Fibrolamellar Cancer Foundation

Oncogenic Addiction of Fibrolamellar Hepatocellular Carcinoma to the Fusion Kinase DNAJB1-PRKACA

Based on another study from the Simon lab, we have known that a gene fusion called DNAJB1-PRKACA produced a mutated protein kinase A (PKA) that causes 99% of cases of fibrolamellar carcinoma. In this study, the researchers found that this gene is not only important for the growth of FLC tumors but also that blocking …

Defining incidence and complications of fibrolamellar liver cancer through tiered computational analysis of clinical data

Rare diseases like fibrolamellar are often poorly understood because of the difficulty in assembling accurate patient data and the lack of ICD billing codes specific to the disease. The most cited study of the incidence of FLC in the US, using data from the National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) Program, reports …

Disruption of proteome by an oncogenic fusion kinase alters metabolism in fibrolamellar hepatocellular carcinoma

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 …

NCI intramural program approach to rare tumors: Natural history study of rare solid tumors in children and adults: A longitudinal, comprehensive data and biospecimen collection protocol

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 …

Organoid models of fibrolamellar carcinoma mutations reveal hepatocyte transdifferentiation through cooperative BAP1 and PRKAR2A loss

“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). …

A pilot study of a DNAJB1-PRKACA fusion kinase peptide vaccine combined with nivolumab and ipilimumab for patients with fibrolamellar hepatocellular carcinoma

In an abstract release for the the 2023 annual meeting of the Society for Immunotherapy of Cancer, the Johns Hopkins University team released the initial results of their peptide vaccine clinical trial launched in 2020. The trial investigated the potential effectiveness of a targeted vaccine against FLC’s DNAJ-PKAc driver when combined with the checkpoint inhibitors …