Literature DB >> 27748023

Precision medicine in pediatric oncology: Lessons learned and next steps.

Rajen J Mody1,2, John R Prensner3, Jessica Everett2,4, D Williams Parsons5,6, Arul M Chinnaiyan2,7,8.   

Abstract

The maturation of genomic technologies has enabled new discoveries in disease pathogenesis as well as new approaches to patient care. In pediatric oncology, patients may now receive individualized genomic analysis to identify molecular aberrations of relevance for diagnosis and/or treatment. In this context, several recent clinical studies have begun to explore the feasibility and utility of genomics-driven precision medicine. Here, we review the major developments in this field, discuss current limitations, and explore aspects of the clinical implementation of precision medicine, which lack consensus. Lastly, we discuss ongoing scientific efforts in this arena, which may yield future clinical applications.
© 2016 Wiley Periodicals, Inc.

Entities:  

Keywords:  next-generation sequencing; oncology; precision medicine; targeted therapy

Mesh:

Year:  2016        PMID: 27748023      PMCID: PMC5683396          DOI: 10.1002/pbc.26288

Source DB:  PubMed          Journal:  Pediatr Blood Cancer        ISSN: 1545-5009            Impact factor:   3.167


  107 in total

1.  NCI-MATCH launch highlights new trial design in precision-medicine era.

Authors:  Caroline McNeil
Journal:  J Natl Cancer Inst       Date:  2015-07-03       Impact factor: 13.506

2.  Rapid Capture Next-Generation Sequencing in Clinical Diagnostics of Kinase Pathway Aberrations in B-Cell Precursor ALL.

Authors:  Udo Zur Stadt; Gabriele Escherich; Daniela Indenbirken; Malik Alawi; Manuela Adao; Martin A Horstmann
Journal:  Pediatr Blood Cancer       Date:  2016-03-23       Impact factor: 3.167

3.  Complementary genomic approaches highlight the PI3K/mTOR pathway as a common vulnerability in osteosarcoma.

Authors:  Jennifer A Perry; Adam Kiezun; Peter Tonzi; Eliezer M Van Allen; Scott L Carter; Sylvan C Baca; Glenn S Cowley; Ami S Bhatt; Esther Rheinbay; Chandra Sekhar Pedamallu; Elena Helman; Amaro Taylor-Weiner; Aaron McKenna; David S DeLuca; Michael S Lawrence; Lauren Ambrogio; Carrie Sougnez; Andrey Sivachenko; Loren D Walensky; Nikhil Wagle; Jaume Mora; Carmen de Torres; Cinzia Lavarino; Simone Dos Santos Aguiar; Jose Andres Yunes; Silvia Regina Brandalise; Gabriela Elisa Mercado-Celis; Jorge Melendez-Zajgla; Rocío Cárdenas-Cardós; Liliana Velasco-Hidalgo; Charles W M Roberts; Levi A Garraway; Carlos Rodriguez-Galindo; Stacey B Gabriel; Eric S Lander; Todd R Golub; Stuart H Orkin; Gad Getz; Katherine A Janeway
Journal:  Proc Natl Acad Sci U S A       Date:  2014-12-15       Impact factor: 11.205

4.  Detection of circulating tumor DNA in early- and late-stage human malignancies.

Authors:  Chetan Bettegowda; Mark Sausen; Rebecca J Leary; Isaac Kinde; Yuxuan Wang; Nishant Agrawal; Bjarne R Bartlett; Hao Wang; Brandon Luber; Rhoda M Alani; Emmanuel S Antonarakis; Nilofer S Azad; Alberto Bardelli; Henry Brem; John L Cameron; Clarence C Lee; Leslie A Fecher; Gary L Gallia; Peter Gibbs; Dung Le; Robert L Giuntoli; Michael Goggins; Michael D Hogarty; Matthias Holdhoff; Seung-Mo Hong; Yuchen Jiao; Hartmut H Juhl; Jenny J Kim; Giulia Siravegna; Daniel A Laheru; Calogero Lauricella; Michael Lim; Evan J Lipson; Suely Kazue Nagahashi Marie; George J Netto; Kelly S Oliner; Alessandro Olivi; Louise Olsson; Gregory J Riggins; Andrea Sartore-Bianchi; Kerstin Schmidt; le-Ming Shih; Sueli Mieko Oba-Shinjo; Salvatore Siena; Dan Theodorescu; Jeanne Tie; Timothy T Harkins; Silvio Veronese; Tian-Li Wang; Jon D Weingart; Christopher L Wolfgang; Laura D Wood; Dongmei Xing; Ralph H Hruban; Jian Wu; Peter J Allen; C Max Schmidt; Michael A Choti; Victor E Velculescu; Kenneth W Kinzler; Bert Vogelstein; Nickolas Papadopoulos; Luis A Diaz
Journal:  Sci Transl Med       Date:  2014-02-19       Impact factor: 17.956

