The Fibrolamellar Cancer Foundation

A FEW MORE SPOTS ARE AVAILABLE......

Registration is now OPEN for the 2024 Patient & Family Gathering in Stowe, Vermont
Sept 13 - Sept 15, 2024
SPACE IS LIMITED
Click on the link below to register.

https://fibrofoundation.org/events/2024-patient-family-gathering/

SAVE THE DATE: FCF PATIENT & FAMILY GATHERING

Virtual registration is still open for FCF's second Fibrolamellar Patient & Research Summit to be held on June 9-10 in Greenwich, Connecticut. This summit is meant to be a collaborative working session in which fibrolamellar patients, caregivers, clinicians and researchers discuss the current state of FLC research, share perspectives, and identify improvement opportunities for the community.

Please visit https://fibrofoundation.org/events/fibrolamellar-patient-research-summit-2024/ for more information or to register.

On-line registration open for next week's FLC Patient & Research Summit Virtual registration is still open for FCF's second Fibrolamellar Patient & Research Summit to be held on Sunday, June 9 through Monday, June 10 in Greenwich, Connecticut. This summit is meant to be a collaborative working session in which fibrolamellar patients, caregivers, clinicians and researche...

This spring, a six-institution team led by Paul Thomas (St. Jude Children’s Research Hospital) and Scott Strome (University of Tennessee) published an article in Cell Reports Medicine about the isolation of T-cell receptors specific for the fusion protein driver of fibrolamellar carcinoma (FLC). The post "Perspectives on Immunotherapy" now listed on FCF's web site discusses some of the implications of that study and other recent research efforts on future FLC immunotherapy treatments.

Please visit https://fibrofoundation.org/perspectives-on-flc-immunotherapy/ to learn more.

Perspectives on FLC immunotherapy Below is a discussion of the implications of recent research efforts on future FLC immunotherapy treatments as summarized by Mark Furth ... Read more

FCF is pleased to announce that it has awarded a grant to Sean Ronnekleiv-Kelly, MD of the University of Wisconsin-Madison School of Medicine to determine if targeting CDK7 could be a useful treatment approach for FLC.

A group of proteins called cyclin-dependent kinases (CDKs) help regulate cell division and other fundamental cellular processes. Previously, Dr. Ronnekleiv-Kelly observed that a particular CDK called CDK7 is present at significantly higher levels in FLC cells than in normal liver cells. CDK7 has already been associated with rapid progression and poor prognosis in several cancers and has become a potential anticancer drug target.

In this study, Dr. Ronnekleiv-Kelly will determine whether the inhibition of CDK7 using an investigational new drug could be an effective therapeutic strategy for FLC. If the results of this study are promising, they could set the stage for a clinical trial of CDK7 inhibition, alone or in combination with other drugs, in FLC patients.

FCF funds grant to University of Wisconsin FCF is pleased to announce that it has awarded a grant to Sean Ronnekleiv-Kelly, MD of the University of Wisconsin-Madison School of Medicine to determine if targeting CDK7 could be a useful treatment approach for FLC.A group of proteins called cyclin-dependent kinases (CDKs) help regulate cell divi...

Patients living with a rare disease have to manage many unique challenges. Included in those is what happens to college and career plans during treatment. That’s why the EveryLife Foundation for Rare Diseases established the Scholarship – a program that provides support for the educational pursuits of rare disease patients. This year, The EveryLife Foundation for Rare Diseases plans to provide scholarship awards of $5,000 to up to 88 patients.

This scholarship program is open to anyone age 17 or older, who is a resident of the United States, and who has been diagnosed as having a rare disease, regardless of treatment status. Applicants must plan to enroll full-time or part-time in undergraduate or graduate study at an accredited 2 or 4-year college, university, or technical/trade school during the fall 2024 semester.

Applications must be received by April 22, 2024 at 2:00 PM ET. Please visit https://everylifefoundation.org/rare-scholarship/ for more information.

Scholarships for rare disease patients Scholarships for rare disease patients April 9, 2024 Patients living with a rare disease have to manage many unique challenges. Included in those is what happens to college and career plans during treatment. That’s why the EveryLife Foundation for Rare Diseases established the Scholarship ...

Welcome to week! This annual awareness effort is an opportunity to focus on the unique challenges facing teen and young adult cancer patients and survivors.

