Patient-targeted molecular therapies

Section edited by Francesco Chiappelli and Francesco Piva

The mission of this section is to enrich the overall mandate of the journal through effectively capturing the growing sector of molecular targeted therapies with broad therapeutic applicability, benefiting from a patient-centered outcomes research focus and translational effectiveness approach, to generate adequate information during research and early development stages, as well as consensus on the best available evidence.

This section has a unique and defining objective and rationale relative to the other sections that focus primarily on translational immunology and oncology. In contrast, this section will add the complementary discourse between translational research and translational effectiveness. In doing so, it will expand the overall vision of the journal, and address current, timely and cutting edge research and reviews about patient-centered targeted therapies, in context of translating cost and benefit effectiveness to specific clinical settings.

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  1. Research

    FGFR1 and NTRK3 actionable alterations in “Wild-Type” gastrointestinal stromal tumors

    About 10–15% of adult, and most pediatric, gastrointestinal stromal tumors (GIST) lack mutations in KIT, PDGFRA, SDHx, or RAS pathway components (KRAS, BRAF, NF1). The identification of additional mutated gene...

    Eileen Shi, Juliann Chmielecki, Chih-Min Tang, Kai Wang, Michael C. Heinrich, Guhyun Kang, Christopher L. Corless, David Hong, Katherine E. Fero, James D. Murphy, Paul T. Fanta, Siraj M. Ali, Martina De Siena, Adam M. Burgoyne, Sujana Movva, Lisa Madlensky…

    Journal of Translational Medicine 2016 14:339

    Published on: 14 December 2016

  2. Research

    Novel patient-derived xenograft mouse model for pancreatic acinar cell carcinoma demonstrates single agent activity of oxaliplatin

    Pancreatic acinar cell carcinoma (PACC) is a rare malignancy, accounting for <1 % of all pancreatic neoplasms. Very few retrospective studies are available to help guide management. We previously reported the ...

    Jason C. Hall, Laura A. Marlow, Adam C. Mathias, Louis K. Dawson, William F. Durham, Kenneth A. Meshaw, Robert J. Mullin, Aidan J. Synnott, Daniel L. Small, Murli Krishna, Daniel von Hoff, Julia Schüler, Steven N. Hart, Fergus J. Couch, Gerardo Colon-Otero and John A. Copland

    Journal of Translational Medicine 2016 14:129

    Published on: 10 May 2016

  3. Research

    PI3K/AKT/mTOR inhibition in combination with doxorubicin is an effective therapy for leiomyosarcoma

    Leiomyosarcoma (LMS) is a common type of soft tissue sarcoma that responds poorly to standard chemotherapy. Thus the goal of this study was to identify novel selective therapies that may be effective in leiomy...

    Yael Babichev, Leah Kabaroff, Alessandro Datti, David Uehling, Methvin Isaac, Rima Al-awar, Michael Prakesch, Ren X. Sun, Paul C. Boutros, Rosemarie Venier, Brendan C. Dickson and Rebecca A. Gladdy

    Journal of Translational Medicine 2016 14:67

    Published on: 8 March 2016

  4. Research

    Effect of the JAK2/STAT3 inhibitor SAR317461 on human glioblastoma tumorspheres

    The STAT3 transcription factor is a major intracellular signaling protein and is frequently dysregulated in the most common and lethal brain malignancy in adults, glioblastoma multiforme (GBM). Activation of ...

    Rajesh Mukthavaram, Xiao Ouyang, Rohit Saklecha, Pengfei Jiang, Natsuko Nomura, Sandeep C Pingle, Fang Guo, Milan Makale and Santosh Kesari

    Journal of Translational Medicine 2015 13:269

    Published on: 18 August 2015

  5. Research

    Early decline in serum phospho-CSE1L levels in vemurafenib/sunitinib-treated melanoma and sorafenib/lapatinib-treated colorectal tumor xenografts

    Although targeted therapies have improved the clinical outcomes of cancer treatment, tumors resistance to targeted drug are often detected too late and cause mortality. CSE1L is secreted from tumor and its pho...

