Noncoding RNAs as Predictive Biomarkers of Therapeutic Response to Tyrosine Kinase Inhibitors in Metastatic Cancer

Since their discovery, noncoding RNAs have acquired extensive attention due to their eminent role in the regulation of gene expression and thus also in the pathogenesis of many diseases. Currently, strong evidence is showing that noncoding RNAs are integral parts of key cancer-related cellular pathways, and the deregulation of their levels is pathogenetic on one hand but feasible as a biomarker of pathogenesis itself on the other hand. In cancer, diagnosis, prognosis, and prediction of therapy outcome can be derived from levels of various noncoding RNAs. This chapter is focused on potential application of noncoding RNAs in prediction of therapeutic response to tyrosine kinase inhibitors commonly used as targeted therapy in a wide range of metastatic cancers

MicroRNAs in colorectal cancer: translation of molecular biology into clinical application

MicroRNAs (miRNAs) are small non-coding RNAs 18-25 nucleotides in length that downregulate gene expression during various crucial cell processes such as apoptosis, differentiation and development. Changes in the expression profiles of miRNAs have been observed in a variety of human tumors, including colorectal cancer (CRC). Functional studies indicate that miRNAs act as tumor suppressors and oncogenes. These findings significantly extend Vogelstein's model of CRC pathogenesis and have shown great potential for miRNAs as a novel class of therapeutic targets. Several investigations have also described the ability of miRNA expression profiles to predict prognosis and response to selected treatments in CRC patients, and support diagnosis of CRC among cancer of unknown primary site. miRNAs' occurrence has been repeatedly observed also in serum and plasma, and miRNAs as novel minimally invasive biomarkers have indicated reasonable sensitivity for CRC detection and compare favorably with the fecal occult blood test. In this review, we summarize the knowledge regarding miRNAs' functioning in CRC while emphasizing their significance in pathogenetic signaling pathways and their potential to serve as disease biomarkers and novel therapeutic targets

The role of ncRNAs in solid tumors prognosis: from laboratory to clinical utility

Today we know that non-coding RNAs (ncRNAs) represent most of the transcribed human genome and participate in relevant cellular processes. NcRNAs regulate from RNA transcription to protein translation, have important epigenetic roles or facilitate protein–protein interactions among other functions. In consequence, their dysregulation has been associated with tumor development and progression. Recently, their expression has also been detected in body fluids, opening the use of circulating ncRNAs for diagnosis and for evaluation and monitoring cancer prognosis

Case report: rapid and durable response to PDGFR targeted therapy in a child with refractory multiple infantile myofibromatosis and a heterozygous germline mutation of the PDGFRB gene

Timeline. This file shows timeline of both described cases. (PDF 466 kb