This week many groups that support AYAs will be sharing helpful information, resources, and programming. Follow the hashtags and to learn more.

Every year, the first week of April is Adolescent and Young Adult (AYA) Cancer Awareness Week. This annual awareness effort is an opportunity to focus on the unique challenges facing teen and young adult cancer patients and survivors.

AYAs with cancer are contending with many challenges. Things like starting a career, finishing school, or finding love are tough for everyone. But combined with a cancer diagnosis, they can seem impossible. This week aims to raise awareness about how hard it is for teens and young adults to deal with cancer while simultaneously juggling their busy lives.

Search for the hashtags and to learn more!

This week is AYA Cancer Awareness Week Every year, the first week of April is Adolescent and Young Adult (AYA) Cancer Awareness Week. This annual awareness effort is an opportunity to focus on the unique challenges facing teen and young adult cancer patients and survivors.According to the National Cancer Institute, nearly 86,000 individu...

The U.S. Department of Defense has asked FCF to help to recruit community members to serve as consumer reviewers for the Peer Reviewed Cancer Research Program’s FY24 peer review panels. Participation in this program is a great way way to influence research into FLC and other cancers!

The Peer Reviewed Cancer Research Program (PRCRP) has been funded by Congress as part of the Congressionally Directed Medical Research Programs (CDMRP) to support innovative, high-impact cancer research. Since 2009. the PRCRP has funded approximately 1,021 awards. Fibrolamellar research has received approximately $2.65 million in awards from the CDMRP since 2016.

Consumer reviewers work with prominent scientists to provide input on how the DOD research funds should be allocated. They act as subject matter experts on their disease, bringing their unique experiences and perspectives to the review panel. This input is critically important to ensure that funded research is relevant and has the potential to make a substantial impact on patients.

Please visit FCF's web site to learn more about the opportunity and application process.

Help review DOD research grants The U.S. Department of Defense has asked FCF to help to recruit community members to serve as consumer reviewers for the Peer Reviewed Cancer Research Program ... Read more

FCF is please to announce that in-person and virtual registration is now open for FCF's second Fibrolamellar Patient & Research Summit to be held on June 9-10, 2024 in Greenwich, Connecticut. This summit will be a collaborative working session involving fibrolamellar patients, caregivers, clinicians and researchers. The key goals of this event will be to bring together the fibrolamellar community to help build mutual understanding among stakeholders, communicate and answer questions about the overall FLC research agenda, and identify issues and potential opportunities the community should address.

The meeting will begin at 5:00 pm on Sunday, June 9 with a welcome reception, dinner and keynote talks. Monday, June 10 will be a day-long session running from 8:30 am until 4:30 pm. It will include a series of interactive presentations, panel discussions and round table sessions on important topics.

Please visit FCF's web site for more information or to register.

Registration open for Fibrolamellar Patient & Research Summit FCF is please to announce that in-person and virtual registration is now open for FCF's second Fibrolamellar Patient & Research Summit to be held on June 9-10, 2024 in Greenwich, Connecticut. This summit will be a collaborative working session involving fibrolamellar patients, caregivers, clinicians...

Rare diseases, also known as orphan diseases, affect a small percentage of the population. In the US, diseases that affect fewer than 200,000 people per year are considered rare. While each rare disease only affects a small percentage of the population, collectively, "rare" is not rare. With over 7,000 rare diseases, nearly 1 in 10 Americans are currently living with a rare disease.

Rare Disease Day takes place on the last day of February each year. Rare Disease Day is a global initiative to raise awareness about the impact of rare diseases on patients’ lives and to emphasize the need for research. Since its establishment in 2008 by EURORDIS (Rare Diseases Europe), the annual event highlights rare disease advocacy work on the local, national and international levels. The National Organization for Rare Disorders (NORD) is the official US sponsor of Rare Disease Day.

We are all touched by rare diseases in many different ways. On this Rare Disease Day, we encourage everyone to think about how they can support the effort. The options are many - donating to organizations that are focused on making research breakthroughs, learning about rare diseases and how they affect those living with them, offering hope to patients, volunteering, and more. Your participation can make a difference!

For information about what’s happening on Rare Disease Day around the world, please visit rarediseaseday.org.