    Woan-Ruoh Lee, Shing-Chuan Shen, Yi-Hsien Shih, Chia-Lun Chou, Jonathan Te-Peng Tseng, Szu-Ying Chin, Kao-Hui Liu, Yen-Chou Chen and Ming-Chung Jiang

    Journal of Translational Medicine 2015 13:191

    Published on: 13 June 2015

  6. Research

    Response of recurrent BRAFV600E mutated ganglioglioma to Vemurafenib as single agent

    Ganglioglioma (GG) and pilocytic astrocytoma (PA) represent the most frequent low-grade gliomas (LGG) occurring in paediatric age. LGGs not amenable of complete resection (CR) represent a challenging subgroup ...

    Francesca del Bufalo, Andrea Carai, Lorenzo Figà-Talamanca, Benedetta Pettorini, Conor Mallucci, Felice Giangaspero, Manila Antonelli, Manuela Badiali, Loredana Moi, Giuseppe Bianco, Antonella Cacchione, Franco Locatelli, Elisabetta Ferretti and Angela Mastronuzzi

    Journal of Translational Medicine 2014 12:356

    Published on: 19 December 2014

  7. Methodology

    Saliva as a sampling source for the detection of leukemic fusion transcripts

    Saliva has long been used as a sampling source for clinical diagnosis of oral disease such as oral squamous cell carcinoma, or therapeutic drug monitoring. The aims of this study was to ascertain if saliva RNA...

    Dongmei Chen, Najie Song, Runfang Ni, Jiangning Zhao, Jiasheng Hu, Quanyi Lu and Qingge Li

    Journal of Translational Medicine 2014 12:321

    Published on: 19 November 2014

  8. Research

    Detection of activated KRAS from cancer patient peripheral blood using a weighted enzymatic chip array

    The KRAS oncogene was one of the earliest discoveries of genetic alterations in colorectal and lung cancers. Moreover, KRAS somatic mutations might be used for predicting the efficiency of anti-EGFR therapeutic d...

    Ming-Yii Huang, Hsueh-Chiao Liu, Li-Chen Yen, Jia-Yuan Chang, Jian-Jhang Huang, Jaw-Yuan Wang, Chao-Peng Hsiao and Shiu-Ru Lin

    Journal of Translational Medicine 2014 12:147

    Published on: 26 May 2014

  9. Review

    The role of quantitative mass spectrometry in the discovery of pancreatic cancer biomarkers for translational science

    In the post-genomic era, it has become evident that genetic changes alone are not sufficient to understand most disease processes including pancreatic cancer. Genome sequencing has revealed a complex set of ge...

    Daniel Ansari, Linus Aronsson, Agata Sasor, Charlotte Welinder, Melinda Rezeli, György Marko-Varga and Roland Andersson

    Journal of Translational Medicine 2014 12:87

    Published on: 5 April 2014

  10. Research

    Peripheral medulloepithelioma: a rare tumor with a potential target therapy

    Medulloepithelioma (ME) is a rare embryonal tumor predominantly located in the eye or in the central nervous system without an established treatment.

    Maria Debora De Pasquale, Maria Antonietta De Ioris, Angela Gallo, Angela Mastronuzzi, Alessandro Crocoli, Raffaele Cozza and Renata Boldrini

    Journal of Translational Medicine 2014 12:49

    Published on: 21 February 2014

  11. Research

    Phosphatase of regenerating liver-3 (PRL-3) is associated with metastasis and poor prognosis in gastric carcinoma

    PRL-3 is a member of phosphatases of regenerating liver family, characterized by phosphatase active domain and C-terminal prenylation motif. Overexpression of PRL-3 has been implicated in multiple cancers. Her...

    Xiaofang Xing, Shenyi Lian, Ying Hu, Ziyu Li, Lianhai Zhang, Xianzi Wen, Hong Du, Yongning Jia, Zhixue Zheng, Lin Meng, Chengchao Shou and Jiafu Ji

    Journal of Translational Medicine 2013 11:309

    Published on: 13 December 2013

    The Erratum to this article has been published in Journal of Translational Medicine 2014 12:73

  12. Research

    Targeting Cdc42 with the small molecule drug AZA197 suppresses primary colon cancer growth and prolongs survival in a preclinical mouse xenograft model by downregulation of PAK1 activity

    Rho GTPases play important roles in cytoskeleton organization, cell cycle progression and are key regulators of tumor progression. Strategies to modulate increased Rho GTPase activities during cancer progressi...