5.  Paradoxical activation and RAF inhibitor resistance of BRAF protein kinase fusions characterizing pediatric astrocytomas.

Authors:  Angela J Sievert; Shih-Shan Lang; Katie L Boucher; Peter J Madsen; Erin Slaunwhite; Namrata Choudhari; Meghan Kellet; Phillip B Storm; Adam C Resnick
Journal:  Proc Natl Acad Sci U S A       Date:  2013-03-26       Impact factor: 11.205

6.  PARP-1 inhibition as a targeted strategy to treat Ewing's sarcoma.

Authors:  J Chad Brenner; Felix Y Feng; Sumin Han; Sonam Patel; Siddharth V Goyal; Laura M Bou-Maroun; Meilan Liu; Robert Lonigro; John R Prensner; Scott A Tomlins; Arul M Chinnaiyan
Journal:  Cancer Res       Date:  2012-01-27       Impact factor: 12.701

7.  Targeting oxidative stress in embryonal rhabdomyosarcoma.

Authors:  Xiang Chen; Elizabeth Stewart; Anang A Shelat; Chunxu Qu; Armita Bahrami; Mark Hatley; Gang Wu; Cori Bradley; Justina McEvoy; Alberto Pappo; Sheri Spunt; Marcus B Valentine; Virginia Valentine; Fred Krafcik; Walter H Lang; Monika Wierdl; Lyudmila Tsurkan; Viktor Tolleman; Sara M Federico; Chris Morton; Charles Lu; Li Ding; John Easton; Michael Rusch; Panduka Nagahawatte; Jianmin Wang; Matthew Parker; Lei Wei; Erin Hedlund; David Finkelstein; Michael Edmonson; Sheila Shurtleff; Kristy Boggs; Heather Mulder; Donald Yergeau; Steve Skapek; Douglas S Hawkins; Nilsa Ramirez; Philip M Potter; John A Sandoval; Andrew M Davidoff; Elaine R Mardis; Richard K Wilson; Jinghui Zhang; James R Downing; Michael A Dyer
Journal:  Cancer Cell       Date:  2013-12-09       Impact factor: 31.743

8.  Targeting wild-type and mutationally activated FGFR4 in rhabdomyosarcoma with the inhibitor ponatinib (AP24534).

Authors:  Samuel Q Li; Adam T Cheuk; Jack F Shern; Young K Song; Laura Hurd; Hongling Liao; Jun S Wei; Javed Khan
Journal:  PLoS One       Date:  2013-10-04       Impact factor: 3.240

9.  Phase II study of olaparib in patients with refractory Ewing sarcoma following failure of standard chemotherapy.

Authors:  Edwin Choy; James E Butrynski; David C Harmon; Jeffrey A Morgan; Suzanne George; Andrew J Wagner; David D'Adamo; Gregory M Cote; Yael Flamand; Cyril H Benes; Daniel A Haber; Jose M Baselga; George D Demetri
Journal:  BMC Cancer       Date:  2014-11-05       Impact factor: 4.430

10.  Obtaining informed consent for clinical tumor and germline exome sequencing of newly diagnosed childhood cancer patients.

