Patients in Fibrolamellar Age Range (12-18) Now Recommended to Participate in Adult Clinical Trials (December, 2016). The FDA is now recommending enrolling adolescents in disease/target-appropriate adult oncology clinical trials. This is an important step that will open up more clinical trials to fibrolamellar patients. Learn more
New Diagnostic Test for Fibrolamellar (December, 2016) A new test, developed at Mayo Clinic, uses FISH technology to identify the fusion gene DNAJB1-PRKACA, reported in fibrolamellar carcinomas.
Fibrolamellar Registry On Stage at White House as Cancer Moonshot Billis Signed (December, 2016) This past week the US House and Senate passed the 21st Century Cures Act. This provides $5 billion for the NIH for The Precision Medicine Initiative, Brain Initiative and the Moonshot on Cancer - a legislation established earlier this year aiming to make therapies more available and improving early stage prevention. Its emphasis was given to pediatric, adolescent and young adult cancers, including fibrolamellar. FibroRegistry board member Elana Simon attended the ceremony, standing on stage with House Speaker Nancy Pelosi, Vice President Joe Biden, other patients and members of Congress, as President Obama signed the bill.
Fibrolamellar Registry Launch Garners Strong Response (December, 2016) Just two weeks into its launch, the FibroRegistry has seen an extremely strong response, with dozens of patients in the United States and around the world taking part. In fact, 23 states, 8 countries and 3 continents are represented. Outreach efforts to patients will continue throughout the next year, along with new efforts to reach relevant physicians and medical organizations.
The Fibrolamellar Registry Goes Live (November, 2016)The Fibrolamellar Registry team proudly announces that the survey is now open for participants. Patients and/or their family representatives are invited to take the survey and help bring us closer to our goals of better treatments, diagnostics and, one day, a cure.
Fibrolamellar Registry Wins Approval from the Genetical Alliance Institutional Review Board (September, 2016) Following nearly two years of development, the Fibrolamellar Registry wins approval from the Genetic Alliance's IRB to launch as a scientific study.
New Fibrolamellar Drug Discovery Program Launches (September, 2016). Blueprint Medicines announces new drug discovery program targeting the fibrolamellar chimera. Research will be conducted in the lab of Dr. Sanford Simon, Rockefeller University professor, president of the Fibrolamellar Registry.
Clinical Trial for Fibrolamellar Enters Phase 2 A trial testing whether a compound called ENMD-2076 is a safe and effective treatment for fibrolamellar meets its Phase 1 endpoint with 16 patients and proceeds to a second stage.
"Humans of New York" Features Fibrolamellar Researcher (May, 2016) Fibrolamellar researcher/ surgeon Dr. Michael La Quaglia, chair of Pediatric Surgery at Memorial Sloan Kettering and scientific collaborator of the FibroRegistry team, is featured in the Humans of New York Pediatric Cancer Series on Facebook.
Fibrolamellar Registry Joins Cancer Coalition Lobbying Effort (May, 2016) Fibrolamellar patients and families are joining the Rally Foundation for Childhood Cancer Research and the Nicholas Conor Institute to support their efforts to have the Department of Defense include funding for young adult and pediatric cancer. Click here to get involved!
Fibrolamellar Highlighted at Vatican International Cancer Conference (April, 2016) Fibrolamellar researcher/ Registry director Elana Simon spoke about fibrolamellar at the Third Annual International Cancer Conference, held at The Vatican, in Rome, to an international audience of clergy, medical researchers and others about the importance of funding rare pediatric and young adult cancer research. More
Fibrolamellar Registry Wins Genetic Alliance/PEER Award (March, 2015) The Fibrolamellar Registry has been named one of the awardees of Genetic Alliance's PEER program. We are one of several disease advocacy groups that have been selected to develop and use the PEER health data registry. This registry will enable fibrolamellar patients to share health information with researchers and each other to advance research and accelerate the development of cohorts for clinical trials. The Fibrolamellar Registry congratulates fellow awardees including the American College of Nurse-Midwives; the Asthma and Allergy Foundation of America; the Celiac Support Association; the Centre for Health, Law and Emerging Technologies; and the Medical Investigation of Neurodevelopmental Disorders Institute at University of California, Davis.
New Drug Trial (February, 2015) CASI Pharmaceuticals announces that it plans to conduct a "single arm" trial to evaluate the response rate to a new drug called "ENMD-2076" which may be a first line therapy for fibrolamellar. If the results of the proposed study show that ENMD-2076 improves overall tumor response, CASI says it "would expect to propose a confirmatory clinical trial and, in parallel, submit an NDA for accelerated approval of the drug." Source
Fibrolamellar Registry Receives New Grant Thanks to the Richard Lounsbery Foundation of Washington, D.C., for providing a grant to support development of the Registry.
Fibrolamellar at the White House (January, 2015) Fibrolamellar Registry vice president/ fibrolamellar patient Elana Simon introduces President Barack Obama at the White House event announcing the federal Precision Medicine Initiative. Click to view A letter with the headline "Fibrolamellar Hepatocellular Carcinoma" is sent from the White House to millions of subscribers on its email list. Click to view White House Blog
Fibrolamellar Blogger/Paramedic Featured in Newspaper (January, 2015) Fibrolamellar patient Matt Cler, who has been blogging about his experience, is featured in the Aberdeen News. Click to view article
Twelve Fibrolamellar Studies Published in 2015Source
Fibrolamellar Blogger/Paramedic Featured on TV (December, 2014) Fibrolamellar patient Matt Cler tells his story on South Dakota television. Click to view
Four Fibrolamellar Studies Published (December, 2014) Source
Vancouver Fibrolamellar Patient Profiled in Huffington Post (November, 2014) Fibrolamellar patient Tessa Williams, of Vancouver, advocates for organ donations in the Huffington Post. View the article and video
Family of Fibrolamellar Victim Making Educational Film (October, 2014) The mother and sister of British fibrolamellar victim, Bronte Doyne, have been asked to participate in an educational film being made by the hospital that treated her. "After Bronte's death, her mother Lorraine Doyne contacted Nottingham University Hospitals Trust with a number of complaints over the way she felt the family were treated. Her story will now be presented to staff in a film to help them support families in similar situations," according to an article. Click to read more
The Fibrolamellar Registry Joins patientINFORM (September, 2014) The Fibrolamellar Registry has been accepted to join the patient advocacy group, patientINFORM. This will allow our website to offer free links to scientific articles normally locked behind costly paywalls. We will now be able to make such articles accessible to patients and their caregivers. patientInform is a collaborative private-sector initiative of medical publishers, health organizations, medical societies, and health information professionals. Its goal is to allow patients and others to learn more about a specific disease or its treatment to access the most important new research articles through the web sites of participating health organizations or publishers.