Program
Program

Program

12:00 Noon — REGISTRATION OPENS



2:00 - 2:10 p.m. — OPENING REMARKS

Jeffrey Trent, Ph.D., Translational Genomics Research Institute (TGen) (US) 
Javed Khan, M.D., National Cancer Institute, Center for Cancer Research (US)



2:10 - 5:50 p.m.
ACCESS TO THERAPEUTICS FOR PEDIATRIC CANCER PATIENTS, ALIGNING INCENTIVES: A WORKSHOP
Sponsored by the New England Journal of Medicine, this forum will explore access to investigational agents, US and international clinical study design, and first-in-human trials for pediatric cancer patients, with leaders in clinical and research oncology, regulatory affairs, and advocacy.

Workshop Co-Chairs

Elizabeth Phimister, Ph.D. – the New England Journal of Medicine (US)
Stefan Pfister, M.D. – Hopp Children’s Cancer Center, University of Heidelberg (Germany)

2:10 - 2:20 p.m.
WORKSHOP OPENING REMARKS

Elizabeth Phimister, Ph.D. – the New England Journal of Medicine (US)
Stefan Pfister, M.D. – Hopp Children’s Cancer Center, University of Heidelberg (Germany)

2:20 - 2:25 p.m.
Panel #1 Introduction
Lee Helman, M.D., Children’s Hospital Los Angeles (US)

2:25 - 3:10 p.m.
Panel #1 — Testing drugs designed for pediatric cancer

Donna Ludwinski, Patient Advocate (US)
Mary Smith, Ph.D., Springworks Therapeutics, LLC (US)
Terry Fry, M.D., University of Colorado School of Medicine and Children's Hospital Colorado (US)
Michael Dyer, Ph.D., St. Jude’s (US)



3:10 - 3:30 p.m. - Break



3:30 - 3:35 p.m.
Panel #2 Introduction
Peter Adamson, M.D. – Children’s Hospital of Philadelphia (US)

3:35 - 4:20 p.m.
Panel #2 – Adult drugs for testing in kids – what, when and who?

Lauren Bendesky, Patient Advocate (US)
Greg Reaman, M.D., US Food and Drug Administration (US)
Ralf Herold, M.D., Bayer (Germany)
Jeffrey Trent, Ph.D., Translational Genomics Research Institute (TGen) (US)
Josh Bilenker, M.D., LOXO Oncology (US)



4:20 - 4:40 p.m. - Break

4:40 - 4:55 p.m.
Panel #3 Introduction 

Nancy Goodman, Founder & Executive Director, Kids v. Cancer (US)

4:55 - 5:30 p.m.
Panel #3 – Implementing the RACE act and Focusing National Efforts to Support Early Therapeutics for Kids with Cancer

Kenny Thomas, Patient Advocate (US)
Kathleen Neville, M.D., M.S. Arkansas Children’s Hospital (US)
Richard Schilsky, M.D., American Society of Clinical Oncology (US)
Michael Caligiuri, M.D., City of Hope National Medical Center and American Association for Cancer Research (US)
Sandy Meech, M.D., Pfizer (US)

5:30 p.m. — Close
Bette Phimister, Ph.D., the New England Journal of Medicine (US)
Jeffrey Trent, Ph.D., Translational Genomics Research Institute (TGen) (US)



6:00 - 7:00 p.m. — NETWORK RECEPTION
7:00 a.m. — REGISTRATION OPENS



7:00 – 8:20 a.m. — POSTER SESSION I (Continental Breakfast provided)



8:20 – 8:30 a.m. — CONFERENCE OPENING REMARKS

Jeffrey Trent, Ph.D., Translational Genomics Research Institute (TGen) (US) 
Javed Khan, M.D., National Cancer Institute, Center for Cancer Research (US)



8:30 am- 9:00 a.m. — OPENING KEYNOTE SPEAKER

“Impressions of a New NCI Director”
Norman "Ned" Sharpless, M.D., National Cancer Institute Director (US)



9:00 a.m. - 6:00 p.m.
MOLECULAR PROFILING AND BIOMARKERS, AND SEQUENCING IN THE CLINIC AS A FOUNDATION FOR PRECISION TRIALS

9:00 - 10:35 a.m.
SESSION I: PRECISION MEDICINE CLINICAL TRIALS/MOLECULAR PROFILING AND BIOMARKERS

Session Chair:

Javed Khan, M.D., National Cancer Institute, Center for Cancer Research (US)

9:00 – 9:20 a.m.
Deep RNA-seq Identifies Clinically Relevant Cytotoxic Immune Cell Signatures and Clonal Expansion of T Cell Receptors in High-risk Neuroblastoma

Javed Khan, M.D., National Cancer Institute, Center for Cancer Research (US)

9:20 – 9:40 a.m.
Genomic and Transcriptomic Landscapes of Pediatric Cancers in the BCC Consortiums

Will Hendricks, Ph.D., Translational Genomics Research Institute (TGen) (US)

9:40 – 9:55 a.m.
Integrative analysis of whole-genome and RNA sequencing in high-risk pediatric malignancies
Avanthi Tayi Shah, M.D., University of California San Francisco (US)


9:55 - 10:15 a.m.

