Mon, 2/20

7:30am – 8:30am

Breakfast

8:30am – 9:00am

Welcome/Opening Remarks:
William L Henrich, MD, MACP

President, UT Health San Antonio
Professor of Medicine

William L. Henrich, M.D., MACP, a specialist in kidney diseases, has served as the president of UT Health San Antonio since 2009. UT Health San Antonio is one of six health institutions under the umbrella of The University of Texas System.

UT Health is a dynamic and rapidly expanding health science center with five professional schools (medicine, nursing, dentistry, health professions, and graduate school of biomedical sciences) with education, clinical care, research, and community service missions. With a budget of nearly one billion dollars, a workforce of 7,100, and a research portfolio of approximately $300 million, UT Health is quickly rising in prominence among academic medical centers in the United States.

Henrich received his undergraduate degree from Columbia University and his medical degree from Baylor College of Medicine and later completed a residency in Internal Medicine at The University of Oregon Medical School and a fellowship in Nephrology at The University of Colorado School of Medicine.

During his career, Henrich has served as Professor of Medicine at The University of Texas Southwestern School of Medicine, as Professor and Chair of Medicine at the Medical College of Ohio, and as the Theodore Woodward Professor and Chairman of the Department of Medicine at The University of Maryland School of Medicine in Baltimore. He became the Dean of the School of Medicine and Vice President for Medical Affairs at UT Health San Antonio in 2006 prior to being selected as its President in 2009. He is the inaugural holder of the John P. Howe, III, M.D., Distinguished Chair in Health Policy.

He is the author of over 300 original articles and chapters and the founding editor of the popular dialysis textbook, “Henrich’s Principles and Practice of Dialysis”. His current areas of interest are in improving dialysis and vascular renal disease.

An elected member of several prominent research societies, Henrich also served as President of the American Society of Nephrology. He is passionate about mentoring students, residents, and young physicians and has received teaching accolades in every institution in which he has served.

9:00am – 10:30am

Session 1: Targeted Therapy

D.Will Parsons, MD, PhD
Baylor College of Medicine
Presentation: “Targeted Therapies for Refractory Pediatric Cancers: the MATCH Experience

About Dr. Parsons: Dr. Will Parsons, MD, PhD, is a board-certified pediatric oncologist and the Sidney L and Donald F. Faust Chair of Pediatric Cancer Precision Medicine at Texas Children’s Cancer and Hematology Center (Baylor College of Medicine, Houston, Texas). Dr. Parsons’ work has been instrumental in characterizing the genetic landscapes of a variety of pediatric and adult cancers, including the first identification of IDH1 and IDH2 as critical oncogenes in gliomas. His current research primarily focuses on the clinical application of genomic technologies in pediatric cancer care. Dr. Parsons has a particular interest in the development and evaluation of molecularly targeted therapies and has a number of leadership roles in this area, including serving as the Children’s Oncology Group (COG) study chair for the NCI-COG Pediatric MATCH trial (the first nationwide precision oncology trial for children with relapsed and refractory solid tumors, lymphomas, and histiocytoses) and as a Steering Committee member for the NIH Pediatric Early Phase Clinical Trials Network.

Angelina Vaseva, PhD
UT Health San Antonio
Presentation: “Targeting RAS in pediatric cancers- are we there yet?”

About Dr. Vaseva: Despite the high cure rates among children with cancer, following treatment with current multimodality therapies, survivors face profound lifelong therapy-related complications and the development of second malignancies, with increasing chronic, life-threatening toxicities as they age. In addition, the survival rate for many high-risk pediatric cancer patients has not improved significantly over the last 30 years. Hopes for the future depend on identifying novel therapies targeting specific cancer-driving cellular and molecular mechanisms, ensuring improved survival for high-risk patients, and providing a better quality of life for survivors.  The focus of the Vaseva lab is to systematically define mechanisms of RAS and MAPK- driven vulnerabilities in pediatric cancers and design directions for novel targeted therapies.

AGENDA NOTE: Due to a scheduling issue, the following speaker from Session Five: Big Data/Omics, will present following Session One.

