title: Current state of pediatric sarcoma biology and opportunities for future discovery: A report from the sarcoma translational research workshop.
abstract: Sarcomas are a rare subgroup of pediatric cancers comprised of a variety of bone and soft-tissue tumors. While significant advances have been made in improving outcomes of patients with localized pediatric sarcomas since the addition of systemic chemotherapy to local control many decades ago, outcomes for patients with metastatic and relapsed sarcoma remain poor with few novel therapeutics identified to date. With the advent of new technologies to study cancer genomes, transcriptomes and epigenomes, our understanding of sarcoma biology has improved tremendously in a relatively short period of time. However, much remains to be accomplished in this arena especially with regard to translating all of this new knowledge to the bedside. To this end, a meeting was convened in Philadelphia, PA, on April 18, 2015 sponsored by the QuadW foundation, Children's Oncology Group and CureSearch for Children's Cancer that brought together sarcoma clinicians and scientists from North America to review the current state of pediatric sarcoma biology and ongoing/planned genomics based clinical trials in an effort to identify and bridge knowledge gaps that continue to exist at present. At the conclusion of the workshop, three key objectives that would significantly further our understanding of sarcoma were identified and a proposal was put forward to develop an all-encompassing pediatric sarcoma biology protocol that would address these specific needs. This review summarizes the proceedings of the workshop.
authors.affiliation: Center for Cancer and Blood Disorders, Phoenix Children's Hospital, Phoenix, AZ, USA. Electronic address: phingorani@phoenixchildrens.com.
authors.forename: Pooja
authors.initials: P
authors.lastname: Hingorani
authors.affiliation: Department of Pediatric Hematology-Oncology, Dana-Farber Cancer Institute and Boston Children's Hospital, Boston, MA, USA.
authors.forename: Katherine
authors.initials: K
authors.lastname: Janeway
authors.affiliation: Department of Pediatric Hematology-Oncology, Dana-Farber Cancer Institute and Boston Children's Hospital, Boston, MA, USA.
authors.forename: Brian D
authors.initials: BD
authors.lastname: Crompton
authors.affiliation: Department of Pediatric Hematology-Oncology, Dana-Farber Cancer Institute and Boston Children's Hospital, Boston, MA, USA.
authors.forename: Cigall
authors.initials: C
authors.lastname: Kadoch
authors.affiliation: Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Crystal L
authors.initials: CL
authors.lastname: Mackall
authors.affiliation: Genetics Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Javed
authors.initials: J
authors.lastname: Khan
authors.affiliation: Genetics Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Jack F
authors.initials: JF
authors.lastname: Shern
authors.affiliation: Huntsman Cancer Institute & Primary Children's Medical Center, University of Utah, Salt Lake City, UT, USA.
authors.forename: Joshua
authors.initials: J
authors.lastname: Schiffman
authors.affiliation: Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Lisa
authors.initials: L
authors.lastname: Mirabello
authors.affiliation: Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Sharon A
authors.initials: SA
authors.lastname: Savage
authors.affiliation: Human Oncology and Pathogenesis Program, Department of Pathology, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Marc
authors.initials: M
authors.lastname: Ladanyi
authors.affiliation: Genetics Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Paul
authors.initials: P
authors.lastname: Meltzer
authors.affiliation: The Jackson Laboratory, Bar Harbor, ME, USA.
authors.forename: Carol J
authors.initials: CJ
authors.lastname: Bult
authors.affiliation: Division of Clinical Pharmacology & Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
authors.forename: Peter C
authors.initials: PC
authors.lastname: Adamson
authors.affiliation: Department of Pediatrics, Section of Hematology-Oncology, Baylor College of Medicine, Houston, TX, USA.
authors.forename: Philip J
authors.initials: PJ
authors.lastname: Lupo
authors.affiliation: Department of Pediatrics, University Of Michigan, Ann Arbor, MI, USA.
authors.forename: Rajen
authors.initials: R
authors.lastname: Mody
authors.affiliation: Department of Pediatric Hematology-Oncology, Dana-Farber Cancer Institute and Boston Children's Hospital, Boston, MA, USA.
authors.forename: Steven G
authors.initials: SG
authors.lastname: DuBois
authors.affiliation: Texas Children's Cancer Center, Baylor College of Medicine, Houston, TX, USA.
authors.forename: D Williams
authors.initials: DW
authors.lastname: Parsons
authors.affiliation: Molecular Oncology Section, Center for Cancer Research, National Cancer Institute, NIH, Bethesda, MD, USA.
authors.forename: Chand
authors.initials: C
authors.lastname: Khanna
authors.affiliation: Department of Pediatrics, Section of Hematology-Oncology, Baylor College of Medicine, Houston, TX, USA.
authors.forename: Ching
authors.initials: C
authors.lastname: Lau
authors.affiliation: Seattle Children's Hospital, University of Washington, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
authors.forename: Douglas S
authors.initials: DS
authors.lastname: Hawkins
authors.affiliation: Huntsman Cancer Institute & Primary Children's Medical Center, University of Utah, Salt Lake City, UT, USA.
authors.forename: R Lor
authors.initials: RL
authors.lastname: Randall
authors.affiliation: National Cancer Institute, NIH, Bethesda, MD, USA.
authors.forename: Malcolm
authors.initials: M
authors.lastname: Smith
authors.affiliation: Department of Pathology, University of British Columbia, Vancouver, BC, Canada; Department of Molecular Oncology, BC Cancer Research Centre, Vancouver, BC, Canada.
authors.forename: Poul H
authors.initials: PH
authors.lastname: Sorensen
authors.affiliation: Texas Children's Cancer Center, Baylor College of Medicine, Houston, TX, USA.
authors.forename: Sharon E
authors.initials: SE
authors.lastname: Plon
authors.affiliation: Division of Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, TX, USA.
authors.forename: Stephen X
authors.initials: SX
authors.lastname: Skapek
authors.affiliation: Division of Hematology/ Oncology, Nationwide Children's Hospital, Columbus, OH, USA.
authors.forename: Stephen
authors.initials: S
authors.lastname: Lessnick
authors.affiliation: Division of Pediatric Hematology/Oncology, Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY, USA.
authors.forename: Richard
authors.initials: R
authors.lastname: Gorlick
authors.affiliation: Moffitt Cancer Center, Sarcoma Department, Adolescent and Young Adult Program, Tampa, FL, USA.
authors.forename: Damon R
authors.initials: DR
authors.lastname: Reed
mesh_terms: D000818:Animals; D002985:Clinical Protocols; D004353:Drug Evaluation, Preclinical; D057890:Epigenomics; D023281:Genomics; D018095:Germ-Line Mutation; D006801:Humans; D057285:Precision Medicine / Q000639:trends; D012008:Recurrence; D012509:Sarcoma / Q000235:genetics* / Q000473:pathology / Q000628:therapy; D057170:Translational Research, Biomedical; D023041:Xenograft Model Antitumor Assays
keywords: Pediatric sarcoma; genomics; molecular profiling; patient derived xenografts; precision medicine; sarcoma biology