Research News

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.

http://www.cancer.gov/about-cancer/treatment/clinical-trials/nci-supported/nci-match

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.

Transplant/Immunotherapy Trial

FCF and the Johns Hopkins University School of Medicine are sponsoring a trial of sequential partial liver transplantation followed by bone marrow transplantation from the same living related donor. This treatment applies to patients whose cancer remains confined to the liver but is too widespread to be removed by surgery or treated by a liver transplant from a deceased donor. The purpose of this combined treatment is to reduce the risk of the cancer coming back after the liver transplant   The bone marrow transplant may reduce the risk of the cancer coming back in two ways.  First, patients who have combined bone marrow and solid organ transplants may be able to get off all anti-rejection drugs, which inhibit the immune system from destroying cancer cells.  Second, the donor’s bone marrow contains cells of the immune system, which can attack any cancer cells that remain after the liver transplant. This trial should be actively recruiting patients early in 2016.

 

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Intellectual property from fibrolamellar discovery dedicated to the public 
Intellectual property resulting from the discovery of specific DNA mutations linked to fibrolamellar hepatocellular carcinoma, has been dedicated to the public by the institutions that made the discovery, The Rockefeller University (“Rockefeller”) and the New York Genome Center (“NYGC”). The institutions made this unusual move in the hope of accelerating progress toward the delivery of diagnostics and therapies for this devastating disease. For additional information click the link above.

Major fibrolmellar research published in Science Magazine
A research paper on genetic mutation in fibrolamellar tissue was published February 28, 2014.  This research was conducted at the Tucker Davis Research Facility at Rockefeller University.  Dr. Sandy Simon is head of the laboratory and his daughter Elana, who is a fibrolamellar patient, was a lead researcher.

Fibrolamellar Tissue Repository opens at the Tucker Davis Research Facility at Rockefeller University.Research on fibrolamellar hepatocellular carcinoma depends on the continued involvement of patients, families and researchers alike.  Donations of fibrolamellar tissue are central to the scientific research that will improve the diagnosis, treatment and understanding of this rare disease. The Fibrolamellar Tissue Repository is a tissue bank for fibrolamellar samples obtained during surgery.  All patients and families should read this link for details about donating tissue samples.

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ARTICLES ON FIBROLAMELLAR

  1.  "Model of fibrolamellar hepatocellular carcinomas reveals striking enrichment in cancer stem cells"     http://www.nature.com/ncomms/2015/151006/ncomms9070/full/ncomms9070.html
  2. "FGFR1 and FGFR2 in Fibrolamellar Carcinoma"http://www.ncbi.nlm.nih.gov/pubmed/26259677

  3. "Clinicopathologic features and survival in fibrolamellar carcinoma: comparison with conventional  hepatocellular carcinoma with and without cirrhosis",  http://www.nature.com/modpathol/journal/v18/n11/full/3800449a.html

  4. "Fibrolamellar carcinoma of the liver:  An immunohistochemical study of nineteen cases and a review of  literature",  (only a preview…have to purchase to read the entire thing) http://www.sciencedirect.com/science/article/pii/S0046817788802612
  5. "Guidelines for the Diagnosis of Hepatocellar Carcinoma (HCC) in Adults",  http://gut.bmj.com/content/52/suppl_3/iii1.long
  6. "Phase II Trial of Systemic Continuous Fluorouracil and Subcutaneous Recombinant Interferon Alfa-2b for Treatment of Hepatocellular Carcinoma"http://jco.ascopubs.org/content/21/3/421.full
  7. "A Translational Study Case Report on the Samll Molecule Emergy Blocer 3-Bromopyruvate (3BP) as a Potent Anticancer Agent:  From Bench Side to Bedside - Report on the novel anti-cancer agent 3BP - 
  8. "Fibrolamellar Carcinoma: 2012 Update" by Dr. Michael Torbenson, http://www.scientifica.com/2012/743790/
  9. "Prolonged Complete Response after GEMOX Chemotherapy in a Patient with Advanced Fibrolamellar Hepatocellular Carcinoma" - this is a singular case study.
  10. "Fibrolamellar Carcinomas are Positive for CD68" Modern Pathology (2010)
    Fibrolamellar carcinomas are a unique type of liver carcinoma that arise in non-cirrhotic livers of young individuals. Despite their distinctive appearance, recent studies have demonstrated a lack of consistency in how fibrolamellar carcinomas are diagnosed by pathologists. Read more  
  11. "Prognostic Indicators and Treatment Outcome in 94 Cases of Fibrolamellar Hepatocellular Carcinoma"
  12. DNAJB1-PRKACA is specific for fibrolamellar carcinoma - co-authored by FCF Scientific Advisory Board member Michael Torbenson from Mayo Clinic.
  13. Article abstract discussing arginine therapy for high ammonia levels in the meeting abstracts of the Society of Hospital Medicine.        

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