February 29 is Rare Disease Day Rare diseases, also known as orphan diseases, affect a small percentage of the population. In the US, diseases that affect fewer than 200,000 people per year ar

In the video below, Mark Yarchoan, MD and Marina Baretti, MD of Johns Hopkins University discuss the launch of a new clinical trial specifically for fibrolamellar patients that leverages the recently-identified "glutamine addiction" of FLC tumors.

The trial, named "DRP-104 (Glutamine Antagonist) in Combination With Durvalumab in Patients With Advanced Stage Fibrolamellar Carcinoma" is now open for patient enrollment at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins in Baltimore, Maryland. The study plans to test the safety and effectiveness the experimental prodrug DRP-104 in combination with an immune checkpoint inhibitor. DRP-104 is an inactive compound that is converted into a broad-acting glutamine antagonist within tumors, while mostly remaining inactivated in other healthy tissues.

In the video (https://youtu.be/FR9RkMSgofo), the team discusses the pre-clinical work that provided the scientific rationale for the effort, as well as the design and enrollment criteria for the trial.

According to Mark Yarchoan, "One of the things I am really proud of is how quickly this trial has moved from inception to opening. ... It wouldn't be possible without the enormous support of multiple different funding sources."

The Fibrolamellar Cancer Foundation has provided the funding to support the launch of this trial and its protocol design, and helped secure drug access for the trial through a partnership with Dracen Pharmaceuticals, the developer of DRP-104. Additional funding for the trial is also being provided by the Cancer Research Institute and the National Cancer Institute.

For more information about this new study, please visit clinicaltrials.gov (NCT06027086) or contact the clinical trials team at Johns Hopkins at [email protected].

Investigators discuss launch of glutamine antagonist trial In the attached video, Mark Yarchoan, MD and Marina Baretti, MD of Johns Hopkins University discuss the launch of a new clinical trial specifically for fibrolam

FCF is proud to be the recipient of the Ryan Couling Memorial Hockey Tournament Fundraiser.

The RCMT donated $8.062.50 to FCF - which will be matched by Chuck and Marna Davis - Co- Chairs of FCF.

We are very grateful to the Couling Family!

The inaugural hockey tournament in January 2023 started with four teams, and this year, they hosted eight teams. They aim to grow the Ryan Couling Memorial Tournament into one of Canada's premier U18AA tournaments.

To learn more about Ryan, and RCMT, please visit their website:
https://ryancoulingmemorialtournament.com/page/about-ryan-couling

Keep On Tuckin'

Over the last few years, a team of investigators at Johns Hopkins University conducted an innovative clinical trial designed to test the potential to induce an immune response against fibrolamellar carcinoma with a FLC-specific therapeutic vaccine.

In a video on YouTube and the FCF website (https://youtu.be/f2eIIzilMBc), Mark Yarchoan, MD and Marina Baretti, MD discuss the results and next steps for that clinical trial - DNAJB1-PRKACA Fusion Kinase Peptide Vaccine Combined with Nivolumab and Ipilimumab for Patients with FLC. A major question for the study was whether the treatment was safe. While some patients suffered adverse events, they were within the range expected for treatment with two checkpoint inhibitors and could be managed. A second question was whether patients would mount an immune response against the peptide vaccine. Out of 12 patients evaluated, 75% (9) generated robust responses to the vaccine – that is they had generated substantial numbers of T cells that showed specific immune reactions to the peptide.

Finally, what clinical benefits were observed? Importantly, 25% (3) of the patients achieved significant tumor shrinkage/strong partial responses to the treatment and 75% of the patients achieved some period of tumor stability.

Achieving three strong, long-lasting clinical responses among 12 subjects is unprecedented in any controlled clinical trial in FLC. A key remaining question is why 75% of the study participants had an immune response to the vaccine, but only 25% experienced strong tumor shrinkage. A goal of the Johns Hopkins team is to answer that question and to identify conditions that can help a higher percentage of patients receive a similar therapeutic benefit.
https://fibrofoundation.org/results-of-peptide-vaccine-clinical-trial-discussed/

FCF is pleased to announce the establishment of a research collaboration with X-Chem, a global leader in DNA-encoded library (DEL) technology. Through the effort, X-Chem will leverage its powerful DEL platform to support drug discovery efforts against fibrolamellar's difficult-to-drug fusion protein. The hope is that this powerful screening technology can be used to identify potential small molecule "hits" that could eventually lead to the identification of drug candidates for FLC.

https://fibrofoundation.org/?p=20537

We have a few more FCF 2024 Calendars available for purchase!
Click on the link below to order your calendar.

