Literature DB >> 25223555

Drug discovery in paediatric oncology: roadblocks to progress.

Peter C Adamson1, Peter J Houghton2, Giorgio Perilongo3, Kathy Pritchard-Jones4.   

Abstract

Approval of new cancer drugs for paediatric patients generally occurs after their development and approval for treating adult cancers. As most drug development occurs in the industry setting, the relatively small market of paediatric oncology does not provide the financial incentives for companies to actively pursue paediatric oncology solutions. Indeed, between 1948 and January 2003 the FDA approved 120 new cancer drugs, of which only 30 have been used in children. This slow rate of development must be addressed in a meaningful way if we are to make progress in the most pressing settings in childhood cancer. In this Viewpoint article, the key opinion leaders in the field weigh in and offer practical advice on how to address this issue.

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Year:  2014        PMID: 25223555      PMCID: PMC4637819          DOI: 10.1038/nrclinonc.2014.149

Source DB:  PubMed          Journal:  Nat Rev Clin Oncol        ISSN: 1759-4774            Impact factor:   66.675


  37 in total

1.  The state of research into children with cancer across Europe: new policies for a new decade.

Authors:  K Pritchard-Jones; G Lewison; S Camporesi; G Vassal; R Ladenstein; Y Benoit; J S Predojevic; J Sterba; J Stary; T Eckschlager; H Schroeder; F Doz; U Creutzig; T Klingebiel; H V Kosmidis; M Garami; R Pieters; A O'Meara; G Dini; R Riccardi; J Rascon; L Rageliene; V Calvagna; P Czauderna; J R Kowalczyk; M J Gil-da-Costa; L Norton; F Pereira; D Janic; J Puskacova; J Jazbec; A Canete; L Hjorth; G Ljungman; T Kutluk; B Morland; M Stevens; D Walker; R Sullivan
Journal:  Ecancermedicalscience       Date:  2011-02-09

2.  New drugs for children and adolescents with cancer: the need for novel development pathways.

Authors:  Gilles Vassal; C Michel Zwaan; David Ashley; Marie Cecile Le Deley; Darren Hargrave; Patricia Blanc; Peter C Adamson
Journal:  Lancet Oncol       Date:  2013-02-20       Impact factor: 41.316

3.  Decoding the regulatory landscape of medulloblastoma using DNA methylation sequencing.

Authors:  Volker Hovestadt; David T W Jones; Simone Picelli; Wei Wang; Marcel Kool; Paul A Northcott; Marc Sultan; Katharina Stachurski; Marina Ryzhova; Hans-Jörg Warnatz; Meryem Ralser; Sonja Brun; Jens Bunt; Natalie Jäger; Kortine Kleinheinz; Serap Erkek; Ursula D Weber; Cynthia C Bartholomae; Christof von Kalle; Chris Lawerenz; Jürgen Eils; Jan Koster; Rogier Versteeg; Till Milde; Olaf Witt; Sabine Schmidt; Stephan Wolf; Torsten Pietsch; Stefan Rutkowski; Wolfram Scheurlen; Michael D Taylor; Benedikt Brors; Jörg Felsberg; Guido Reifenberger; Arndt Borkhardt; Hans Lehrach; Robert J Wechsler-Reya; Roland Eils; Marie-Laure Yaspo; Pablo Landgraf; Andrey Korshunov; Marc Zapatka; Bernhard Radlwimmer; Stefan M Pfister; Peter Lichter
Journal:  Nature       Date:  2014-05-18       Impact factor: 49.962

4.  Two consecutive phase II window trials of irinotecan alone or in combination with vincristine for the treatment of metastatic rhabdomyosarcoma: the Children's Oncology Group.

Authors:  Alberto S Pappo; Elizabeth Lyden; Phillip Breitfeld; Sarah S Donaldson; Eugene Wiener; David Parham; Kristine R Crews; Peter Houghton; William H Meyer
Journal:  J Clin Oncol       Date:  2007-02-01       Impact factor: 44.544

5.  R1507, a monoclonal antibody to the insulin-like growth factor 1 receptor, in patients with recurrent or refractory Ewing sarcoma family of tumors: results of a phase II Sarcoma Alliance for Research through Collaboration study.

Authors:  Alberto S Pappo; Shreyaskumar R Patel; John Crowley; Denise K Reinke; Klaus-Peter Kuenkele; Sant P Chawla; Guy C Toner; Robert G Maki; Paul A Meyers; Rashmi Chugh; Kristen N Ganjoo; Scott M Schuetze; Heribert Juergens; Michael G Leahy; Birgit Geoerger; Robert S Benjamin; Lee J Helman; Laurence H Baker
Journal:  J Clin Oncol       Date:  2011-10-24       Impact factor: 44.544

6.  The anti-CD19 antibody-drug conjugate SAR3419 prevents hematolymphoid relapse postinduction therapy in preclinical models of pediatric acute lymphoblastic leukemia.