INFORM – Harnessing genome-wide profiling for stratification of pediatric patients at relapse
Stefan Pfister, M.D., Hopp Children’s Cancer Center, University of Heidelberg (Germany)

10:15 – 10:35 a.m.
European Precision Médicine Program:  the MAPPYACTS molecular profiling study

MAPYACTS; Gudrun Schleiermacher, M.D., Ph.D., Institut Curie (France)




10:35 – 10:50 a.m.  -  Break



10:50 a.m. - 12:20 p.m.
SESSION II: PRECISION MEDICINE CLINICAL TRIALS/MOLECULAR PROFILING AND BIOMARKERS

Session Chair:
Donald W. (Will) Parsons, M.D., Ph.D., Texas Children’s Hospital (US)

10:50 – 11:10 a.m.
NCI-COG Pediatric MATCH trial for treatment-refractory cancers
Donald W. Parsons, M.D., Ph.D., Texas Children’s Hospital (US)

11:10 – 11:25 a.m.
Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory or High-risk Leukemias: A Report from the LEAP Consortium
Yana Pikman, M.D., Dana Farber Cancer Institute (US) 

11:25 - 11:45 a.m.

AcSé-ESMART - a European therapeutic proof-of concept PCM trial
Birgit Geoerger, M.D., Ph.D., Institut Gustave Roussy, ITCC (France)

11:45 a.m. – 12:05 p.m.
INFORM2 biomarker driven combination trial series
Olaf Witt, M.D., Department of Pediatric Oncology, University of Heidelberg, ITCC (Germany)

12:05 – 12:20 p.m.
Applying tumor whole exome sequencing to the care of childhood solid tumor patients: Results from the BASIC3 clinical sequencing study
Frank Lin, M.D., Texas Children’s Hospital (US) 



12:20 – 1:30 p.m. — LUNCH



1:30 - 3:05 p.m.
SESSION III: PRECISION MEDICINE CLINICAL TRIALS/MOLECULAR PROFILING AND BIOMARKERS

Session Chair:

Yael Mosse,  M.D. - Children’s Hospital of Philadelphia (US)  

1:30 – 1:50 p.m.
Targeting evolving neuroblastoma therapy resistance

Yael Mosse, M.D., Children’s Hospital of Philadelphia (US)

1:50 – 2:10 p.m.
SCOUTing a Path to NAVIGATE Expedited Pediatric Development

Michael Cox, PharmD, LOXO Oncology (US)

2:10 – 2:25 p.m.
Searching for Translational Opportunities in the Complex Genomes of Osteosarcoma
Paul Meltzer, M.D., Ph.D., National Cancer Institute, Center for Cancer Research (US)

2:25 – 2:45 p.m.
Precision Medicine Approach for Children with Diffuse Intrinsic Pontine Glioma

Sabine Mueller, M.D, Ph.D., University of California, San Francisco  (US) 

2:45 – 3:05 p.m.
Clinical Outcomes Across Molecularly Guided Therapy Trials
Giselle Sholler, M.D., Helen DeVos Children's Hospital, Beat Childhood Cancer (US)



3:05 – 3:30 p.m. — BREAK



3:30 - 4:00 p.m. — KEYNOTE SPEAKER

Real World Evidence: Genomic Assessment Informs Novel Therapy (GAIN) Consortium and GENIE
Katherine A. Janeway, M.D., Harvard Medical School, Dana-Farber Cancer Institute (US) 



4:00 - 6:00 p.m.
SESSION IV: PRECISION MEDICINE CLINICAL TRIALS/MOLECULAR PROFILING AND BIOMARKERS

Session Chair:

Katherine A. Janeway, M.D., Harvard Medical School, Dana-Farber Cancer Institute (US) 

4:00 – 4:20 p.m.
The Targeted Agent and Profiling Utilization Registry (TAPUR) Study: A pragmatic, multi-basket, phase II study
Richard Schilsky, M.D., American Society of Clinical Oncology (US)

4:20 – 4:40 p.m.
Incorporating Patient Derived Models #1: Zero Childhood Cancer Program

Michelle Haber, Ph.D., Children’s Cancer Institute (Australia)

