Public Release: 

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

Rockefeller University

NEW YORK -- 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."

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About Fibrolamellar Hepatocellular Carcinoma

Fibrolamellar hepatocellular carcinoma (FHC) is a rare liver cancer that usually occurs in teens and young adults who have no history of liver disease. FHC occurs in only 1 in 5,000,000 people and is considered an ultra-rare cancer. Typical symptoms include abdominal, shoulder or back pain, loss of appetite, weight loss, jaundice, and occasionally a palpable mass, some of which are associated with advanced disease. The typical treatment is surgical removal of the tumor, which can be curative if done before the tumor has spread, or metastasized. When the tumor cannot be removed surgically or when there is distant spread, other therapies may be used to slow down the spread of the disease. There is no standard regimen so non-surgical therapy varies from patient to patient. These therapies are not typically curative but may shrink tumors for a period of time. New treatments that offer curative potential in patients with advanced disease are urgently needed.

About The Rockefeller University

The Rockefeller University is the world's leading biomedical research university and is dedicated to conducting innovative, high-quality research to improve the understanding of life for the benefit of humanity. Our 81 laboratories conduct research in neuroscience, immunology, biochemistry, genomics, and many other areas, and a community of 1,800 faculty, students, postdocs, technicians, clinicians, and administrative personnel work on our 14-acre Manhattan campus. Our unique approach to science has led to some of the world's most revolutionary and transformative contributions to biology and medicine. During Rockefeller's 115-year history, 24 of our scientists have won Nobel Prizes, 22 have won Albert Lasker Medical Research Awards, and 20 have garnered the National Medal of Science, the highest science award given by the United States.

About the Fibrolamellar Cancer Foundation

The Fibrolamellar Cancer Foundation (FCF), established in 2009, is a nonprofit organization devoted to finding cures and treatment options for patients with fibrolamellar hepatocellular carcinoma. FCF's mission is three fold: to find a cure and treatment options; to raise awareness of the disease; and to connect and support the fibrolamellar community of patients and their families. Based in Greenwich, CT, FCF is an IRS-approved 501(c)(3) public charity whose funding decisions are guided by medical and scientific advisors from top U.S. cancer research and treatment centers. To learn more, visit http://www.fibrofoundation.org

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