Jinghui Zhang, PhD
St. Jude
Presentation Title: “Therapy-Related Clonal Evolution in Pediatric Cancer Patients and Long-Term Survivors”

About Dr. Zhang: I am a computational biologist focused on the integrative analysis of large-scale, multi-dimensional genomic data to understand the initiation and progression of diseases. My research interest has been developing highly accurate and sensitive computational methods for analyzing large-scale genomic data, especially in detecting and analyzing genetic variations and somatic mutations.

10:30am – 11:00am

Coffee/Tea Break

11:00am – 12:30pm

Session 2: Clinical Trials

Julia Glade-Bender, MD
Memorial Sloan Kettering
Presentation: “Pediatric  Early Phase Trials: Challenges and Opportunities.”

About Dr. Glade-Bender:

Julia Glade Bender, MD, is a pediatric oncologist and Vice Chairman for Clinical Research within the Department of Pediatrics at Memorial Sloan Kettering Cancer Center (MSK Kids).  She has accumulated over 20 years of experience leading highly productive pediatric experimental therapeutics and early drug development programs. Her primary translational work has been on developing targeted agents for childhood solid tumors and the clinical implementation of precision oncology. She has provided the broad scientific leadership for early-phase clinical trials sponsored by the National Cancer Institute (NCI), the Children’s Oncology Group (COG), as well as smaller disease-oriented consortia and the pharmaceutical industry. She is a consultant to the Pediatric Oncologic Drugs Advisory Committee (ODAC) of the Food and Drug Administration (FDA), sits on the Steering Committee for the Pediatric NCI-MATCH (Pediatric Molecular Analysis for Therapy Choice), and currently serves as the COG Study Chair of the NCI-ComboMATCH.

Richard Gorlick, MD
MD Anderson
Presentation: “The Future of Trickle-Down Drug Development for Pediatric Cancer”

About Dr. Gorlick: The research in my lab is focused on conducting translational studies aimed at improving outcomes for children, adolescents, and young adults with osteosarcoma, the most common primary malignant bone tumor in this patient population. Utilizing genomic and proteomic approaches, our lab identifies novel targets in osteosarcoma ripe for therapeutic intervention with the goal of developing new immunotherapies to be assessed in pre-clinical studies and clinical trials.

Brenda Weigel, PhD
Univ of Minnesota
Presentation: “Pediatric  Early Phase Trials: Challenges and Opportunities”.

About Dr. Weigel: Dr. Brenda Weigel is currently the Director of the Division of Pediatric Hematology/Oncology. She is a professor cross-appointed at the University of Minnesota’s Cancer Center and the Department of Pediatrics and the recipient of the Lehman/Children’s Cancer Research Fund Endowed Chair in Pediatric Cancer. She is also the Co-Director of the Sarcoma Program for the Masonic Cancer Center and an Associate Director of the Cancer Experimental Therapeutics Initiative for the Masonic Cancer Center.

12:30pm – 1:30pm

Lunch

1:30pm – 3:00pm

Session 3:
Current Advances in Pediatric Solid Tumors Therapy

Patrick Grohar, MD, PhD
CHOP
Presentation: “The Importance of Mechanistic Pharmacology to the Clinical Translation ofTargeted Therapy” 

About Dr. Grohar: The goal of the Grohar lab is to develop effective targeted therapies for pediatric solid tumors. We believe in an integrated bench-to-bedside and back-again approach that utilizes mechanistic pharmacology as a bridge between discovery science and the clinic. Our lab utilizes unbiased screens to identify novel targeted agents and drug combinations. We then focus on the mechanism of target suppression to identify novel combination therapies and translate the observations to the in vivo setting. Finally, we design clinical trials with pharmacokinetic/pharmacodynamic biomarkers of target suppression and correlative biology that further inform our preclinical studies.

