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Journal of Experimental & Clinical Cancer Research

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Open Access 01-12-2017 | Review

Liquid dynamic medicine and N-of-1 clinical trials: a change of perspective in oncology research

Authors: Nicola Silvestris, Gennaro Ciliberto, Paolo De Paoli, Giovanni Apolone, Maria Luisa Lavitrano, Marco A. Pierotti, Giorgio Stanta, On the behalf of the “dynamic medicine OECI group”

Published in: Journal of Experimental & Clinical Cancer Research | Issue 1/2017

Abstract

The increasing use of genomics to define the pattern of actionable mutations and to test and validate new therapies for individual cancer patients, and the growing application of liquid biopsy to dynamically track tumor evolution and to adapt molecularly targeted therapy according to the emergence of tumor clonal variants is shaping modern medical oncology., In order to better describe this new therapeutic paradigm we propose the term “Liquid dynamic medicine” in the place of “Personalized or Precision medicine”. Clinical validation of the “Liquid dynamic medicine” approach is best captured by N-of-1 trials where each patient acts as tester and control of truly personalized therapies.

Langreth R, Waldholz M. New era of personalized medicine targeting drugs for each unique genetic profile. Oncologist. 1999;4(5):426–7.PubMed

Personalized medicine, Wikipedia: https://en.wikipedia.org/wiki/Personalized_medicine.

Redekop WK, Mladsi D. The faces of personalized medicine: a framework for understanding its meaning and scope. Value Health. 2013;6:Suppl 4–9.CrossRef

Bria E, Di Maio M, Carlini P, Cuppone F, Giannarelli D, Cognetti F, Milella M. Targeting targeted agents: open issues for clinical trial design. J Exp Clin Cancer Res. 2009;28:66. https://doi.org/10.1186/1756-9966-28-66.CrossRefPubMedPubMedCentral

Garattini L, Curto A, Freemantle N. Personalized medicine and economic evaluation in oncology: all theory and no practice? Exp Rev Pharmacoeconom Res. 2015;15(5):733–8.

National Cancer Institute. Personalized medicine. In: NCI dictionary of cancer terns; 2012. Available from: http://www.cancer.gov/dictionary?cdrid=561717.

Druker BJ, Guilhot F, O'Brien SG, Gathmann I, Kantarjian H, Gattermann N, Deininger MW, Silver RT, Goldman JM, Stone RM, et al. Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med. 2006;355(23):2408–17.CrossRefPubMed

Martin V, Cappuzzo F, Mazzucchelli L, Frattini M. HER2 In solid tumors: more than 10 years under the microscope; where are we now? Future Oncol. 2014;10(8):1469–86.CrossRefPubMed

Vogel A, Hofheinz RD, Kubicka S, Arnold D. Treatment decisions in metastatic colorectal cancer - beyond first and second line combination therapies. Cancer Treat Rev. 2017;59:54–60.CrossRefPubMed

10.

Lee DH. Treatments for EGFR-mutant non-small cell lung cancer (NSCLC): the road to a success, paved with failures. Pharmacol Ther. 2017;174:1–21.CrossRefPubMed

11.

Sherwood J, Dearden S, Ratcliffe M, Walker J. Mutation status concordance between primary lesions and metastatic sites of advanced non-small-cell lung cancer and the impact of mutation testing methodologies: a literature review. J Exp Clin Cancer Res. 2015;34:92. https://doi.org/10.1186/s13046-015-0207-9. ReviewCrossRefPubMedPubMedCentral

12.

Simeone E, Grimaldi AM, Festino L, Vanella V, Palla M, Ascierto PA. Combination treatment of patients with BRAF-mutant melanoma: a new standard of care. BioDrugs. 2017;31(1):51–61.CrossRefPubMed

13.

Lin JJ, Riely GJ, Shaw AT. Targeting ALK: precision medicine takes on drug resistance. Cancer Discov. 2017;7(2):137–55.CrossRefPubMed

14.

Blackhall F, Cappuzzo F. Crizotinib: from discovery to accelerated development to front-line treatment. Ann Oncol. 2016;27 Suppl(3):35–41.CrossRef

15.

Schwaederle M, Zhao M, Lee JJ, Eggermont AM, Schilsky RL, Mendelsohn J, Lazar V, Kurzrock R. Impact of precision medicine in diverse cancers: a meta-analysis of phase II clinical trials. J Clin Oncol. 2015;33(32):3817–25.CrossRefPubMedPubMedCentral

16.

Schwaederle M, Zhao M, Lee JJ, Lazar V, Leyland-Jones B, Schilsky RL, Mendelsohn J, Kurzrock R. Association of Biomarker-Based Treatment Strategies with Response Rates and Progression-Free Survival in refractory malignant Neoplasms: a meta-analysis. JAMA Oncol. 2016;2(11):1452–9.CrossRefPubMed

17.

Falcone G, Felsani A, D'Agnano I. Signaling by exosomal microRNAs in cancer. J Exp Clin Cancer Res. 2015;34:32. https://doi.org/10.1186/s13046-015-0148-3.CrossRefPubMedPubMedCentral

18.

