Research News

Yvonne Janssen-Heininger, Ph.D., foreground.

Clinical Trials

Johns Hopkins University, Baltimore, MD

A new clinical trial of an immune therapy for fibrolamellar carcinoma (FLC) is now recruiting subjects at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University in Baltimore, MD. The study asks if individuals can mount an effective immune response against FLC by specifically targeting the unique chimeric protein (resulting from a DNAJB1-PRKACA gene fusion) believed to drive the growth of almost all such tumors. Trial subjects will be given an experimental vaccine (the first patient in the trial received this vaccine on April 21st) containing a peptide (small segment of a protein) that corresponds to the junction region linking the two parts of the chimeric protein. They will simultaneously receive two FDA-approved drugs, Opdivo (nivolumab) and Yervoy (ipilimumab), that may enhance the immune response against FLC by overcoming “checkpoint” systems that can limit the immune system’s ability to fight a cancer. The study’s principal investigator is Dr. Mark Yarchoan. Details and contact information can be found at:


Neratinib HER Mutation Basket Study

This is an open-label, multicenter, multinational, Phase 2 study exploring the efficacy and safety of neratinib therapy in patients with solid tumors with activating HER2, HER3 or EGFR mutations or with EGFR gene amplification. The trial will consist of a screening period, a treatment period, and an end of treatment visit occurring when neratinib is discontinued for any reason, a safety follow-up visit occurring 28 to 42 days after the last dose of neratinib and a survival follow-up period lasting for a maximum of 12 months for each patient after their last dose of neratinib or until initiation of additional anti-cancer therapy. For more information go to:

NCI Clinical Trial

The National Cancer Institute has announced the launch of a Molecular Analyses for Therapy Choice (MATCH) Clinical Trial, exploring the effectiveness of targeted cancer therapy based on genetic mutation rather than type of cancer. There is a Rare Cancer arm to this trial, so Fibrolamellar patients should be eligible to participate.  Please click the link below for details and talk to your doctor to see if this is right for you. Learn More

Note: while at this point only adults 18 and over may participate, a pediatric version for those under 18 is in development for early 2016 and is referenced if you scroll down on the page in the above link.

CASI Pharmaceuticals Clinical Trial

Casi Pharmaceuticals is sponsoring a Phase 2 clinical trial which is currently recruiting patients. The purpose of the study is to determine whether once-daily dosing with ENMD-2076 will be a safe and effective treatment in patients with fibrolamellar. Safety will be measured by looking at the adverse events that may happen and the efficacy will look at the progression of the disease over time.  The primary objective of the trial is to determine the 6-month progression free survival (PFS6) rate when patients with advanced fibrolamellar carcinoma (FLC) are treated with daily oral ENMD 2076.  A detailed explanation of this trial can be found here.  The study chair for this trial is Ghassan Abou Alfa, who is one of FCF’s medical and scientifc advisors.

Report on 2017 FCF Scientific Conference

The distinquished Journal of Hepatocellular Carcinoma has published a report on FCF’s 2017 Scientific Conference. Authors are Drs. Ted Kastenhuber, Ghassan AbouAlfa, and John Craig who coordinated the writing and editing. FCF thanks these doctors for their time and commitment to this article. The article, which can be viewed here,  is titled, “Road map for fibrolamellar carcinoma: progress and goals of a diversified approach”.


Articles on Fibrolamellar 

May 2019

An acquired scaffolding function of the DNAJ-PKAc fusion contributes to oncogenic signaling in fibrolamellar carcinoma – by multiple authors including FCF funded researchers at U of Washington, Rigney Turnham and Donelson Smith

Jan 2019

MicroRNA-375 suppresses the growth and invasion of fibrolamellar carcinoma – by multiple authors and collaborating laboratories supported by FCF funding

Jan 2019 


Road map for fibrolamellar carcinoma – progress and goals of a diversified approach – this is a report from FCF’s Scientific Conference November 2017