Poul Sorensen, MD, PhD
Univ of British Columbia
Presentation: “Translational control of aggressive behavior in high-risk childhood sarcomas”

About Dr. Sorenson: Dr. Sorensen is an internationally renowned expert in cancer biology and genetics, mRNA translation, and the use of biochemical and proteomic strategies to characterize deregulated signaling pathways in childhood and adult solid cancers. His group has discovered many genetic alterations in childhood cancer, including the EWS-ERG fusion in Ewing sarcoma, loss of the HACE1 tumor suppressor in Wilms’ tumor, and the ETV6-NTRK3 fusion in infantile fibrosarcoma and secretory breast carcinoma. The latter is the first description of NTRK fusions as recurrent drivers in human tumors, now thought to occur in >25 different human tumor types and in ~1% of human malignancies, leading to the development and worldwide approval of NTRK inhibitors for clinical use. His research utilizes biochemical methods, including mRNA translational profiling and proteomic strategies, to identify and characterize cell stress pathways that are deregulated in childhood and adult solid cancers.

Jason Yustein, MD, PhD
Emory University
Presentation: “Implementing Novel Mouse Models to Identify and Test New Therapies for Pediatric Solid Tumors”

About Dr. Yustein: Our laboratory is dedicated to understanding mechanisms of metastasis and therapeutic resistance by using our experience integrating novel murine and patient-derived models with molecular approaches and functional genomic studies geared towards identifying new treatment avenues for the treatment of sarcomas.

3:00pm -3:30pm

Coffee/Tea Break

3:30pm – 5:00pm

Session 4: Challenges in Immunology & Immunotherapy

Julie Bailis, PhD
Amgen, Inc.
Presentation: “AMG 509, a STEAP1-Targeting T Cell Engager for Ewing Sarcoma.”
About Dr. Bailis:  Julie Bailis, Ph.D., is an executive director at Amgen, Inc, where her team is focused on developing multispecific immune therapies, including T cell engager molecules. Julie obtained her PhD from Yale University and was a postdoctoral fellow at the Salk Institute for Biological Studies.

Yaël P. Mossé, MD, PhD
CHOP
Presentation: “ALK Inhibition for Childhood Cancers: Neuroblastoma as a Model”

About Dr. Mosse: Dr. Mossé’s lab research focuses on the genetic mutations responsible for neuroblastoma. The team recently discovered that a region of chromosome 2 was associated with the disease and identified mutations in the anaplastic lymphoma kinase (ALK) gene. ALK is an oncogene (cancer-causing gene) with genetic and acquired mutations. Many pharmaceutical companies already make drugs that turn off the ALK gene, so Dr. Mossé’s team is now working on translating its discovery to the therapeutic use of ALK-inhibiting drugs.

Manjeet Rao, PhD
UT Health San Antonio
Presentation: “RNA Demethylase: A Critical Regulator of Anti-Tumor Immunity in Osteosarcoma”

About Dr. Rao: The overall goal of my laboratory is to develop more potent and less toxic drugs for treating adult and pediatric cancer patients. We have employed unbiased high throughput genomewide functional screens and small molecular screens to identify novel targets critical for growth, progression, and drug sensitivity in medulloblastoma and osteosarcoma. In particular, using the loss of function screens, we have identified key genes that play critical/causal roles in the growth and metastasis as well as in the chemo-sensitivity of osteosarcoma. Furthermore, by performing small molecule library screens, we have identified inhibitors of these proteins that may act as novel therapeutics for treating osteosarcoma.

5:00pm -6:00pm

Poster Previews & Poster Blast

(1min “elevator speech” of each poster)

6:00pm -6:30pm

Wine & Cheese Mixer

6:45pm -9:00pm

SAPCS Banquet
Mariachi Melodias De Mex​ico​

Tues, 2/21

7:30am-8:30am

Breakfast

8:30am-9:30am

Keynote Speaker

John M. Maris, MD
CHOP
Presentation: “Recent advances in the immunotherapeutic targeting of childhood cancer oncoproteins.”

About Dr. Maris: Dr. John Maris is Giulio D’Angio, a Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania and the Children’s Hospital of Philadelphia. He is a physician-scientist who has focused for over three decades on childhood cancer neuroblastoma with the dual goals of improving patient outcomes and using the disease as a model to understand cancer in general. His group has discovered all the known neuroblastoma susceptibility genes and his group 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.

Over the last decade, he has led a multi-institutional St. Baldrick’s Foundation-Stand Up to Cancer Pediatric Cancer Dream Team project to bring the fields of genomics and immunology together to combat childhood cancers, and more recently a Beau Biden Moonshot Center Award to extend this rapidly evolving area of research. Dr. Maris is an internationally recognized practicing pediatric oncologist who cares for children with refractory neuroblastoma from around the world, typically in the context of early-phase clinical trials.

