The Fibrolamellar Cancer Foundation

08/01/2025

As summer heats up, so does FCF's commitment to fibrolamellar research. We've launched our 2025 Summer Appeal and invite you to join our effort to develop more effective treatments for fibrolamellar patients.

The Fibrolamellar Cancer Foundation (FCF) is now supporting four groundbreaking clinical trials, an extraordinary milestone for our rare disease community. One of these trials has already saved lives. But advancing this important work is expensive. Each of these clinical trials has required over $1 million of FCF support.
Continuing this progress is only possible with your help. Every donation—large or small—helps us test and deliver innovative new fibrolamellar treatments. And now, through September 10, 2025, Chuck and Marna Davis will match every gift to FCF dollar for dollar.

Please visit https://fibrofoundation.org/giving to make a tax-deductible gift today. As always, 100% of your donation will fund research.

Thank you!

As summer heats up, so does FCF's commitment to fibrolamellar research. We've launched our 2025 Summer Appeal and invite you to join our effort to develop mor

06/23/2025

REGISTRATION IS NOW OPEN for
FCF 2025 Patient & Family Gathering
Sept 12 - Sept 14, 2025
Lake Mansfield Trout Club, Stowe, VT

Registration is now open for the Patient & Family Gathering to be held on September 12 - 14, 2025, at the Lake Mansfield Trout Club in Stowe, Vermont. This

06/19/2025

On June 9 – 11, 2025, FCF hosted its sixth Fibrolamellar Scientific Summit. The goal of this meeting was to bring together the fibrolamellar research community to share knowledge and to collaborate in the search for a cure.

The opening session featured a panel discussion highlighting the latest advancements in targeted immunotherapy for FLC. Titled "Oncogene-Targeted Immune Therapy in FLC", this panel featured two clinician-researchers pioneering clinical trials of therapeutic vaccines aimed at FLC's fusion protein, and a patient currently enrolled in one of those trials.

Specifically, the panelists included:
- Juliane Walz, MD – University of Tübingen,
- Mark Yarchoan, MD – Johns Hopkins University,
- Molly Hones – FLC patient and clinical trial participant, and
- Mark Furth, PhD – Chief Science Officer, FCF (moderator).

You can watch the full discussion on FCF's YouTube channel using the link below:
https://youtu.be/zFAMigwsb-0
https://fibrofoundation.org/panel-discussion-at-2025-fibrolamellar-scientific-summit/

06/18/2025

FCF is pleased to announce that it has awarded a grant to Aleksander Skardal, PhD, of Ohio State University to develop new models of fibrolamellar carcinoma (FLC). A major challenge to researching FLC is the scarcity of lab models that accurately mimic how this cancer behaves in the human body. To address this, the team plans to build three-dimensional mini-tumors that can be used for testing drugs and studying cancer behavior. The approach developed by the Skardal lab has already been used in several other cancers, including some rare ones.

In this effort, the team plans to use FLC tumor samples obtained from the FCF Biobank to:
- grow 3D mini-tumors
- study the cells within these models — including tumor, immune, and support cells
- if necessary, add specific liver-related immune cells to make the models behave more like real FLC in the body.

If successful, this effort will provide a mechanism to study FLC in a more realistic way and to test new treatments — especially immune-based therapies — faster and more reliably.
https://fibrofoundation.org/grant-awarded-to-ohio-state-university/

06/13/2025

The FCF is pleased to announce the funding of a study led by Dr. Sean Ronnekleiv-Kelly of the University of Wisconsin-Madison. This research will explore a key protein, SLC16A14, that is involved in the growth and survival of fibrolamellar cancer (FLC) cells using a new mouse model that the team has developed. Evidence suggests that SLC16A14 is strongly connected to FLC's DNAJB1-PRKACA gene fusion and may be an important link driving the metabolic changes of the cancer cells. This study plans to investigate this possibility with the goal to develop a therapy against SLC16A14 that can eliminate cancer cells that contain the DNAJB1-PRKACA protein (DP) while sparing normal cells.