Sept 2017

CRISPR/Cas9 Engineering of Adult Mouse Liver Demonstrates That the Dnajb1-Prkaca Gene Fusion is Sufficient to Induce Tumors Resembling Fibrolamellar Hepatocellular Carcinoma. – by multiple authors including Francesco Niola, FCF grant applicant

May 2017

Genome and transcriptome profiling of fibrolamellar hepatocellular   carcinoma demonstrates p53 and IGF2BP1 dysregulation – by multiple authors including FCF advisors Drs. DeMatteo and Klimstra

March 2017

 “Comprehensive analysis of The Cancer Genome Atlas reveals a unique gene and non-coding RNA signature of fibrolamellar carcinoma”– by multiple authors including FCF advisors Dr. Michael S.Torbenson, Lola M. Reid, PH.D & Praveen Sethupathy, PH.D

March 2017

“Environmental Exposures as a Risk Factor for Fibrolamellar Carcinoma” – by multiple authors including Dr. John Craig, Board member of FCF, and Dr. Michael Torbenson, advisor to FCF.

May 2016

“Hyperammonemic Encephalopathy Associated With Fibrolamellar Hepatocellular Carcinoma: Case Report, Literature Review, and Proposed Treatment Algorithm” – by multiple authors including Dr. Olga N. Kozyreva, a grant reviewer for FCF.

December 2015

The DNAJB1-PRKACA chimera: Candidate biomarker and therapeutic target for fibrolamellar carcinomas – by Lola M. Reid, PH,D., and Praveen Sethupathy PH.D

October 2015

Model of fibrolamellar hepatocellular carcinomas reveals striking enrichment in cancer stem cells” – by multiple authors including FCF funded researchers Dr. Nancy Carrasco, Dr. Praveen Sethupathy and Dr. Lola M. Reid.

October 2015

 “FGFR1 and FGFR2 in Fibrolamellar Carcinoma“, numerous authors including FCF medical advisor Dr. Michael Torbenson.

August 2015

 “Fibrolamellar carcinoma treatment and management” – by FCF Scientific/Medical Advisor, Dr. Michael Choti, Medscape Reference.

February 2015

 DNAJB1-PRKACA is specific for fibrolamellar carcinoma – multiple authors including FCF medical advisor Dr. Michael Torbenson

December 2014

 Lim II, Farber BA, LaQuaglia MP. “Advances in fibrolamellar hepatocellular carcinoma: a review” Eur J Pediatr Surg. December 2014; 24(6):461-466.

November 2014

 Arif Suriawinata, MD. Pathology of malignant liver tumors. UpToDate. November 2014; Accessed 4/22/2015.

February 2014

May 2013


September 2013

 “A Rare but Rapidly Reversible Cause of Hyperammonemic Encephalopathy”, multiple authors from Cleveland Clinic

Prognostic Indicators and Treatment Outcome in 94 Cases of Fibrolamellar Hepatocellular Carcinoma“, multiple authors including FCF medical advisor Dr. Ahmed Kaseb

April 2012

 Prolonged Complete Response after GEMOX Chemotherapy in a Patient with Advanced Fibrolamellar Hepatocellular Carcinoma”. This is a singular case study.

January 2012

 “A Translational Study Case Report on the Small Molecule Energy Blocker 3-Bromopyruvate (3BP) as a Potent Anticancer Agent:  From Bench Side to Bedside” 


 “Fibrolamellar Carcinoma: 2012 Update” – by FCF Medical/Scientific Advisor Dr. Michael Torbenson

May 2005

 Clinicopathologic features and survival in fibrolamellar carcinoma: comparison with conventional  hepatocellular carcinoma with and without cirrhosis


 “Phase II Trial of Systemic Continuous Fluorouracil and Subcutaneous Recombinant Interferon Alfa-2b for Treatment of Hepatocellular Carcinoma”

November 2002

 “Guidelines for the Diagnosis of Hepatocellar Carcinoma (HCC) in Adults”

July 1988

 “Fibrolamellar carcinoma of the liver:  An immunohistochemical study of nineteen cases and a review of  literature”