The National Institutes of Health and many other funding bodies have continuously funded Dr. Maris. He currently holds a National Cancer Institute Outstanding Investigator Award and has received several prestigious awards, including election into the American Society of Clinical Investigation, the Oski award for outstanding pediatric oncologists, the Berwick award at Penn for melding basic and clinical teaching, the William Osler Patient-Oriented Research Award at Penn, and the AACR 2021 Team Science Award for his leadership in pediatric immunoncology research.

9:30am-10:00am

Coffee/Tea Break

10:00am-11:30am

Session 5: Big Data/Omics

Adam Resnick, PhD
CHOP
Presentation: “Data Platforms and Ecosystems: The Emerging Pediatric Brain Tumor Data Landscape”

About Dr. Resnick

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 accelerated discovery and clinical translation for all children.  The D3b Center comprises a trans-disciplinary team that spans the clinical research unit, biospecimen research unit, molecular diagnostics research unit, pre-clinical research unit, bioinformatics unit, translational imaging unit, and the advanced data applications and platform technologies unit.

Yang Xie, PhD
UT Southwestern
Presentation: “CPRIT Pediatric Cancer Data Core”

About Dr. Xie: Dr. Yang Xie received a BMedSc from Peking University Health Science Center in 2000 and a PhD in Biostatistics from the University of Minnesota in 2006. She is a Professor with a joint appointment in the Department of Population and Data Sciences and the Department of Bioinformatics at UT Southwestern Medical Center. Her research focuses on algorithm development, machine learning, and data integration for biomedical research. She is the founding director of the Quantitative Biomedical Research Center and the Pediatric Cancer Data Commons (PCDC) at UT Southwestern Medical Center. She is also a member of the NIH Biodata Management and Analysis Study Section [BDMA].

AGENDA NOTE: Due to a scheduling issue, the following speaker from Session One: Targeted Therapy, will present right after Session Five. 

William Marsiglia, PhD
UAB
Presentation “A Toolkit to Measure the Effect of Protein-Protein Interactions on MAPK and PI3K-AKT Inhibitor Pharmacology”

Billy received a BA in Music and a BS in Biochemistry from SUNY Binghamton in 2013. He then earned his PhD in Chemistry at NYU using NMR spectroscopy to explore the activation mechanisms underlying pathogenic FGFR kinase domain mutations. During his postdoctoral work at the Icahn School of Medicine at Mount Sinai starting in 2019, Billy developed a NanoBRET assay that helped to elucidate the mechanism of action for the MEK inhibitor, Trametinib. In 2022, Billy joined the Department of Pharmacology and Toxicology at the University of Alabama at Birmingham.

11:30am – 12noon

Short Talk Session One

Format: 10min presentation + 5min Q&A

Short Talk A:
Nourhan Abdelfattah, PhD
Houston Methodist Research Institute
Presentation: “Sex Differences in Yap1 Oncogene Function in Medulloblastoma Immune Evasion”

Short Talk B:
Katsumi Kitagawa, PharmD, PhD
UT Health San Antonio
Presentation: “EWSR1 (Ewing Sarcoma Breakpoint Region 1) Maintains Centromere Identity”

12noon – 1pm

Lunch

1:00pm – 2:30pm

Short Talk Session Two

Format: 10min presentation + 5min Q&A

Short Talk A:
David M. Loeb, M.D., Ph.D.
Albert Einstein College of Medicine
Presentation: “Polyamine Depletion Inhibits Ewing Sarcoma Metastasis by Inducing Ferroptosis.”

