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Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma
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...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Nature Publishing Group UK
2023
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10022574/ https://www.ncbi.nlm.nih.gov/pubmed/36932129 http://dx.doi.org/10.1038/s41568-023-00554-w |
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author | Simon, Sanford M. |
author_facet | Simon, Sanford M. |
author_sort | Simon, Sanford M. |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-10022574 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Nature Publishing Group UK |
record_format | MEDLINE/PubMed |
spelling | pubmed-100225742023-03-17 Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma Simon, Sanford M. Nat Rev Cancer Perspective 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. Nature Publishing Group UK 2023-03-17 2023 /pmc/articles/PMC10022574/ /pubmed/36932129 http://dx.doi.org/10.1038/s41568-023-00554-w Text en © Springer Nature Limited 2023, corrected publication 2023Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law. This article is made available via the PMC Open Access Subset for unrestricted research re-use and secondary analysis in any form or by any means with acknowledgement of the original source. These permissions are granted for the duration of the World Health Organization (WHO) declaration of COVID-19 as a global pandemic. |
spellingShingle | Perspective Simon, Sanford M. Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma |
title | Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma |
title_full | Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma |
title_fullStr | Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma |
title_full_unstemmed | Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma |
title_short | Fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma |
title_sort | fighting rare cancers: lessons from fibrolamellar hepatocellular carcinoma |
topic | Perspective |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10022574/ https://www.ncbi.nlm.nih.gov/pubmed/36932129 http://dx.doi.org/10.1038/s41568-023-00554-w |
work_keys_str_mv | AT simonsanfordm fightingrarecancerslessonsfromfibrolamellarhepatocellularcarcinoma |