journal publication
Fibrolamellar carcinoma (FLC) is a cancer that doesn’t respond well to current treatments. In past research, the Rockefeller University team conducted a broad-based, small-molecule drug screen to identify potential drug candidates to use against FLC. That effort identified several potential drugs including napabucasin, panobinostat, irinotecan (a topoisomerase I inhibitor), and navitoclax (an inhibitor of the …
This paper reports an important research tool, the first example of a tumor line from a human fibrolamellar carcinoma (FLC) patient that can be maintained and expanded by growing the tumors in special mice with a compromised immune system. Such so-called “patient-derived xenografts” (PDX) can be used to provide large amounts of tumor tissue for …
This publication summarizes the current understanding of fibrolamellar carcinoma based on a review of currently available literature. It outlines the epidemiology, pathology, and biology of FLC, plus provides a high-level overview of the evidence supporting existing therapies for the disease — including surgical, radiotherapy, interventional radiology and systemic treatments. The full article can be read …
Proteomics studies assess the composition, structure, expression, and the interactions and connections between proteins at an overall level in tissue. Metabolomics studies focus on characterizing metabolites, the small molecules generated by specific cellular processes. Both types of studies can yield valuable insights into the complex biochemical pathways, networks, and molecular systems that control important cell …
This publication describes the results of the first large scale screening of known drugs and compounds for their effectiveness against FLC. It identified six new PDX lines derived by direct implantation of tumor specimens, both from the liver (one primary and one recurrent tumor) and metastases. These facilitated the first reported large-scale screening of compounds …
Although immune checkpoint inhibitors (ICIs) are becoming the standard of care in some types of cancers, few studies have looked at the safety and efficacy of ICIs for treating FLC. This multicenter, retrospective analysis aimed to determine if treatment with ICIs could be helpful for FLC patients. Patient data included in this study was identified …
While FLC in almost all patients is caused by the DNAJB1-PRKACA gene fusion, the mechanism by which that fusion causes the cancer is still unclear. In order to get insight into that question, this team engineered the DNAJB1-PRKACADP fusion gene into “immortalized” liver cells and measured the impact that the change had on the functions …
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 …
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 …