Reserve your fibrolamellar calendar today Donate just $20 to receive your calendar Between now and Friday, December 1st, anyone who donates $20 or more and completes the following form will get a fib

As we start a brand new year, the Fibrolamellar Cancer Foundation extends warm wishes to the entire fibrolamellar community.

May this year bring:
- Strength to those bravely facing the challenges of FLC
- Unity among patients, caregivers, clinicians, researchers and advocates, as we support those impacted by this horrible disease
- Progress in research, and a renewed sense of hope for the discovery of effective treatments
- Increased awareness and resources for all rare diseases, including FLC.
FCF is committed to work collaboratively to build a brighter future for those touched by this disease. Please join us in the effort to reduce the fear and uncertainty associated with a fibrolamellar diagnosis.

Wishing you peace, comfort, and serenity for the New Year!
The FCF team
https://fibrofoundation.org/new-year-wishes/

By supporting FCF, you can play a crucial role in funding groundbreaking research and clinical trials attempting to find improved treatment options for fibrolamellar cancer patients.

Together, we can make a difference in the fight against this rare cancer. Your donations can “fund the fight” and move us closer to a future free from fibrolamellar carcinoma. As always, 100% of donations to FCF directly fund research efforts.
https://fibrofoundation.org/giving-tuesday/

Giving Tuesday 2023 Each year, the Tuesday after Thanksgiving celebrates giving as a way to transform the world. Giving Tuesday was created in 2012 as a day that encourages people

REMINDER:

Deadline to submit photos for the FCF Calendar is Monday, November 20 and to purchase a calendar, orders must be placed by December 1st.

Thanks to the efforts of FCF's Patient & Caregiver Advisory Board, we will be producing fibrolamellar calendars for 2024! The calendars, featuring fibrolamellar fighters past and present, are currently being designed. Please visit www.fibrofoundation.org to learn more, to provide pictures to be featured in the calendar, or to reserve your copy.
https://fibrofoundation.org/join.../fibrolamellar-calendar/

Get a fibrolamellar calendar The 2024 calendar is currently being designed, but here's a copy of the 2023 version (click to view) We are excited to announce that fibrolamellar calendars

FCF is please to announce that it has funded a new study directed by Roshni Dasgupta, MD of the Cincinnati Children’s Hospital Medical Center (CCHMC). Dr. Dasgupta is a leader in surgical oncology and the chair of the Pediatric Surgical Oncology Research Collaborative (PSORC). PSORC includes 41 member institutions and has a mission “to improve surgical outcomes for children, adolescents and young adults with cancer through multi-institutional collaborative investigation.”

She and the other co-investigators - Soo-Jin Cho, MD, PhD of the University of California, San Francisco (UCSF); Dolores López-Terrada, MD, PhD of the Texas Children’s Hospital; and Kimberly Riehle, MD of the University of Washington - will lead a project under the PSORC umbrella to address questions about the true incidence, accuracy of diagnosis, and optimal treatments for rare pediatric liver tumors, including FLC.

The study - "How fibrolamellar carcinoma differs from hepatocellular carcinoma in children – Creation of a multi-institutional database including central pathologic review" - will include a centralized pathology review and molecular analysis of 262 tumor samples in the PSORC collection to confirm the actual number of FLC cases versus other types of liver cancers. A longer-term goal, beyond accurate diagnosis of FLC, will be to determine whether molecular analysis can offer individualized information for FLC patients about disease prognosis and optimal treatment regimens.
https://fibrofoundation.org/?p=17577

Another AMAZING fundraiser hosted by The Alexander Family to benefit FCF.

Pig Gig 8 is THIS SUNDAY, October 22nd at the Italian American Hall in Scotch Plains, New Jersey.

Pig Gig 8 is all about raising money for the The Fibrolamellar Cancer Foundation for research and all profits go directly to the foundation. It is a full day of food, fun, music and comedy planned with The Weeklings, comedian Mike Marino, The Lonesome Pines, Blue Americana, Anthony Tamburro and Louis Bevere and Glenn Alexander & Shadowland!!! With special guest star Southside Johnny!

