Grant awarded to Sanford Simon will fund research to treat rare liver cancer

Dr. Sanford Simon, head of the Laboratory of Cellular Biophysics at Rockefeller University, has been awarded a $600,000 grant by the Fibrolamellar Cancer Foundation (FCF) to develop a therapy for fibrolamellar hepatocellular carcinoma. This disease typically occurs in adolescents and young adults who have no history of liver disease, and there is no standard regimen for this often lethal form of cancer.

The FCF is a public, non-profit organization established primarily to support the funding of research related to fibrolamellar hepatocellular carcinoma. The organization also aims to raise awareness about the disease and foster a global community of patients and caregivers affected by this cancer. In total, the FCF has contributed $1.7 million to Simon's research on fibrolamellar since 2010.

"It can be difficult for rare diseases like fibrolamellar, especially those in children, to receive attention and resources," says Simon. "Yet, these are important to study. Young kids have the potential of many decades of life ahead of them. In addition, studies of these rare pediatric cancers have provided critical lessons for all cancers. From studying the rare pediatric cancer retinoblastoma, researchers learned about a large class of molecules called tumor suppressors. Fibrolamellar in particular is caused by single mutations, which means there is great potential for designing and testing diagnostics and cures. The support of the Fibrolamellar Cancer Foundation was essential for us to identify the driver for this cancer and will allow us to continue this important work."

The most recent grant will support work to identify compounds to treat fibrolamellar by halting the cancer-causing activity that results from a mutation the lab discovered in 2014.

"Dr. Simon's research over the past four years has made a critical difference in uncovering promising discoveries in fibrolamellar," says John Hopper, FCF Executive Director. "The foundation's $1.7 million investment to date, supporting Rockefeller's efforts, is the largest of any kind behind this cancer. This attests to our hope and belief that curative therapies are possible with both great research institutions and great investigators focused on it."

Provided by Rockefeller University