Publications







Evaluation of Genetic Predisposition for MYCN-Amplified Neuroblastoma.
J Natl Cancer Inst 2017 Oct;109(10)
Affiliations of authors: Section of Hematology/Oncology, Department of Pediatrics (EAH, MAA, ADS, SLV, SLC), Section of Genetic Medicine, Department of Medicine (BES), and Institute for Genomics and Systems Biology, Center for Data Intensive Science (BES), University of Chicago, Chicago, IL; Division of Oncology and Center for Childhood Cancer Research and Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA (ZV, MD, LM, JMM, SJD); Department of Pediatrics and Abramson Family Cancer Research Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA (JMM, SJD); Department of Pediatrics, Hofstra-Northwell School of Medicine and The Feinstein Institute for Medical Research, Manhasset, NY (KO).



Identification of GPC2 as an Oncoprotein and Candidate Immunotherapeutic Target in High-Risk Neuroblastoma.
Cancer Cell 2017 Sep;32(3):295-309.e12
Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Colket Translational Research Building, 3501 Civic Center Boulevard, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address:


Revisions to the International Neuroblastoma Response Criteria: A Consensus Statement From the National Cancer Institute Clinical Trials Planning Meeting.
J Clin Oncol 2017 Aug 4;35(22):2580-2587. Epub 2017 May 4.
Julie R. Park, Seattle Children's Hospital and University of Washington School of Medicine, Seattle, WA; Rochelle Bagatell and John M. Maris, Children's Hospital of Philadelphia and University of Pennsylvania School of Medicine, Philadelphia, PA; Susan L. Cohn, University of Chicago, Chicago, IL; Andrew D. Pearson, Institute of Cancer Research and Royal Marsden National Health Service (NHS) Foundation Trust, Sutton, Surrey; Susan Burchill, Leeds Institute of Cancer and Pathology, St James University Hospital, Leeds; Kieran McHugh and Penelope Brock, Great Ormond Street Hospital for Children, NHS Trust, London, United Kingdom; Judith G. Villablanca, Children's Hospital Los Angeles and University of Southern California Keck School of Medicine, Los Angeles; Katherine K. Matthay, University of California San Francisco School of Medicine, San Francisco, CA; Frank Berthold, Children's Hospital and University of Cologne, Köln, Germany; Ariane Boubaker, Institute of Radiology, Clinique de La Source, Lausanne, Switzerland; Jed G. Nuchtern, Texas Children's Hospital and Baylor College of Medicine, Houston, TX; Wendy B. London, Dana-Farber/Boston Children's Cancer and Blood Disorder Center, Harvard Medical School, Boston, MA; Nita L. Seibel and O. Wolf Lindwasser, National Cancer Institute, Bethesda, MD; Gudrun Schleiermacher, Institut Curie, Paris; Dominique Valteau-Couanet, Gustave Roussy, Villejuif, France; and Ruth Ladenstein, Children's Cancer Research Institute, St Anna Children's Hospital, Vienna, Austria.


Association Between Telomere Length and Risk of Cancer and Non-Neoplastic Diseases: A Mendelian Randomization Study.
JAMA Oncol 2017 May;3(5):636-651
MRC Integrative Epidemiology Unit, University of Bristol, Bristol, England2School of Social and Community Medicine, University of Bristol, Bristol, England.

Preclinical Therapeutic Synergy of MEK1/2 and CDK4/6 Inhibition in Neuroblastoma.
Clin Cancer Res 2017 Apr 11;23(7):1785-1796. Epub 2016 Oct 11.
Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.



Phase I Study of the Aurora A Kinase Inhibitor Alisertib in Combination With Irinotecan and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma: A NANT (New Approaches to Neuroblastoma Therapy) Trial.
J Clin Oncol 2016 Apr 16;34(12):1368-75. Epub 2016 Feb 16.
Steven G. DuBois, Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston, MA; Araz Marachelian, Susan Groshen, Jemily Malvar, Hollie Lai, Fariba Goodarzian, Hiroyuki Shimada, Scarlett Czarnecki, and Denice Tsao-Wei, University of Southern California Keck School of Medicine and Children's Hospital Los Angeles, Los Angeles; Randall Hawkins, Jesse Courtier, and Katherine K. Matthay, University of California, San Francisco, School of Medicine and Benioff Children's Hospital, San Francisco, CA; Elizabeth Fox, Rochelle Bagatell, John M. Maris, and Yael P. Mosse, Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia, PA; Rachel A. Kudgus and Joel M. Reid, Mayo Clinic, Rochester, MN; and Lars Wagner, University of Kentucky College of Medicine, Lexington, KY.




