journal publication
In this study, the researchers used a method called mass spectrometry to study the proteins and metabolites inside FLC tumors and compared them to normal liver tissue. They found that the proteins in FLC tumors are different from those in healthy liver tissue, which could explain why FLC cells react differently to certain drugs and …
This article describes work conducted at the University Hospital Tübingen on the creation of a therapeutic peptide vaccine for fibrolamellar carcinoma. Using FLC’s characteristic DNAJB1-PRKACA fusion protein, they designed a personalized peptide vaccine using fragments of that DNAJB1-PRKACA protein and administered it to a patient with FLC. The patient, a recipient of a liver transplant, …
This article, published in Nature Reviews Gastroenterology & Hepatology, summarizes the key advances made in understanding the disease since the 2014 discovery that nearly all FLC tumors contain the DNAJB1-PRKACA chimeric fusion transcript. The paper outlines the advances made in understanding the molecular pathways driven by the fusion protein and the potential to identify actionable …
Rare tumors contribute significantly to the burden of cancer in the United States, yet are often poorly understood and have inadequate research support. To address this situation, the National Cancer Institute’s (NCI) My Pediatric and Adult Rare Tumor Network (MyPART) was developed. MyPART’s goals include connecting participants and investigators through shared infrastructure and networks; accelerating …
While the significance of the DNAJB1-PRKACA fusion gene in fibrolamellar carcinoma has been established and validated, the role of microRNAs (miRNAs) in FLC remains unclear. MicroRNAs have been implicated as drivers in many diseases including a variety of cancers. This study analyzed small RNA sequencing data to identify dysregulated miRNAs in FLC tumors and identified …
This study was the first published research effort that identified the DNAJB1-PRKACA chimeric transcript in fibrolamellar carcinoma tumors. By performing RNA sequencing on tumor and healthy tissue samples, a chimeric transcript was discovered that had not been previously reported in the literature. This fusion transcript arises due to a ~400-kilobase deletion on chromosome 19. In …
The DNAJB1-PRKACA fusion gene is found in almost all FLC tumors. In order to determine if this gene fusion is a cause of the tumors, these researchers used a CRISPR-Cas9 technique to create a chimeric DNAJB1-PRKACA fusion gene in the livers of otherwise healthy mice. Using the CRISPR-Cas9 system, the researchers created guide RNAs that …
“Organoids,” three-dimensional cell cultures that mimic critical features of normal human organs, are a novel tool to study fundamental steps in the development of cancers. The authors of this paper, a team from Utrecht, The Netherlands, wanted to understand how specific genetic alterations contribute to the transformation of normal liver cells into fibrolamellar carcinoma (FLC). …
In this study, researchers reviewed the detailed medical records of 262 patients under age 20 diagnosed with liver cancers, assembled from 19 different hospitals and covering the years 1990 to 2017. This represents the richest available source of data on these pediatric liver cancers. They found that 119 (45%) of the tumors had been classified …