies the role of histones in pediatric brain tumors and how these affect the genome and the epigenome.

During this lecture, participants will:

• Understand how sequencing technologies are being adopted to diagnose bloodstream infections in pediatric cancer patients.
• Understand the impact of genetic heterogeneity in pediatric cancers on treatment resistance.
• Learn the fundamental technological advances that have enabled the use of single-cell genomics to study genetic heterogeneity in pediatric cancers.

Dr. Charles Gawad is an Associate Professor of Pediatrics and the Taube Distinguished Scholar for Pediatric Cancer Research at Stanford University where he is a practicing physician-scientist. His lab works at the interface of biotechnology, computational biology, cellular biology, and clinical medicine to develop and apply new tools for characterizing genetic variation across single cells within a tissue with unparalleled sensitivity and accuracy. He received his medical degree from the University of Arizona and PhD in Cancer Biology from Stanford. Among a number of honors, he is a recipient of a Career Award for Medical Scientists from the Burroughs Wellcome Fund, NIH Director’s New Innovator Award and was appointed an Investigator by the Chan Zuckerberg Biohub.

During this lecture, participants will:

• Learn about the different forms of immunotherapy being deployed to fight pediatric cancers.
• Understand the challenges facing development of CAR T cells for solid tumors.
• Learn about new ways to effectively target GD2 in neuroblastoma and osteosarcoma.

Robbie Majzner, MD is an Assistant Professor at Stanford University School of Medicine. His team focuses on the development of novel immunotherapies for pediatric cancers.

During this lecture, participants will:

• Understand how DIPG cells harbor aberrant chromatin/transcriptional states that can be shifted by epigenetic modulators.
• Understand how DIPG cells are activated by direct and indirect contacts with neurons and their electric activity.
• Learn about strategies for blocking both intrinsic and extrinsic pathways necessary for DIPG growth.

Mariella G. Filbin, MD, PhD, is a Pediatric Neuro-Oncologist and Principal Investigator at Dana-Farber Cancer Institute and Boston Children’s Hospital, and an Assistant Professor in Pediatrics at Harvard Medical School. Her research focuses on pediatric brain tumors, particularly the high-grade gliomas and malignant embryonal brain tumors that are in greatest need of therapeutic improvements. In her studies, she is combining single-cell genetics and transcriptomics with gene editing, epigenetic, stem cell, and pharmacologic methods to identify networks underlying tumorigenesis, with the goal of establishing new druggable targets.

Michelle Monje, MD, PhD, is an associate professor of neurology at Stanford University. She is a neuroscientist and pediatric neuro-oncologist focused on caring for children and young adults with cancers of the brain and spinal cord. She received her MD and PhD in Neuroscience from Stanford, training in neurology at Massachusetts General Hospital/Brigham and Women’s Hospital/Harvard Medical School and in pediatric neuro-oncology at Stanford University. Her research program focuses at the intersection of neuroscience and brain cancer biology, seeking to elucidate how brain cancers interact with the normal brain, and how to develop better treatments for brain cancer based on these new insights.

During this lecture, participants will:

• Learn how epidemiologists conceive of and determine causality.
• Understand the study designs and data are available to understand causation in childhood cancer.
• Gain an overview of the relative contribution of genes and environment in causing childhood cancer.

Logan G. Spector, PhD is Professor of Pediatrics at the University of Minnesota and Suzanne Holmes Hodder Chair in Pediatric Cancer Research. He and his Division study the causes of childhood cancer using both classic and genetic epidemiology. He is immediate past Chair for Epidemiology with the Children's Oncology Group and current Chair of the Childhood Cancer and Leukemia International Consortium (CLIC).

Cigall Kadoch, PhD is an Associate Professor of Pediatric Oncology at the Dana-Farber Cancer Institute, Affiliate Faculty of Biological Chemistry and Molecular Biology at Harvard Medical School, and Institute Member and Epigenomics Program Co-Director at the Broad Institute of MIT and Harvard.

During this lecture, participants will:

• Learn about some historic clinical trial design and efforts in pediatric central nervous system tumors.
• Gain exposure to recent precision-based trials for pediatric central nervous system tumors and some of their findings and contributions to ongoing or upcoming trials.
• Expand their understanding of novel clinical trial designs and combination therapies that are underway for the treatment of children and young adults with central nervous system tumors.

Cassie Kline, MD is the Director of Neuro-Oncology Clinical Research at the Children’s Hospital of Philadelphia (CHOP). Dr. Kline completed her residency in pediatrics at CHOP followed by a fellowship in pediatric hematology-oncology at the University of California San Francisco, where she also completed a Master’s in Clinical Research and subsequently served on the faculty. Her focus is clinical research and trial development for pediatric brain tumors. She currently leads several clinical trials dedicated to novel therapies for children and young adults with central nervous system tumors, serves as the Director of Data Quality and Integration for the Pacific Pediatric Neuro-Oncology Consortium (PNOC), and co-leads the PNOC and Children’s Brain Tumor Network (CBTN) craniopharyngioma working group.