MYC Disrupts the Circadian Clock and Metabolism in Cancer Cells.
Cell Metab 2015 Dec 17;22(6):1009-19. Epub 2015 Sep 17.
Abramson Family Cancer Research Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Hematology-Oncology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address:

Convergence of Acquired Mutations and Alternative Splicing of CD19 Enables Resistance to CART-19 Immunotherapy.
Cancer Discov 2015 Dec 29;5(12):1282-95. Epub 2015 Oct 29.
Division of Cancer Pathobiology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. Immunology Graduate Group, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania. Cell & Molecular Biology Graduate Group, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania. Department of Pathology & Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania.


Defining Why Cancer Develops in Children.
N Engl J Med 2015 Dec 18;373(24):2373-5. Epub 2015 Nov 18.
From the Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, and the Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania - both in Philadelphia.

Treatment of neuroblastoma in congenital central hypoventilation syndrome with a PHOX2B polyalanine repeat expansion mutation: New twist on a neurocristopathy syndrome.
Pediatr Blood Cancer 2015 Nov 22;62(11):2007-10. Epub 2015 May 22.
Division of Hematology, Oncology & Transplantation, Robert Lurie Comprehensive Cancer Center, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, Illinois.

A LIN28B-RAN-AURKA Signaling Network Promotes Neuroblastoma Tumorigenesis.
Cancer Cell 2015 Nov 17;28(5):599-609. Epub 2015 Oct 17.
Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Abramson Family Cancer Research Institute, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address:

Pilot study of intravenous melphalan combined with continuous infusion L-S,R-buthionine sulfoximine for children with recurrent neuroblastoma.
Pediatr Blood Cancer 2015 Oct 7;62(10):1739-46. Epub 2015 Jul 7.
Cancer Center and Departments of Cell Biology & Biochemistry, Pediatrics and Medicine, School of Medicine, Texas Tech University Health Sciences Center, Lubbock, Texas.

CASC15-S Is a Tumor Suppressor lncRNA at the 6p22 Neuroblastoma Susceptibility Locus.
Cancer Res 2015 Aug 22;75(15):3155-66. Epub 2015 Jun 22.
Division of Oncology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania. The Abramson Family Cancer Research Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania.







Common genetic variants in NEFL influence gene expression and neuroblastoma risk.
Cancer Res 2014 Dec 13;74(23):6913-24. Epub 2014 Oct 13.
Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania. Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania. Abramson Family Cancer Research Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania. Genomics and Computational Biology, Biomedical Graduate Studies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.

Age-dependent prognostic effect by Mitosis-Karyorrhexis Index in neuroblastoma: a report from the Children's Oncology Group.
Pediatr Dev Pathol 2014 Nov-Dec;17(6):441-9. Epub 2014 Sep 10.
1 Department of Pathology & Laboratory Medicine, Children's Hospital Los Angeles, and University of Southern California Keck School of Medicine, 4650 Sunset Boulevard, MS #43, Los Angeles, CA 90027, USA.

The promises and pitfalls of genetic epidemiologic approaches to pediatric cancers: lessons from MDM2.
Pediatr Blood Cancer 2014 Oct 17;61(10):1717-8. Epub 2014 Jun 17.
Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania; Abramson Family Cancer Research Institute, Perelman School of Medicine, The University of Pennsylvania, Philadelphia, Pennsylvania.