Short Talk B:
Emily Selig, PhD
UT Health San Antonio
Presentation: “Liquid-Liquid Phase Separation of the Oncogenic Fusion Protein EWS-FLI1 is Modulated by its DNA-binding domain”

Session 6: Epigenetics/Transcriptional Dysregulation

Miguel N. Rivera, MD
Massachusetts General Hospital, Harvard Medical School
Presentation: “Mechanisms of chromatin regulation by EWS oncogenic fusion proteins”

Agata Smogorzewska, MD, PhD
Rockefeller University
Presentation: “Mechanism of Tumorigenesis in Fanconi Anemia”

About Dr. Smogorzewska: Using Fanconi anemia and other genetic diseases as a backdrop, Dr. Smogorzewska’s research aims to elucidate pathways that prevent stem cell dysfunction, and cancer development, with a focus on those that repair DNA. Throughout its lifetime, a cell’s DNA is under constant metabolic and environmental assault that can lead to damage. If left unchecked, the resulting genome instability can initiate cancer and a variety of other human disorders. Using Fanconi anemia and other genetic diseases as a backdrop, Smogorzewska’s research aims to elucidate the pathways that protect organ function and prevent cancer, with a focus on those that replicate and repair DNA.

2:30pm – 3:00pm

Coffee/Tea break

3:00pm -4:00pm

Session 7: Disparities in Pediatric Cancer

Amelie G. Ramirez, Dr. P.H., M.P.H.
UT Health San Antonio
Presentation:
Exploring the Latino Cancer Burden in South Texas

About Dr. Ramirez: Dr. Amelie G. Ramirez is director of the Institute for Health Promotion Research (IHPR), chair of the Department of Population Health Sciences, and associate director of cancer outreach and engagement at the Mays Cancer Center, all at UT Health San Antonio. The IHPR, founded in 2006, investigates causes and solutions to cancer and chronic disease disparities to improve Latino health in San Antonio, South Texas, and the nation. The IHPR has a faculty and staff of researchers who specialize in population health, prevention and screening, and health promotion and communication on issues of obesity, cancer, tobacco, nutrition, physical activity, and more among Latino and underserved populations.

Phil Lupo, PhD, MPH
Baylor College of Medicine
Presentation:
Ethnic disparities in acute lymphoblastic leukemia susceptibility and outcomes.

About Dr. Lupo: Dr. Lupo is a Professor in the Department of Pediatrics at Baylor College of Medicine, Director of the Epidemiology and Population Sciences Program at Texas Children’s Cancer and Hematology Center, Chair of the Children’s Oncology Group (COG) Epidemiology Committee and has served in various capacities in the National Birth Defects Prevention Network (NBDPN), including President and Chair of the Data Committee. Dr. Lupo’s research is focused on characterizing susceptibility to pediatric cancer and limiting outcome disparities for children diagnosed with cancer.

.

4:00pm – 4:30pm

Short Talk Session Three

(Format: 10min presentation + 5min + Q&A) 

Short Talk A:
Sarah Lucht, University of Minnesota
Presentation Title: “Disparities in Trial Enrollment for Hepatoblastoma and Wilms Tumor Patients by Age at Diagnosis and Distance to Care: a Report from the Children’s Oncology Group”

Short Talk B:
Anna Seffernick, St. Jude Children’s Research Hospital
Presentation Title: “Bootstrap Evaluation of Association Matrices (BEAM) for Integrating Multiple Omics Profiles with Multiple Outcomes” 

4:30pm – 5:30pm

Poster Session, Odd #s

5:30pm – 6:30pm

Poster Session, Even #s

Wed, 2/22

7:30am-8:30am

Breakfast

8:30am-10:00am

Session 8: Transcription Factors, Structure to Function in Pediatric Cancer

Martin Eilers, PhD
Univ of Wurzburg
Presentation: “Sensing Aberrant Transcription by the MYCN Oncoprotein in Neuroblastoma”

About Dr. Eilers: Many cancers are life-threatening diseases, and there is an urgent need for novel therapeutic strategies. The Eilers lab works on the human MYC protein family, which is involved in the development of most human cancers. Our aim is both to understand the function of MYC proteins and explore new strategies to inhibit their function

Marcel Kool, PhD
Children’s Brain Tumor Network (Germany)
Presentation: ITCC-P4 Single-cell analyses of embryonal brain tumors provide new insights into cellular hierarchies, drug targets, and the tumor micro-environment.

