Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma

Nat Rev Cancer. 2023 May;23(5):335-346. doi: 10.1038/s41568-023-00554-w. Epub 2023 Mar 17.

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.

Publication types

  • Review
  • Research Support, N.I.H., Extramural

MeSH terms

  • Carcinoma, Hepatocellular* / diagnosis
  • Carcinoma, Hepatocellular* / genetics
  • Carcinoma, Hepatocellular* / pathology
  • Humans
  • Liver Neoplasms* / genetics
  • Liver Neoplasms* / pathology
  • Phenotype

Supplementary concepts

  • Fibrolamellar hepatocellular carcinoma