Koinis F, Voutsina A, Kalikaki A, Koutsopoulos A, Lagoudaki E, Tsakalaki E, Dermitzaki EK, Kontopodis E, Pallis AG, Georgoulias V, et al. Long-term clinical benefit from salvage EGFR tyrosine kinase inhibitors in advanced non-small-cell lung cancer patients with EGFR wild-type tumors. Clin Transl Oncol. 2017; https://doi.org/10.1007/s12094-017-1702-6.

19.

Welsh SJ, Rizos H, Scolyer RA, Long GV. Resistance to combination BRAF and MEK inhibition in metastatic melanoma: where to next? Eur J Cancer. 2016;62:76–85.CrossRefPubMed

20.

Schütte M, Ogilvie LA, Rieke DT, Lange BMH, Yaspo ML, Lehrach H. Cancer precision medicine: why more is more and DNA is not enough. Public Health Genomics. 2017; https://doi.org/10.1159/000477157.

21.

Hyman DM, Solit DB, Arcila ME, Cheng DT, Sabbatini P, Baselga J, Berger MF, Ladanyi M. Precision medicine at memorial Sloan Kettering cancer center: clinical next-generation sequencing enabling next-generation targeted therapy trials. Drug Discov Today. 2015;20(12):1422–8.CrossRefPubMedPubMedCentral

22.

Johnson A, Zeng J, Bailey AM, Holla V, Litzenburger B, Lara-Guerra H, Mills GB, Mendelsohn J, Shaw KR, Meric-Bernstam F. The right drugs at the right time for the right patient: the MD Anderson precision oncology decision support platform. Drug Discov Today. 2015;20(12):1433–8.CrossRefPubMed

23.

Hughes KS, Ambinder EP, Hess GP, Yu PP, Bernstam EV, Routbort MJ, Clemenceau JR, Hamm JT, Febbo PG, Domchek SM, et al. Identifying health information technology needs of oncologists to facilitate the adoption of genomic medicine: recommendations from the 2016 American Society of Clinical Oncology Omics and precision oncology workshop. J Clin Oncol 2017Jul 24:JCO2017741744. doi: https://doi.org/10.1200/JCO.2017.74.1744.

24.

Mina M, Raynaud F, Tavernari D, Battistello E, Sungalee S, Saghafinia S, Laessle T, Sanchez-Vega F, Schultz N, Oricchio E, Ciriello G. Conditional selection of genomic alterations dictates cancer evolution and Oncogenic dependencies. Cancer Cell. 2017 Aug 14;32(2):155–168.e6.CrossRefPubMed

25.

Abbosh C, Birkbak NJ, Wilson GA, Jamal-Hanjani M, Constantin T, Salari R, Le Quesne J, Moore DA, Veeriah S, Rosenthal R, et al. Phylogenetic ctDNA analysis depicts early-stage lung cancer evolution. Nature. 2017;545(7655):446–51.CrossRefPubMed

26.

Toledo RA, Cubillo A, Vega E, Garralda E, Alvarez R, de la Varga LU, Pascual JR, Sánchez G, Sarno F, Prieto SH, et al. Clinical validation of prospective liquid biopsy monitoring in patients with wild-type RAS metastatic colorectal cancer treated with FOLFIRI-cetuximab. Oncotarget. 2017;8(21):35289–300.PubMed

27.

Bardelli A. Medical research: personalized test tracks cancer relapse. Nature. 2017;545(7655):417–8.CrossRefPubMed

28.

Bauman Z. Globalization: the human consequences. New York: Columbia University Press. September, 1998.

29.

van Ommen GJ, Törnwall O, Bréchot C, Dagher G, Galli J, Hveem K, Landegren U, Luchinat C, Metspalu A, Nilsson C, et al. BBMRI-ERIC as a resource for pharmaceutical and life science industries: the development of biobank-based expert Centres. Eur J Hum Genet. 2015;23(7):893–900.CrossRefPubMed

30.

Melief CJM. Cancer: precision T-cell therapy targets tumours. Nature. 2017;547(7662):165–7.CrossRefPubMed

31.

Woodcock J, LaVange LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med. 2017;377(1):62–70.CrossRefPubMed

32.

Demeyin WA, Frost J, Ukoumunne OC, Briscoe S, Britten N. N of 1 trials and the optimal individualisation of drug treatments: a systematic review protocol. Syst Rev. 2017;6(1):90. https://doi.org/10.1186/s13643-017-0479-6.CrossRefPubMedPubMedCentral

33.

Markman M, Kramer K, Alvarez RH, Weiss GJ, Ahn E, Daneker GW. Evaluating the utility of a ‘N-of-1’ precision cancer medicine strategy: the case for ‘time-to-subsequent-disease progression’. Oncology. 2016;91(6):299–301.CrossRefPubMed

Title: Liquid dynamic medicine and N-of-1 clinical trials: a change of perspective in oncology research
Authors: Nicola Silvestris
Gennaro Ciliberto
Paolo De Paoli
Giovanni Apolone
Maria Luisa Lavitrano
Marco A. Pierotti
Giorgio Stanta
On the behalf of the “dynamic medicine OECI group”
Publication date: 01-12-2017
Publisher: BioMed Central
Published in: Journal of Experimental & Clinical Cancer Research / Issue 1/2017
Electronic ISSN: 1756-9966
DOI: https://doi.org/10.1186/s13046-017-0598-x

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