Year: 2023
Type: review
Abstract: The fight against rare cancers faces myriad challenges, including missed or wrong diagnoses, lack of information and diagnostic tools, too few samples and too little funding. Yet many advances in cancer biology, such as the realization that there are tumour suppressor genes, have come from studying well-defined, albeit rare, cancers. Fibrolamellar hepatocellular carcinoma (FLC), a typically lethal liver cancer, mainly affects adolescents and young adults. FLC is both rare, 1 in 5 million, and problematic to diagnose. From the paucity of data, it was not known whether FLC was one cancer or a collection with similar phenotypes, or whether it was genetically inherited or the result of a somatic mutation. A personal journey through a decade of work reveals answers to these questions and a road map of steps and missteps in our fight against a rare cancer. Fibrolamellar hepatocellular carcinoma is a rare cancer type and, as such, research into this disease comes with many challenges. In this Perspective, Sanford Simon tells of his personal journey and experiences in the fight against this rare cancer type.
Source: Nature reviews. Cancer
Author: Sanford M. Simon
Institution: Rockefeller University
Language: English
Subfield: Hepatology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Awards: Center for therapeutic targeting of the Fusion Oncoprotein of Fibrolamellar Hepatocellular Carcinoma, ASO and shRNA for targeting the oncogenic transcript driving fibrolamellar hepatocellular carcinoma, Mayo Clinic Hepatobiliary SPORE, Developing, Demonstrating, and Disseminating Innovative Programs to Achieve Translational Success
OpenAlex ID: W4327716983