4:40 – 4:55 p.m.
The Long Tail of Diagnoses in a Cohort of Pediatric Patients with Extracranial Solid Tumors in a Clinical Sequencing Study
Suzanne Forrest, M.D., Dana-Farber Cancer Institute (US)

4:55 - 5:05 p.m.
Children, Adolescents, and Young Adult ALL: Translating genomic discoveries into therapeutic opportunities
 
Mignon Loh, M.D., University of California, San Francisco (US)

5:05 – 5:25 p.m.
Implementing Personalized Models And Combination Treatment In The Dutch Ither Precision Medicine Program

Jan Molenaar M.D., Ph.D., Prinses Maxima Center for Pediatric Oncology Dept. of Translational Research (Netherlands)

5:25 – 5:45 p.m.
Leveraging Comparative Oncology to Advance Translational Outcomes in Pediatric Patients

Cheryl London, D.V.M., Ph.D., Cummings School of Veterinary Medicine at Tufts University, The Ohio State University (US)

5:45 – 6:00 p.m.
Genome Informed Therapy for Osteosarcoma
Alejandro Sweet-Cordero, M.D., University of California San Francisco (US)




6:00 – 7:00 p.m. — BREAK



7:00 – 9:00 p.m. — GROUP DINNER AND NETWORKING


7:30 – 9:00 a.m. — POSTER SESSION II (Continental Breakfast Provided)



9:00 - 10:25 a.m.
SESSION V: GERMLINE


Session Co-Chairs:
Sharon Plon, M.D., Ph.D. - Baylor College of Medicine (US)
David Malkin, M.D., - The Hospital for Sick Children, University of Toronto (Canada)

9:00 – 9:30 a.m.
Utilizing Genomes and Epigenomes to Refine Tumor Surveillance in Li-Fraumeni Syndrome

David Malkin, M.D., - The Hospital for Sick Children, University of Toronto (Canada)

9:30 – 9:45 a.m.
Gabriella Miller Kids First Data Resource Center: Advancing Genetic Research in Childhood Cancer and Structural Birth Defects through Large Scale Integrated Data-Driven Discovery and Cloud-Based Platforms for Collaborative Analysis
Adam Resnick, Ph.D., Children’s Hospital of Philadelphia (US)
 

9:45 – 10:10 a.m.
The Value of Reporting Germline Cancer Susceptibility Findings in Precision Oncology Trials

Sharon Plon, M.D., Ph.D., Baylor College of Medicine (US)

10:10 – 10:25 a.m.
Somatic Hypermutation in Sporadic and Inherited Cancers
Adam Shlien, Ph.D., The Hospital for Sick Children, University of Toronto (Canada)



10:25 -10:45 a.m. —  BREAK



10:45 a.m. - 1:00 p.m.
SESSION VI: EMERGING TECHNOLOGIES FOR PRECISION THERAPIES

Session Co-Chairs:
Poul Sorensen, M.D., Ph.D. – BC Cancer Research Centre (Canada)
Markus Muschen, M.D., Ph.D. – City of Hope (US)

10:45 – 11:15 a.m.
Probing the mRNA translatome for novel targets in pediatric cancer

Poul Sorensen, Ph.D., University of British Columbia BC Cancer Research Center (Canada)

11:15 – 11:45 a.m.
Omics integration for the identification of cancer polygenes 
Mike Moran, Ph.D., The Hospital for Sick Children, University of Toronto (Canada)

11:45 a.m. – 12:00 Noon
PRKDC, the catalytic subunit of DNA-dependent protein kinase, regulates the stability of the EWS-FLI1 fusion oncoprotein
Natasha Caplen, Ph.D., National Cancer Institute (US)
 

12:00 – 12:30 p.m.
Autoimmunity checkpoints (AIC) as therapeutic targets in pediatric B-cell lineage ALL

Markus Müschen, M.D., Ph.D., City of Hope (US)

12:30 – 1:00 p.m.
Metabolic heterogeneity in human cancer 

Ralph Deberardinis, M.D., Ph.D., University of Texas Southwestern (US)



1:00 – 2:00 p.m. — LUNCH



2:00 – 2:30 p.m. — KEYNOTE SPEAKER

Clinical Genomics of Pediatric Brain Cancer
Elaine Mardis, Ph.D., Nationwide Children's Hospital, The Ohio State University College of Medicine (US)



2:30 - 5:20 p.m.

