Fibrolamellar hepatocellular carcinoma (FLHCC) is an often-lethal disease affecting primarily children and young adults. The poor prognosis is exacerbated by the lack of both existing diagnostic tests and systemic therapy. We have recently demonstrated that there is a single, consistent deletion in one copy of chromosome 19 in FLHCC patients that results in a chimeric gene. This chimera encodes a fusion protein, a constitutively active catalytic subunit of protein kinase A. This project has two aims: (1) develop the first diagnostic tests for this cancer and (2) develop the first blockers for the driver for this cancer.

Public Health Relevance

Our ability to understand human cancers has been revolutionized the past two decades by the sequencing of the human genome and the ability to introduce altered genes into cells. This project builds upon this by using the study of a pediatric cancer to drive the study of critical genes in human hepatic cells. The project will develop the first diagnostic tests and therapeutic treatments for fibrolamellar hepatocellular carcinoma.

Agency
National Institute of Health (NIH)
Institute
National Cancer Institute (NCI)
Type
Specialized Center (P50)
Project #
1P50CA210964-01A1
Application #
9418221
Study Section
Special Emphasis Panel (ZCA1)
Project Start
Project End
Budget Start
2018-09-01
Budget End
2019-08-31
Support Year
1
Fiscal Year
2018
Total Cost
Indirect Cost
Name
Mayo Clinic, Rochester
Department
Type
DUNS #
006471700
City
Rochester
State
MN
Country
United States
Zip Code
55905