Toward First Drug to Treat a Rare, Lethal Liver Cancer

Treatment options for a deadly liver cancer, fibrolamellar carcinoma, are severely lacking. Drugs that work on other liver cancers are not effective, and although progress has been made in identifying the specific genes involved in driving the growth of fibrolamellar tumors, these findings have yet to translate into any treatment. For now, surgery is the only option for those affected—mostly children and young adults with no prior liver conditions. Sanford M. Simon, PhD, and his group at The Rockefeller University understood that patients dying of fibrolamellar could not afford to wait. “There are people who need therapy now,” he says. So, his group threw the kitchen sink at the problem and tested over 5,000 compounds, either already approved for other clinical uses or in clinical trials, to see whether any of the compounds could be repurposed to treat fibrolamellar. The team ultimately discovered a few classes of therapeutics that destroy fibrolamellar tumor cells growing in mice. Their findings were published online on June 14, 2021 in Cancer Discovery. The open-access article is titled “Identification of Novel Therapeutic Targets for Fibrolamellar Carcinoma Using Patient Derived Xenografts and Direct from Patient Screening.”