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Plain English summary of "Transcriptomic characterization of fibrolamellar hepatocellular carcinoma"

Published in the Proceedings of the National Academy of Sciences, November 3, 2015.  Link to article

In previous publications this group showed that there is only one change in the DNA of fibrolamellar patients: a deletion in one copy of chromosome 19 that causes two genes to fuse together.  In this paper the group shows that this single change in the DNA results in many different changes in the expression of different genes: over 3500 genes either increase or decrease very significantly. 

The group looked at two kinds of changes in the expression of the genes: They measured the changes in the levels of the messenger RNAs and they measure the levels of proteins in the cell.  Why is this significant?  Some of the genes that are increased help explain many of the previously observed properties in fibrolamellar cells such as the fibers which give the disease its name.  Most importantly, the work identified a few pathways that are increased in fibrolamellar - pathways that are known to drive other tumors.  Based on these results, the group proposed that a number drugs that are currently used for other cancers, could be “repurposed” for fibrolamellar.  One that they proposed are drugs that affect the Aurora Kinase A pathway.  The results of this paper, even a year before they were published, were provided to the FDA and became the basis, on the recommendations of the authors of this paper, for the current clinical trial ENMD2076.  Other clinical trials, based on other results in this paper, are in preparation.