Literature DB >> 27000960

Equal access to innovative therapies and precision cancer care.

Agnès Buzyn1, Jean-Yves Blay2,3, Natalie Hoog-Labouret1, Marta Jimenez4, Frédérique Nowak1, Marie-Cécile Le Deley5,6, David Pérol2, Christian Cailliot4, Jacques Raynaud7, Gilles Vassal5.   

Abstract

Patients with cancers of differing histologies that express certain biomarkers are likely to benefit from treatment with targeted therapies. However, targets can be present in malignancies other than those indicated by a drug's label, and as a result, affected patients will have no access to those potentially useful drugs. To tackle this issue, the French National Cancer Institute developed the AcSé Programme in 2013. This programme is designed to make treatment decisions or recommendations on the basis of the presence of relevant biomarkers for malignancies with no targeted therapies available and also aims to improve safety, and evaluate the efficacy of targeted drugs used outside of their approved indications. Patients across France have access to molecular testing in 28 molecular genetics centres and to targeted therapies within phase II trials provided no other trials exist in which they could reasonably be included. Trials include patients below the age of 18 if safe dosing data are available. As of January 2016, 183 French clinical sites and over 7,000 patients are participating in AcSé led trials. Proof of concept is being demonstrated through trials designed to investigate the effectiveness of crizotinib and vemurafenib in a wide variety of cancers.

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Year:  2016        PMID: 27000960     DOI: 10.1038/nrclinonc.2016.31

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


  45 in total

Review 1.  ROS1 as a 'druggable' receptor tyrosine kinase: lessons learned from inhibiting the ALK pathway.

Authors:  Sai-Hong Ignatius Ou; Jackie Tan; Yun Yen; Ross A Soo
Journal:  Expert Rev Anticancer Ther       Date:  2012-04       Impact factor: 4.512

2.  Prevalence of off-label use and spending in 2010 among patent-protected chemotherapies in a population-based cohort of medical oncologists.

Authors:  Rena M Conti; Arielle C Bernstein; Victoria M Villaflor; Richard L Schilsky; Meredith B Rosenthal; Peter B Bach
Journal:  J Clin Oncol       Date:  2013-02-19       Impact factor: 44.544

3.  Anaplastic lymphoma kinase aberrations in rhabdomyosarcoma: clinical and prognostic implications.

Authors:  J Carlijn van Gaal; Uta E Flucke; Melissa H S Roeffen; Eveline S J M de Bont; Stefan Sleijfer; Annelies M C Mavinkurve-Groothuis; Albert J H Suurmeijer; Winette T A van der Graaf; Yvonne M H Versleijen-Jonkers
Journal:  J Clin Oncol       Date:  2011-12-19       Impact factor: 44.544

4.  Germline and somatic mutations in the tyrosine kinase domain of the MET proto-oncogene in papillary renal carcinomas.

Authors:  L Schmidt; F M Duh; F Chen; T Kishida; G Glenn; P Choyke; S W Scherer; Z Zhuang; I Lubensky; M Dean; R Allikmets; A Chidambaram; U R Bergerheim; J T Feltis; C Casadevall; A Zamarron; M Bernues; S Richard; C J Lips; M M Walther; L C Tsui; L Geil; M L Orcutt; T Stackhouse; J Lipan; L Slife; H Brauch; J Decker; G Niehans; M D Hughson; H Moch; S Storkel; M I Lerman; W M Linehan; B Zbar
Journal:  Nat Genet       Date:  1997-05       Impact factor: 38.330

5.  An optimal three-stage design for phase II clinical trials.

Authors:  L G Ensign; E A Gehan; D S Kamen; P F Thall
Journal:  Stat Med       Date:  1994-09-15       Impact factor: 2.373

6.  Fusion of FIG to the receptor tyrosine kinase ROS in a glioblastoma with an interstitial del(6)(q21q21).

Authors:  Alain Charest; Keara Lane; Kevin McMahon; Julie Park; Elizabeth Preisinger; Helen Conroy; David Housman
Journal:  Genes Chromosomes Cancer       Date:  2003-05       Impact factor: 5.006

7.  MET gain in diffuse astrocytomas is associated with poorer outcome.

Authors:  Daniela Pierscianek; Young-Ho Kim; Kazuya Motomura; Michel Mittelbronn; Werner Paulus; Benjamin Brokinkel; Kathy Keyvani; Karsten Wrede; Yoichi Nakazato; Yuko Tanaka; Luigi Mariani; Anne Vital; Ulrich Sure; Hiroko Ohgaki
Journal:  Brain Pathol       Date:  2012-07-03       Impact factor: 6.508

Review 8.  The anaplastic lymphoma kinase in the pathogenesis of cancer.