According to Dr. Ronnekleiv-Kelly, “This funding from the Fibrolamellar Cancer Foundation will be indispensable for allowing us to gain a better understanding of how DNAJB1-PRKACA transforms normal calls into cancer cells. Furthermore, we hope to make significant progress in determining the therapeutic potential of a very promising target (SLC16A14).”
https://fibrofoundation.org/fcf-funds-effort-at-university-of-wisconsin/

06/06/2025

A new clinical trial for fibrolamellar patients is now enrolling patients! The study, "Testing the Addition of an Anti-cancer Drug, DT2216, to the Usual Chemotherapy Treatment for Relapsed or Refractory Solid Tumors and Fibrolamellar Carcinoma" will test the safety, side effects and best dose of DT2216 in combination with the chemotherapy agent, irinotecan. DT2216 is an experimental drug developed by Dialectic Therapeutics, Inc. that is designed to overcome a major mechanism through which cancer cells may resist chemotherapy.

The clinical trial is led by pediatric oncologists Dr. Michael Ortiz (Memorial Sloan Kettering Cancer Center) and Dr. Allison O’Neill (Dana-Farber Cancer Institute). Patients will be able to enroll at multiple hospitals affiliated with the Children’s Oncology Group's Pediatric Early Phase-Clinical Trial Network (PEP-CTN).

The initial Phase 1 portion of this study will enroll in patients between ages 1 and 21 years with solid tumors that have recurred or failed to respond to other treatments. Its main goal will be to determine a recommended Phase 2 dose of DT2216, administered in combination with a standard dose of irinotecan. The Phase 2 portion of the study will be carried out exclusively in FLC patients up to 39 years old. DT2216 will be administered at the recommended dose established in Phase 1, together with irinotecan at its standard dose. The main goal of the Phase 2 portion will be to define anti-tumor activity of the DT2216/irinotecan combination in FLC patients.

FCF is supporting this effort by funding the manufacturing of the DT2216 necessary to conduct the trial. According to John D. Harkey, Jr., Dialectic Therapeutic's Executive Chairman & Co-founder, “We are excited about our partnership with the Fibrolamellar Cancer Foundation which will provide Dialectic the financial backing necessary to allow the clinical testing of this drug in fibrolamellar patients."

FLC patients who are interested in participating should consult their medical oncologist as soon as possible, since enrollment will be limited. More information about the trial is available at clinicaltrials.gov/study/NCT06620302.
https://fibrofoundation.org/dt2216-clinical-trial-now-open/

06/04/2025

From June 9 - 11, FCF is hosting its sixth Fibrolamellar Scientific Summit. The goal of this meeting is to bring together the fibrolamellar research community to share research knowledge and to collaborate in the search for a cure.

To encourage the sharing of unpublished information, the scientific discussions in the main session will be open only to researchers who have signed a multi-party non-disclosure agreement. However, the event’s introductory keynote discussion (June 9, 2025 from 8 – 9 pm ET) will be open for virtual participation by the entire fibrolamellar community – patients, caregivers, clinicians and researchers.

This year's keynote will be a panel discussion exploring the great progress being made in targeted immunotherapy against FLC. It will feature:
- Mark Furth, PhD, FCF (moderator)
- Juliane Walz, MD, University of Tübingen
- Mark Yarchoan, MD, Johns Hopkins University
- Molly Hones, FLC patient and clinical trial participant.

To attend the Zoom webinar, please click on the following link to register: https://us02web.zoom.us/webinar/register/WN_ZR9l28ANSqOZUP2OHTw9Xw #/registration
https://fibrofoundation.org/keynote-discussion-at-fibrolamellar-scientific-summit/

05/31/2025

Congratulations to Marina Baretti, MD of Johns Hopkins University! On May 30, she was recognized with a Career Development Award (CDA) at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. This highly prestigious award provides three years of research funding to clinical investigators to establish an independent clinical cancer research program.

These awards are funded by Conquer Cancer®, the ASCO Foundation. To be considered for this Career Development Award, an investigator must be conducting clinical or translational research involving patients. U.S. and international applicants active in all areas of cancer are considered. While Dr. Baretti has been involved in treating and researching a range of liver, pancreatic and biliary tree cancers, she was recognized for this award largely due to her activities in fibrolamellar - in particular her leadership in both the peptide vaccine and glutamine antagonist clinical trials now underway at Johns Hopkins.

FCF is thrilled that the impact of Dr. Baretti's clinical research in fibrolamellar is being broadly recognized by the medical community!
https://fibrofoundation.org/dr-baretti-wins-prestigious-asco-award/

05/30/2025

SAVE THE DATE!
FCF 2025 Patient & Family Gathering
Sept 12 - Sept 14, 2025
Lake Mansfield Trout Club
Stowe, VT

05/27/2025

The Fibrolamellar Cancer Foundation is pleased to announce that it has awarded an exploratory research grant to Dr. Hugo Jimenez at Wayne State University to investigate the effectiveness of using low energy electromagnetic fields to treat fibrolamellar carcinoma (FLC). In this study, Dr. Jimenez and his team will conduct initial feasibility testing with a device that delivers low energy amplitude modulated 27.12 MHz radiofrequency electromagnetic fields (AM RF EMF) to see if they can disrupt tumor growth in cell models of FLC.