SESSION VII: IMMUNOTHERAPY

Session Co-Chairs:
Terry Fry, M.D., University of Colorado School of Medicine and Children's Hospital Colorado (US)
Michael Jensen, M.D., University of Washington School of Medicine, Seattle Children’s Research Institute (US)
Rimas Orentas, Ph.D., Seattle Children's Research Institute (US)

2:30 – 2:50 p.m.
Identifying and overcoming obstacles to curative CAR T cell therapy for acute lymphoblastic leukemia

Michael Jensen, M.D., University of Washington School of Medicine, Seattle Children’s Research Institute (US) 

2:50 – 3:10 p.m.
Beyond CD19: Challenges and Opportunities for CAR Therapy for Pediatric Hematologic

Terry Fry, M.D., University of Colorado School of Medicine and Children's Hospital Colorado (US)

3:10 – 3:30 p.m.
Adoptive T-cell therapy for epithelial cancers
 
Christian Hinrichs, M.D., National Cancer Institute, Center for Cancer Research (US)

3:30 – 3:45 p.m. 
Towards precision CAR T cell therapy for pediatric brain tumors
Leo D. Wang, MD, Ph.D., City of Hope (US)
 



3:45 - 4:05 p.m. — BREAK



4:05 – 4:25 p.m. 
Engineering T cells for Effective Immunotherapy of Solid Cancers
Nabil Ahmed, M.D., Baylor College of Medicine (US)

4:25 – 4:45 p.m.
Targeting the Surfaceome for Immunotherapy
 
Rimas Orentas, Ph.D., Seattle Children's Research Institute (US)

4:45 – 5:05 p.m.
Strategies to Enhance Immune Sensitivity of Metastatic Sarcoma
Alex Huang, M.D., Ph.D., Case Western Reserve University (US) 

5:05 – 5:20 p.m.
Identification of cell surface proteins as potential immunotherapy targets in pediatric ependymoma
Nipun Kakkar, Baylor College of Medicine (US)
 



5:20 p.m. — DINNER ON OWN

7:00 – 8:00 am — CONTINENTAL BREAKFAST



8:00 - 8:30 — SPECIAL LECTURE

SWI/SNF (BAF) Chromatin Remodeling Complexes are Frequently Mutated in Cancer: Mechanisms and Vulnerabilities
Charles Roberts, M.D., St. Jude’s Children’s Hospital (US)



8:30 a.m. - 10:10 a.m.
SESSION VIII: EPIGENETICS

Session Chair:
Carol Thiele, Ph.D, National Cancer Institute, Center for Cancer Research (US)

8:30 – 8:50 a.m.
Epigenetic Roadblocks to Reprogramming Neuroblastoma

Carol Thiele, Ph.D., National Cancer Institute, Center for Cancer Research (US)

8:50 – 9:10 a.m.
Mechanisms of chromatin regulation by EWS-FLI1

Miguel N. Rivera, M.D., Massachusetts General Hospital (US)

9:10 – 9:30 a.m.
Targeting Epigenetic Dysregulation in Pediatric Cancer

Kim Stegmaier, M.D., Dana-Farber Cancer Institute (US)

9:30 – 9:50 a.m.
Gynecologic cancers as models for oncogenesis through SWI/SNF disruption & miRNA processing defects
David Huntsman, M.D., FRCPC, FCCMG, University of British Columbia BC Cancer Research Center (Canada)

9:50 – 10:10 a.m.
Neuroblastoma: A Developmental Twist in Cancer

Rogier Versteeg, Amsterdam Medical Center (Amsterdam)



10:10 – 10:30 a.m. — BREAK



10:30 a.m. - 12:30 p.m.
SESSION IX: BIG DATA ANALYTICS 


Session Co-Chairs:
Jinghui Zhang, Ph.D., St. Jude’s (US)
Robert Grossman, PH.D., University of Chicago (US)



10:30 – 11:00 a.m. — KEYNOTE SPEAKER


How Data Commons Are Changing the Way That Large Biomedical Datasets are Analyzed
and Shared
Robert Grossman, Ph.D., University of Chicago (US)



11:00 – 11:20 a.m.
Integration of pan-cancer genome and transcriptome data for variant classification in pediatric cancer precision oncology  

Jinghui Zhang, Ph.D., St. Jude Children’s Research Hospital (US)

11:20 – 11:35 a.m. 
A systems biology driven drug repositioning strategy identifies digoxin as a potential treatment for Groups 3 and 4 medulloblastoma
Ching C. Lau, M.D., Ph.D., 
Connecticut Children's Medical Center (US) 

11:35 a.m. – 11:55 p.m.
Vetting Personalized, Big Data Approaches to Cancer Treatment
Nik Schork, Ph.D., Translational Genomics Research Institute (US) 

11:55 a.m. – 12:10 p.m.
The Data Refinery: A resource of harmonized public gene expression data sets
Jaclyn N. Taroni, Ph.D., 
University of Pennsylvania Perelman School of Medicine (US)

12:10 - 12:15 p.m.
Closing Comments - Javed Khan, M.D., National Cancer Institute, Center for Cancer Research (US)



12:15 pm — CONFERENCE ADJOURNS