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Publication : Preclinical Models Provide Scientific Justification and Translational Relevance for Moving Novel Therapeutics into Clinical Trials for Pediatric Cancer.

First Author  Langenau DM Year  2015
Journal  Cancer Res Volume  75
Issue  24 Pages  5176-5186
PubMed ID  26627009 Mgi Jnum  J:227331
Mgi Id  MGI:5700247 Doi  10.1158/0008-5472.CAN-15-1308
Citation  Langenau DM, et al. (2015) Preclinical Models Provide Scientific Justification and Translational Relevance for Moving Novel Therapeutics into Clinical Trials for Pediatric Cancer. Cancer Res 75(24):5176-86
abstractText  Despite improvements in survival rates for children with cancer since the 1960s, progress for many pediatric malignancies has slowed over the past two decades. With the recent advances in our understanding of the genomic landscape of pediatric cancer, there is now enthusiasm for individualized cancer therapy based on genomic profiling of patients' tumors. However, several obstacles to effective personalized cancer therapy remain. For example, relatively little data from prospective clinical trials demonstrate the selective efficacy of molecular-targeted therapeutics based on somatic mutations in the patient's tumor. In this commentary, we discuss recent advances in preclinical testing for pediatric cancer and provide recommendations for providing scientific justification and translational relevance for novel therapeutic combinations for childhood cancer. Establishing rigorous criteria for defining and validating druggable mutations will be essential for the success of ongoing and future clinical genomic trials for pediatric malignancies. Cancer Res; 75(24); 5176-86. (c)2015 AACR.
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