Rare variants in TP53 and susceptibility to neuroblastoma.
J Natl Cancer Inst 2014 Apr 14;106(4):dju047. Epub 2014 Mar 14.
Affiliations of authors: Division of Oncology (SJD, MDi, DAO, KC, KRB, MRR, JMM), Center for Childhood Cancer Research (SJD, MDi, DAO, KC, KRB, MRR, JMM), Center for Applied Genomics (HH), and Division of Genetics (HH, MDe) Children's Hospital of Philadelphia, Philadelphia, PA; Department of Pediatrics (SJD, DAO, KRB, HH, MDe, JMM), Abramson Cancer Center (SJD, JMM), Genomics and Computational Biology, Biomedical Graduate Studies (SJD, DAO, JMM), and Department of Biostatistics and Epidemiology (MDe), Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA (SJD, HH, MDe, JMM); Dipartimento di Medicina Molecolare e Biotecnologie Mediche, Università degli Studi di Napoli Federico II, Naples, Italy (MC, AI); Ceinge-Biotecnologie Avanzate, Naples, Italy (MC, AI); University of Rome "La Sapienza," Department of Molecular Medicine, Rome, Italy (MDe).


Initial testing (Stage 1) of the antibody-maytansinoid conjugate, IMGN901 (Lorvotuzumab mertansine), by the pediatric preclinical testing program.
Pediatr Blood Cancer 2013 Nov 24;60(11):1860-7. Epub 2013 Jun 24.
Hematology-Oncology Division of Oncology, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine and Abramson Family Cancer Research Institute, Philadelphia, Pennsylvania.

Phase I trial of fenretinide delivered orally in a novel organized lipid complex in patients with relapsed/refractory neuroblastoma: a report from the New Approaches to Neuroblastoma Therapy (NANT) consortium.
Pediatr Blood Cancer 2013 Nov 29;60(11):1801-8. Epub 2013 Jun 29.
Department of Cell Biology & Biochemistry, Texas Tech University Health Sciences Center, Lubbock, Texas; Department of Pediatrics, Texas Tech University Health Sciences Center, Lubbock, Texas; Department of Medicine, Texas Tech University Health Sciences Center, Lubbock, Texas.

Dual CDK4/CDK6 inhibition induces cell-cycle arrest and senescence in neuroblastoma.
Clin Cancer Res 2013 Nov 17;19(22):6173-82. Epub 2013 Sep 17.
Authors' Affiliations: Division of Oncology and Center for Childhood Cancer Research; Division of Pathology, Children's Hospital of Philadelphia; Department of Pediatrics; Abramson Family Cancer Research Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania; and Novartis Institutes for Biomedical Research, Cambridge, Massachusetts.

Neuroblastoma of undifferentiated subtype, prognostic significance of prominent nucleolar formation, and MYC/MYCN protein expression: a report from the Children's Oncology Group.
Cancer 2013 Oct 30;119(20):3718-26. Epub 2013 Jul 30.
Department of Pathology and Laboratory Medicine, Children's Hospital Los Angeles and University of Southern California Keck School of Medicine, Los Angeles, California.




Safety and activity of crizotinib for paediatric patients with refractory solid tumours or anaplastic large-cell lymphoma: a Children's Oncology Group phase 1 consortium study.
Lancet Oncol 2013 May 16;14(6):472-80. Epub 2013 Apr 16.
Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, and Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. Electronic address:




Pediatric phase I trial and pharmacokinetic study of MLN8237, an investigational oral selective small-molecule inhibitor of Aurora kinase A: a Children's Oncology Group Phase I Consortium study.
Clin Cancer Res 2012 Nov 17;18(21):6058-64. Epub 2012 Sep 17.
Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA.



Differential killing of CD56-expressing cells by drug-conjugated human antibodies targeting membrane-distal and membrane-proximal non-overlapping epitopes.
MAbs 2016 May-Jun;8(4):799-810. Epub 2016 Feb 24.
a Protein Interactions Section, Cancer and Inflammation Program, Center for Cancer Research, National Cancer Institute , Frederick , MD , USA.




A family-based study of gene variants and maternal folate and choline in neuroblastoma: a report from the Children's Oncology Group.
Cancer Causes Control 2016 10 19;27(10):1209-18. Epub 2016 Aug 19.
Department of Epidemiology, Gillings School of Global Public Health, University of North Carolina, Campus Box 7435, 2106 McGavran-Greenberg Hall, Chapel Hill, NC, 27599-7435, USA.



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