Over the years, we got quite a good idea of what is driving most of these less frequent but often aggressive brain tumors. However, there is still much to learn from further (epi)genomic analyses using state-of-the-art technologies, including, for instance also, single-cell omics, as the main drivers and/or therapeutic targets are not clearly defined in all tumors. Therefore, the aim is to characterize these better, identify clinically relevant and distinct molecular subgroups and the genes and pathways that drive them, and, most importantly, develop proper human and mouse model systems reflecting the different diseases and the inter-and intra-tumor heterogeneity. Additionally, we strive to find novel candidates for rational targeted therapies. We aim to translate knowledge obtained from these (epi)genomic studies into novel strategies for the most optimal treatments of patients.

Kajsa Paulsson, PhD
Professor, Division of Clinical Genetics
Research team manager, Aneuploidy in cancer
Principal investigator, LUCC: Lund University Cancer Centre
Presentation: “The role of CTCF and cohesin in hyperdiploid pediatric acute lymphoblastic leukemia”

Dr. Paulsson is a Professor of Medical Genetics at the Division of Clinical Genetics, Lund University, Sweden. Her research is focused on understanding how aneuploidy, i.e., changes in chromosome numbers, affect leukemogenesis. The overall aims are to understand how pediatric acute lymphoblastic leukemia gains and loses chromosomes, how changes in chromosome numbers affect the leukemic cells, and whether the aneuploidy renders leukemia susceptible to novel treatments.

10:00am-10:30am

Coffee Break

10:30am-12:00pm

Session 9: Policy & Advocacy in Childhood Cancer/Survivorship

Wendy Darlington, MD, MAPP
Univ of Chicago

Wendy Darlington, MD, MAPP, is an Assistant Professor in the Department of Pediatrics at the University of Chicago and a member of the Chicago Sickle Cell Disease Research Group. Dr. Darlington is a pediatric hematologist/oncologist whose primary research focuses on examining the health outcomes among patients with sickle cell disease. Clinically she is the director of the pediatric neuro-oncology program at the University of Chicago. She is also interested in caring for both benign and malignant childhood hematologic disorders. Dr. Darlington graduated from the University of Texas Medical Branch in Galveston with her MD in 2009. She completed her pediatrics residency at the University of Texas Southwestern Medical Center/Children’s Medical Center Dallas and her pediatric hematology/oncology fellowship at the University of Chicago.

Danielle Leach, MPA
Chief of Community & Government Relations
National Brain Tumor Society

Danielle Leach is the Chief of Community & Government Relations at the National Brain Tumor Society. She brings a track record of success in patient advocacy and education, fundraising, advancing legislative and regulatory policies, stakeholder engagement, and advocating for cancer research. She is a dedicated volunteer and grassroots advocate for cancer and children’s issues, driven by the death of her son Mason at age 5 from a pediatric brain tumor as well as being a sibling of a childhood cancer survivor. Danielle has led several public policy initiatives for childhood cancer research and funding. She serves as the Co-Chair of the National Cancer Institute’s Patient Advocate Steering Committee and as a member of the Pediatric and Adolescent Solid Tumor Steering Committee, and she previously served on NCI’s National Council of Research Advocates and on Vice President Biden’s Cancer Moonshot initiative, as a patient advocate on the Pediatric Cancer Working Group. Danielle was recognized in 2018 with the ASCO Partners in Progress award and the Rare Disease Legislative Advocates “RareVoice” Award.

Laura Rutledge
Executive Director
Rutledge Cancer Foundation
Laura Rutledge is Executive Director of Rutledge Cancer Foundation, a 501(c)3 created in 2011 to help advance less toxic, more curative therapies for sarcomas and other solid tumor cancers and to provide resources and care for adolescent and young adult patients.  Laura’s determination to find new, more effective cancer therapies for young adult cancer patients began when Laura’s 15-year-old daughter Carley was diagnosed with stage IV Ewing sarcoma. When their community learned that there had been little change in over 30 years in cure rates or treatment options for the over 89,000 young adult cancer patients diagnosed in the US each year, they were moved to action. Carley relapsed a year after frontline treatment and chose to participate as the first Ewing sarcoma patient in an early-stage autologous vaccine trial, called VIGIL. 8 years later, Carley was diagnosed again with Ewings and sadly died 14
months later, leaving a legacy of unrelenting positivity and the determination to find a cure for this devastating disease.

12:00noon-12:30pm

Poster Awards

12:30pm

Final words

Peter J. Houghton, PhD

SAPCS concluded