Authors:  Hernan Carol; Barbara Szymanska; Kathryn Evans; Ingrid Boehm; Peter J Houghton; Malcolm A Smith; Richard B Lock
Journal:  Clin Cancer Res       Date:  2013-02-20       Impact factor: 12.531

7.  Phase II testing of melphalan in children with newly diagnosed rhabdomyosarcoma: a model for anticancer drug development.

Authors:  M E Horowitz; E Etcubanas; M L Christensen; J A Houghton; S L George; A A Green; P J Houghton
Journal:  J Clin Oncol       Date:  1988-02       Impact factor: 44.544

8.  Funding of US biomedical research, 2003-2008.

Authors:  E Ray Dorsey; Jason de Roulet; Joel P Thompson; Jason I Reminick; Ashley Thai; Zachary White-Stellato; Christopher A Beck; Benjamin P George; Hamilton Moses
Journal:  JAMA       Date:  2010-01-13       Impact factor: 56.272

9.  The role of SIOP as a platform for communication in the global response to childhood cancer.

Authors:  Gabriele Calaminus; Jillian R Birch; Rachel Hollis; Benson Pau; Mariana Kruger
Journal:  Pediatr Blood Cancer       Date:  2013-08-12       Impact factor: 3.167

10.  Challenges for children and adolescents with cancer in Europe: the SIOP-Europe agenda.

Authors:  Gilles Vassal; Edel Fitzgerald; Martin Schrappe; Frédéric Arnold; Jerzy Kowalczyk; David Walker; Lars Hjorth; Riccardo Riccardi; Anita Kienesberger; Kathy-Pritchard Jones; Maria Grazia Valsecchi; Dragana Janic; Henrik Hasle; Pamela Kearns; Giulia Petrarulo; Francesco Florindi; Samira Essiaf; Ruth Ladenstein
Journal:  Pediatr Blood Cancer       Date:  2014-04-07       Impact factor: 3.167

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  7 in total

1.  Predictors of Success of Phase II Pediatric Oncology Clinical Trials.

Authors:  Laura Franshaw; Maria Tsoli; Jennifer Byrne; Chelsea Mayoh; Siva Sivarajasingam; Murray Norris; Glenn M Marshall; David S Ziegler
Journal:  Oncologist       Date:  2019-02-26

Review 2.  Challenges and Opportunities for Childhood Cancer Drug Development.

Authors:  Peter J Houghton; Raushan T Kurmasheva
Journal:  Pharmacol Rev       Date:  2019-10       Impact factor: 25.468

Review 3.  Essential medicines for universal health coverage.

Authors:  Veronika J Wirtz; Hans V Hogerzeil; Andrew L Gray; Maryam Bigdeli; Cornelis P de Joncheere; Margaret A Ewen; Martha Gyansa-Lutterodt; Sun Jing; Vera L Luiza; Regina M Mbindyo; Helene Möller; Corrina Moucheraud; Bernard Pécoul; Lembit Rägo; Arash Rashidian; Dennis Ross-Degnan; Peter N Stephens; Yot Teerawattananon; Ellen F M 't Hoen; Anita K Wagner; Prashant Yadav; Michael R Reich
Journal:  Lancet       Date:  2016-11-08       Impact factor: 79.321

Review 4.  Neuroblastoma, a Paradigm for Big Data Science in Pediatric Oncology.

Authors:  Brittany M Salazar; Emily A Balczewski; Choong Yong Ung; Shizhen Zhu
Journal:  Int J Mol Sci       Date:  2016-12-27       Impact factor: 5.923

Review 5.  The contributions of the European Medicines Agency and its pediatric committee to the fight against childhood leukemia.

Authors:  Klaus Rose; Philip D Walson
Journal:  Risk Manag Healthc Policy       Date:  2015-11-05

Review 6.  MDM2/X inhibitors under clinical evaluation: perspectives for the management of hematological malignancies and pediatric cancer.

Authors:  Veronica Tisato; Rebecca Voltan; Arianna Gonelli; Paola Secchiero; Giorgio Zauli
Journal:  J Hematol Oncol       Date:  2017-07-03       Impact factor: 17.388

7.  Text-mining clinically relevant cancer biomarkers for curation into the CIViC database.

Authors:  Jake Lever; Martin R Jones; Arpad M Danos; Kilannin Krysiak; Melika Bonakdar; Jasleen K Grewal; Luka Culibrk; Obi L Griffith; Malachi Griffith; Steven J M Jones
Journal:  Genome Med       Date:  2019-12-03       Impact factor: 11.117

  7 in total

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