During this lecture, participants will:

• Understand evidence linking elevated LIN28 levels to disease severity and poor prognosis in NB.
• Learn about in vitro and mouse models linking LIN28 to metastasis and phenotypes of cell migration and invasion.
• Understand mechanisms linking LIN28 to translational control.

George Q. Daley, dean of Harvard Medical School and the Caroline Shields Walker Professor of Medicine, is a physician-scientist and authority on stem cell science and cancer biology. Important contributions from the Daley laboratory include the creation of customized stem cells to treat genetic immune deficiency in a mouse model, the generation of disease-specific pluripotent stem cells by direct reprogramming of human fibroblasts, and demonstration of the role of the LIN28/let-7 pathway in cancer.

During this lecture, participants will:

• Learn about the role of CAR-T cell therapy in relapsed, refractory, and/or high risk ALL.
• Understand the risk factors and treatment for Cytokine Release Syndrome (CRS).
• Understand the importance of T cell proliferation and persistence in CAR-T cell efficacy.

Stephan A. Grupp, MD/PhD is the Chief of the Cellular Therapy and Transplant Section, Director of the Cell Therapy and Cancer Immunotherapy Program, and Medical Director of the Cell and Gene Therapy Lab at CHOP, as well as the Novotny Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. His primary area of research is the use of CAR T and other engineered cell therapies in pediatric cancers and other life threatening disorders such as sickle cell disease.

During this lecture, participants will:

• Learn about the development of small molecule inhibitors and degraders for epigenetic proteins.
• Learn about how to reveal mechanism of epigenetic protein function in pediatric cancers using chemical biology approach.
• Learn about the landscape on chemical probe development on targeting epigenetic proteins.

Jun Qi, PhD is an Assistant Professor at Dana-Farber Cancer Institute and Harvard Medical School. His team studies a variety of epigenetic proteins in cancers, including pediatric cancers via a combined chemical biology and biology approach.

During this lecture, participants will:

• Learn about the role of macrophages and phagocytosis checkpoint in brain tumor malignancies.
• Understand unique immune suppressive mechanisms in pediatric brain tumor malignancies.
• Therapeutic strategies to make brain tumors more immunogenic.

Siddhartha “Sid“ Mitra PhD is an assistant professor of Pediatrics at the University of Colorado School of Medicine and a Principal Investigator in the Morgan Adams Pediatric Brain Tumor Research Program of The Children’s Hospital in Colorado. His lab studies the immune microenvironment of high-grade brain tumors and focused on developed innate immune modulation therapies against pediatric malignancies.

During this lecture, participants will:

• Understand the current potential benefits and limitations of clinical genomics in pediatric oncology.
• Understand the rationale for and concept of a Pediatric Cancer Dependency Map.
• Learn about examples of new pediatric cancer dependencies emerging from the Pediatric Cancer Dependency Map.
• Learn about approaches to target validation, such as CRISPR-Cas9, shRNA, and dTAG systems.

Kimberly Stegmaier, MD, Professor of Pediatrics at Harvard Medical School and the Ted Williams Chair at Dana-Farber Cancer Institute, has advanced the application of genomics to drug and protein target discovery for pediatric malignancies. She is the Vice Chair for Pediatric Oncology Research, Co-director of the Pediatric Hematologic Malignancy Program, and an Institute Member of the Broad Institute. She has served as a Council Member with the Society for Pediatric Research and as the Chair for the AACR Pediatric Cancer Working Group.

During this lecture, participants will:

• Learn the molecular analysis of pediatric medulloblastoma and vulnerabilities.
• Learn about the development of models that recapitulate the patient’s diseases.
• Learn how models allow to identify new therapies using a pre-clinical pipeline.

Martine F. Roussel, PhD is a full member and Professor at St. Jude Children’s Research Hospital and holds the position of Endowed Chair in Molecular Oncology. She’s published ~ 250 publications. She is a Fellow of the American Academy of Arts & Sciences, one of the 50 top ranked personalities in the USA, Mentor of the Year, a member of the National Academy of Sciences and Fellow of the American Association of Cancer Research (FAACR). She’s made landmark discoveries in molecular oncology, cell cycle control, and translational development of novel strategies for the treatment of pediatric medulloblastoma. Her team studies pediatric brain tumors including medulloblastoma to develop novel therapies for the most aggressive forms of the disease.