The device, the TheraBionic P1, is a portable handheld device that generates low levels of electromagnetic fields. These fields are amplitude-modulated at frequencies specific to tumor types. Already, this Therabionic P1 device has received FDA approval for the treatment of hepatocellular carcinoma (HCC), based on clinical trials that showed a 34% increase in survival rates without any significant adverse events. During treatment, a spoon-shaped antenna is placed on a patient’s tongue to deliver low levels of electromagnetic fields throughout the patient’s body, blocking the growth of tumor cells while leaving healthy tissue unharmed.

Preliminary work has shown that the growth of a "FLC-like" cell line can be inhibited by the electromagnetic fields that are used currently to treat HCC patients. However, much work remains to test the approach in actual FLC cell lines and model systems. This study will determine the feasibility of continuing the investigation of the use of this device as a potential treatment option for FLC patients.
https://fibrofoundation.org/grant-awarded-to-wayne-state-university/

05/21/2025

FCF is pleased to announce that it is funding an extension of a clinical trial at the Johns Hopkins Kimmel Cancer Center in Baltimore, MD that tests the ability of a FLC-specific vaccine to produce an immune response against fibrolamellar carcinoma. With this new funding, the trial will be able to enroll 16 additional patients.

This groundbreaking clinical study, led by Mark Yarchoan, MD and Marina Baretti, MD, began enrolling subjects with metastatic FLC in April 2020. In it, participants are given an experimental vaccine containing a peptide (a small segment of a protein) that corresponds to the junction region of the fusion protein that causes FLC. They also simultaneously receive two FDA-approved drugs, Opdivo (nivolumab) and Yervoy (ipilimumab), that are designed to enhance a patient's immune response against tumors by overcoming “checkpoint” systems that can limit their immune system’s ability to fight a cancer.

Interim results of the study have been reported at several international conferences and are currently being reviewed for publication in a scientific journal. So far, roughly three-fourths of participants in the trial developed a strong immune response to the peptide vaccine. Nearly all of these responding patients also showed some clinical benefit - at a minimum their tumors stopped expanding. Importantly, in about one-third of those patients, the tumors shrank dramatically. In some cases, the participants now have “clean” scans, and several are classified as being in complete remission. While the total number of patients who enrolled in the study is still relatively small, the results are the most positive ever seen in a clinical trial focused on FLC.

To participate in this study expansion, patients must:
- Be age 12 or older
- Have measurable disease and the confirmed presence of the DNAJB1-PRKACA (DP) fusion transcript
- Meet minimum health and performance criteria
- Not have had prior treatment with immune checkpoint inhibitors.

For more information about the trial, please visit clinicaltrials.gov (NCT04248569).
https://fibrofoundation.org/clinical-trial-at-johns-hopkins-extended/

05/14/2025

In this video ( https://youtu.be/wz5qenw0sak ) , Dr. Juliane Walz discusses a new clinical trial that is now open to patient enrollment at The University Hospital Tübingen in Tübingen, Germany. This FusionVAC22_02 clinical trial is designed to test the safety and efficacy of a peptide vaccine to delay or prevent the recurrence of fibrolamellar carcinoma (FLC). The peptide vaccine, made from a short sequence of amino acids, is designed to induce a patient's immune system to recognize the fusion protein that causes FLC. In the trial, the vaccine will be administered alone, without additional immunotherapy or any other systemic agent.

The study will be open to patients who are judged free of cancer by radiological scans after surgery or other treatments. Patients must have a confirmed diagnosis of FLC or other cancer with the DNAJB1-PRKACA fusion transcript. Initially, the trial will only be open to patients 18 years old or older. After 6 adult patients have been enrolled, the study will open to patients age 12 and older.

FCF is providing the funding necessary to support this trial. For more information about this new study, please visit clinicaltrials.gov (NCT06789198) or the trial's information page at The University Hospital ( https://www.medizin.uni-tuebingen.de/en-de/fusionvac-krebsimpfstoffstudie ).

https://fibrofoundation.org/new-immunotherapy-trial.../