Timeframe: 2016 – 2018
Goal: Develop a pre-clinical mouse model for fibrolamellar carcinoma
Principal Investigator: Dr. Julien Sage, Ph.D., Associate Professor, Department of Pediatrics and Genetics
There are few effective therapies for FLC patients and development of improved therapeutics are hampered by the rarity of the disease and the challenge of including pediatric patients in many clinical trials. One solution to this problem is the development of accurate models of FLC. This study proposed to generate a mouse model for FLC. Because FLC occurs primarily in children and young adults, they hypothesized that the expression of the DnaJ-PKA fusion found in FLC tumors cells initiates cancer in specific liver stem/progenitor cell populations during development. Using genetic engineering techniques, they attempted to introduce the DnaJ-PKA fusion into the mouse genome in an inducible manner. Therefore, they hoped to be able to switch on expression of DnaJ-PKA at specific stages of liver development to examine exactly how this fusion contributes for FLC formation. Such mouse models would provide a novel preclinical platform to test future therapeutic strategies.
For additional details see the article published by Stanford on FCF’s grant and collaboration.