    Karin Zins, Sandun Gunawardhana, Trevor Lucas, Dietmar Abraham and Seyedhossein Aharinejad

    Journal of Translational Medicine 2013 11:295

    Published on: 27 November 2013

  13. Research

    Patient-derived tissue slice grafts accurately depict response of high-risk primary prostate cancer to androgen deprivation therapy

    Effective eradication of high-risk primary prostate cancer (HRPCa) could significantly decrease mortality from prostate cancer. However, the discovery of curative therapies for HRPCa is hampered by the lack of...

    Hongjuan Zhao, Alan Thong, Rosalie Nolley, Stephen W Reese, Jennifer Santos, Alexandre Ingels and Donna M Peehl

    Journal of Translational Medicine 2013 11:199

    Published on: 28 August 2013

  14. Research

    Flotillin-2 is associated with breast cancer progression and poor survival outcomes

    Flotillin-2 (FLOT2) has been implicated in several signaling pathways in tumor cells. Our study aimed to investigate the expression pattern and clinicopathological significance of FLOT2 in patients with breast...

    Xi Wang, Qi Yang, Ling Guo, Xing-Hua Li, Xiao-Hui Zhao, Li-Bing Song and Huan-Xin Lin

    Journal of Translational Medicine 2013 11:190

    Published on: 15 August 2013

  15. Editorial

    Assessment of risk of bias in translational science

    Risk of bias in translational medicine may take one of three forms: A. a systematic error of methodology as it pertains to measurement or sampling (e.g., selection bias), B. a systematic defect of design that ...

    Andre Barkhordarian, Peter Pellionisz, Mona Dousti, Vivian Lam, Lauren Gleason, Mahsa Dousti, Josemar Moura and Francesco Chiappelli

    Journal of Translational Medicine 2013 11:184

    Published on: 8 August 2013

  16. Research

    Metallothionein 2A inhibits NF-κB pathway activation and predicts clinical outcome segregated with TNM stage in gastric cancer patients following radical resection

    Metallothionein 2A (MT2A) as a stress protein, plays a protective role in gastric mucosal barrier. Its role in the development of gastric cancer (GC) is unclear. The mechanism of MT2A will be investigated in g...

    Yuanming Pan, Jiaqiang Huang, Rui Xing, Xin Yin, Jiantao Cui, Wenmei Li, Jun Yu and Youyong Lu

    Journal of Translational Medicine 2013 11:173

    Published on: 19 July 2013

  17. Research

    Establishment of patient-derived non-small cell lung cancer xenograft models with genetic aberrations within EGFR, KRAS and FGFR1: useful tools for preclinical studies of targeted therapies

    Patient-derived tumor xenograft models have been established and increasingly used for preclinical studies of targeted therapies in recent years. However, patient-derived non-small cell lung cancer (NSCLC) xen...

    Xu-chao Zhang, Jingchuan Zhang, Ming Li, Xiao-sui Huang, Xue-ning Yang, Wen-zhao Zhong, Liang Xie, Lin Zhang, Minhua Zhou, Paul Gavine, Xinying Su, Li Zheng, Guanshan Zhu, Ping Zhan, Qunsheng Ji and Yi-long Wu

    Journal of Translational Medicine 2013 11:168

    Published on: 10 July 2013

  18. Research

    Low expression of LOC285194 is associated with poor prognosis in colorectal cancer

    The long non-coding RNAs (lncRNAs) study has gradually become one of the hot topics in the field of RNA biology. One lncRNA which has attracted attention is LOC285194, a lncRNA demonstrated the potential tumor...

    Peng Qi, Mi-die Xu, Shu-juan Ni, Dan Huang, Ping Wei, Cong Tan, Xiao-yan Zhou and Xiang Du

    Journal of Translational Medicine 2013 11:122

    Published on: 16 May 2013

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