title: A Patient-Derived Xenograft Model of Parameningeal Embryonal Rhabdomyosarcoma for Preclinical Studies.
abstract: Embryonal rhabdomyosarcoma (eRMS) is one of the most common soft tissue sarcomas in children and adolescents. Parameningeal eRMS is a variant that is often more difficult to treat than eRMS occurring at other sites. A 14-year-old female with persistent headaches and rapid weight loss was diagnosed with parameningeal eRMS. She progressed and died despite chemotherapy with vincristine, actinomycin-D, and cyclophosphamide plus 50.4 Gy radiation therapy to the primary tumor site. Tumor specimens were acquired by rapid autopsy and tumor tissue was transplanted into immunodeficient mice to create a patient-derived xenograft (PDX) animal model. As autopsy specimens had an ALK R1181C mutation, PDX tumor bearing animals were treated with the pan-kinase inhibitor lestaurtinib but demonstrated no decrease in tumor growth, suggesting that single agent kinase inhibitor therapy may be insufficient in similar cases. This unique parameningeal eRMS PDX model is publicly available for preclinical study.
authors.affiliation: Department of Pathology, Johns Hopkins Medicine, 600 N. Wolfe Street, Pathology B-106, Baltimore, MD 21287, USA.
authors.forename: Jody E
authors.initials: JE
authors.lastname: Hooper
authors.affiliation: Pediatric Cancer Biology Program, Papé Family Pediatric Research Institute, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA ; Department of Pediatrics, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA.
authors.forename: Emma L
authors.initials: EL
authors.lastname: Cantor
authors.affiliation: Pediatric Cancer Biology Program, Papé Family Pediatric Research Institute, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA ; Department of Pediatrics, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA.
authors.forename: Macgregor S
authors.initials: MS
authors.lastname: Ehlen
authors.affiliation: Pediatric Cancer Biology Program, Papé Family Pediatric Research Institute, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA ; Department of Pediatrics, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA.
authors.forename: Avirup
authors.initials: A
authors.lastname: Banerjee
authors.affiliation: Department of Pediatrics, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA.
authors.forename: Suman
authors.initials: S
authors.lastname: Malempati
authors.affiliation: Department of Pathology, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA.
authors.forename: Peter
authors.initials: P
authors.lastname: Stenzel
authors.affiliation: Department of Pathology, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA.
authors.forename: Randy L
authors.initials: RL
authors.lastname: Woltjer
authors.affiliation: University of California Davis School of Medicine and Cancer Center, Sacramento, CA 95817, USA.
authors.forename: Regina
authors.initials: R
authors.lastname: Gandour-Edwards
authors.affiliation: The Jackson Laboratory, 1650 Santa Ana Avenue, Sacramento, CA 95838, USA ; Champions Oncology, Hackensack, NJ 07601, USA.
authors.forename: Neal C
authors.initials: NC
authors.lastname: Goodwin
authors.affiliation: The Jackson Laboratory, 1650 Santa Ana Avenue, Sacramento, CA 95838, USA ; Leo Universal, Inc., Torrance, CA 90505, USA.
authors.forename: Yan
authors.initials: Y
authors.lastname: Yang
authors.affiliation: The Jackson Laboratory, 1650 Santa Ana Avenue, Sacramento, CA 95838, USA.
authors.forename: Pali
authors.initials: P
authors.lastname: Kaur
authors.affiliation: The Jackson Laboratory, 1650 Santa Ana Avenue, Sacramento, CA 95838, USA.
authors.forename: Carol J
authors.initials: CJ
authors.lastname: Bult
authors.affiliation: The Jackson Laboratory, 1650 Santa Ana Avenue, Sacramento, CA 95838, USA.
authors.forename: Susan D
authors.initials: SD
authors.lastname: Airhart
authors.affiliation: Pediatric Cancer Biology Program, Papé Family Pediatric Research Institute, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA ; Department of Pediatrics, Oregon Health & Science University, 3181 S.W. Sam Jackson Park Road, Portland, OR 97239, USA ; Children's Cancer Therapy Development Institute, Beaverton, OR 97005, USA.
authors.forename: Charles
authors.initials: C
authors.lastname: Keller