Authors:  Sarah Scollon; Katie Bergstrom; Robin A Kerstein; Tao Wang; Susan G Hilsenbeck; Uma Ramamurthy; Richard A Gibbs; Christine M Eng; Murali M Chintagumpala; Stacey L Berg; Laurence B McCullough; Amy L McGuire; Sharon E Plon; D Williams Parsons
Journal:  Genome Med       Date:  2014-09-17       Impact factor: 11.117

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  18 in total

1.  Cryptic ETV6-ABL1 Fusion and MLL2 Truncation Revealed by Integrative Clinical Sequencing in Multiply Relapsed Acute Lymphoblastic Leukemia.

Authors:  Nikhil P Mankuzhy; Emily Walling; Bailey Anderson; Rajen Mody
Journal:  J Pediatr Hematol Oncol       Date:  2019-11       Impact factor: 1.289

Review 2.  The genomic landscape of pediatric cancers: Implications for diagnosis and treatment.

Authors:  E Alejandro Sweet-Cordero; Jaclyn A Biegel
Journal:  Science       Date:  2019-03-15       Impact factor: 47.728

3.  Approaching the third decade of paediatric palliative oncology investigation: historical progress and future directions.

Authors:  Abby R Rosenberg; Joanne Wolfe
Journal:  Lancet Child Adolesc Health       Date:  2017-07-24

Review 4.  Precision medicine in pediatric oncology.

Authors:  Suzanne J Forrest; Birgit Geoerger; Katherine A Janeway
Journal:  Curr Opin Pediatr       Date:  2018-02       Impact factor: 2.856

5.  Clinical Impact of Genomic Information in Pediatric Leukemia.

Authors:  Emilie Lalonde; Gerald Wertheim; Marilyn M Li
Journal:  Front Pediatr       Date:  2017-12-14       Impact factor: 3.418

6.  Deep Functional and Molecular Characterization of a High-Risk Undifferentiated Pleomorphic Sarcoma.

Authors:  Noah E Berlow; Catherine S Grasso; Michael J Quist; Mingshan Cheng; Regina Gandour-Edwards; Brian S Hernandez; Joel E Michalek; Christopher Ryan; Paul Spellman; Ranadip Pal; Lynn S Million; Mark Renneker; Charles Keller
Journal:  Sarcoma       Date:  2020-05-04

7.  Clinical cancer genomic profiling by three-platform sequencing of whole genome, whole exome and transcriptome.

Authors:  Michael Rusch; Joy Nakitandwe; Sheila Shurtleff; Scott Newman; Zhaojie Zhang; Michael N Edmonson; Matthew Parker; Yuannian Jiao; Xiaotu Ma; Yanling Liu; Jiali Gu; Michael F Walsh; Jared Becksfort; Andrew Thrasher; Yongjin Li; James McMurry; Erin Hedlund; Aman Patel; John Easton; Donald Yergeau; Bhavin Vadodaria; Ruth G Tatevossian; Susana Raimondi; Dale Hedges; Xiang Chen; Kohei Hagiwara; Rose McGee; Giles W Robinson; Jeffery M Klco; Tanja A Gruber; David W Ellison; James R Downing; Jinghui Zhang
Journal:  Nat Commun       Date:  2018-09-27       Impact factor: 14.919

8.  Targetable BRAF and RAF1 Alterations in Advanced Pediatric Cancers.

Authors:  Andrew Rankin; Adrienne Johnson; Alison Roos; Geoffrey Kannan; Jeffrey Knipstein; Nicholas Britt; Mark Rosenzweig; James Haberberger; Dean Pavlick; Eric Severson; Jo-Anne Vergilio; Rachel Squillace; Rachel Erlich; Pratheesh Sathyan; Stuart Cramer; David Kram; Jeffrey Ross; Vince Miller; Prasanth Reddy; Brian Alexander; Siraj M Ali; Shakti Ramkissoon
Journal:  Oncologist       Date:  2020-09-25

9.  The impact of hereditary cancer gene panels on clinical care and lessons learned.

Authors:  Volkan Okur; Wendy K Chung
Journal:  Cold Spring Harb Mol Case Stud       Date:  2017-11-21

Review 10.  Cell death-based treatment of childhood cancer.

Authors:  Mike-Andrew Westhoff; Nicolas Marschall; Michael Grunert; Georg Karpel-Massler; Stefan Burdach; Klaus-Michael Debatin
Journal:  Cell Death Dis       Date:  2018-01-25       Impact factor: 8.469

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