Authors:  Roberto Chiarle; Claudia Voena; Chiara Ambrogio; Roberto Piva; Giorgio Inghirami
Journal:  Nat Rev Cancer       Date:  2008-01       Impact factor: 60.716

9.  Rare Cancers Europe (RCE) methodological recommendations for clinical studies in rare cancers: a European consensus position paper.

Authors:  P G Casali; P Bruzzi; J Bogaerts; J-Y Blay
Journal:  Ann Oncol       Date:  2014-10-01       Impact factor: 32.976

10.  RON is a heterodimeric tyrosine kinase receptor activated by the HGF homologue MSP.

Authors:  G Gaudino; A Follenzi; L Naldini; C Collesi; M Santoro; K A Gallo; P J Godowski; P M Comoglio
Journal:  EMBO J       Date:  1994-08-01       Impact factor: 11.598

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  7 in total

1.  Discussion of Trial Designs for Biomarker Identification and Validation Through the Use of Case Studies.

Authors:  Fang-Shu Ou; Ming-Wen An; Amy S Ruppert; Sumithra J Mandrekar
Journal:  JCO Precis Oncol       Date:  2019-10-24

2.  Efficacy of histology-agnostic and molecularly-driven HER2 inhibitors for refractory cancers.

Authors:  Luc Cabel; Alina Fuerea; Ludovic Lacroix; Capucine Baldini; Patricia Martin; Antoine Hollebecque; Sophie Postel-Vinay; Andrea Varga; Rastilav Balheda; Anas Gazzah; Jean-Marie Michot; Aurélien Marabelle; Etienne Rouleau; Eric Solary; Thierry De Baere; Eric Angevin; Jean-Pierre Armand; Stefan Michiels; Jean Yves Scoazec; Samy Ammari; Fabrice André; Jean-Charles Soria; Christophe Massard; Loic Verlingue
Journal:  Oncotarget       Date:  2018-01-12

Review 3.  Long-term efficacy of crizotinib in a metastatic papillary renal carcinoma with MET amplification: a case report and literature review.

Authors:  Philippe Rochigneux; Jeanne Thomassin-Piana; Sophy Laibe; Serge Brunelle; Naji Salem; Bernard Escudier; Gilles Vassal; Gwenaelle Gravis
Journal:  BMC Cancer       Date:  2018-11-22       Impact factor: 4.430

Review 4.  Expanded access to investigational drugs: balancing patient safety with potential therapeutic benefits.

Authors:  Elena Fountzilas; Rabih Said; Apostolia M Tsimberidou
Journal:  Expert Opin Investig Drugs       Date:  2018-01-22       Impact factor: 6.206

Review 5.  Importance of genetic screens in precision oncology.

Authors:  Antonio Mulero-Sánchez; Ziva Pogacar; Loredana Vecchione
Journal:  ESMO Open       Date:  2019-05-24

6.  Pharmacogenomics Implementation and Hurdles to Overcome; In the Context of a Developing Country.

Authors:  Nayyereh Ayati; Monireh Afzali; Mandana Hasanzad; Abbas Kebriaeezadeh; Ali Rajabzadeh; Shekoufeh Nikfar
Journal:  Iran J Pharm Res       Date:  2021       Impact factor: 1.696

Review 7.  Current and Future Molecular Testing in NSCLC, What Can We Expect from New Sequencing Technologies?

Authors:  Simon Garinet; Pierre Laurent-Puig; Hélène Blons; Jean-Baptiste Oudart
Journal:  J Clin Med       Date:  2018-06-09       Impact factor: 4.241

  7 in total

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