Tickets are only $70 and are available now at Ticketbud.com - https://tinyurl.com/4xwxh4bc. Your ticket includes a full day of music and entertainment, plus all you can eat food and drink. Wow, that's a lot of bang for your buck! And it's all for a great cause, so c'mon out and join us - Here's To Jay!

Thanks to the efforts of FCF's Patient & Caregiver Advisory Board, we will be producing fibrolamellar calendars for 2024! The calendars, featuring fibrolamellar fighters past and present, are currently being designed. Please visit www.fibrofoundation.org to learn more, to provide pictures to be featured in the calendar, or to reserve your copy.
https://fibrofoundation.org/join-the-fight/fibrolamellar-calendar/

Get a fibrolamellar calendar The 2024 calendar is currently being designed, but here's a copy of the 2023 version (click to view) We are excited to announce that fibrolamellar calendars

This week, a new clinical trial of a peptide vaccine treated its first patient in Tübingen, Germany. The trial, named "FusionVAC22_01: Fusion Transcript-based Peptide Vaccine Combined With Immune Checkpoint Inhibition" is expected to enroll 20 patients with fibrolamellar carcinoma or other cancers carrying the DNAJB1-PRKACA fusion transcript.

This phase I study will examine the viability of using a peptide vaccine to provide a patient’s immune system the ability to identify and destroy FLC tumor cells. It will evaluate the safety and effectiveness of treating patients with a peptide vaccine (Fusion-VAC-XS15) in combination with the immune checkpoint inhibitor atezolizumab (Tecentriq). The peptide vaccine, made from a fragment of FLC’s DNAJB1-PRKACA fusion protein, is being produced at the University Hospital Tübingen.

Enrollment in this trial is open to patients 18 years old and older whose disease has the confirmed presence of the DNAJB1-PRKACA fusion.

This clinical trial is being conducted at the University Hospitals of Freiburg, Heidelberg, Tübingen and Ulm in Baden-Württemberg, Germany. The Tübingen location is currently open and enrolling patients. The other locations will open soon.
https://fibrofoundation.org/new-clinical-trial-open-in-germany/

Thanks to the efforts of FCF's Patient & Caregiver Advisory Board, we will be producing fibrolamellar calendars for 2024! The calendars, featuring fibrolamellar fighters past and present, are currently being designed. Please visit www.fibrofoundation.org to learn more, to provide pictures to be featured in the calendar, or to reserve your copy.

2024 fibrolamellar calendars Thanks to the efforts of FCF's Patient & Caregiver Advisory Board, we will be producing fibrolamellar calendars in 2024! Read more

FCF is pleased to announce that it has awarded a grant for additional studies to be conducted by John Scott, PhD of the University of Washington in Seattle. As the Chair of the Department of Pharmacology, Dr. Scott has been a key leader of a multi-disciplinary team working on FLC at the University of Washington and the Fred Hutchinson Cancer Center in Seattle.

This current grant is a renewal of a grant initially awarded by FCF in 2019.

A major goal of the current study is to understand precisely how the altered anchoring of FLC's fusion protein and its deregulated activity contribute to the cancerous transformation of liver cells. The team believes that such insight could point to novel ways to attack FLC with specific drugs or combination therapies.
https://fibrofoundation.org/?p=17576

A heartfelt thank you goes out to Stone Point Capital, as well as the sponsors and participants in the 2023 Stone Point Capital Charity Golf Tournament. Since 2009 Stone Point Capital has dedicated the proceeds from its annual charity event to FCF. This years event, held in Connecticut on October 2nd, raised a record amount of funding for the Foundation. Gross donations from sponsors and participants totaled $1.5 million. We thank everyone for their generosity!

As always, 100% of the contributed funds will be directed towards vital FLC research initiatives and grants. Every dollar raised brings us one step closer to finding a cure and more effective treatments for those affected by this devastating disease.

Thank you to Stone Point Capital! A heartfelt thank you goes out to Stone Point Capital, as well as the sponsors and participants in the 2023 Stone Point Capital Charity Golf Tournament. Since 2

October is Liver Cancer Awareness Month, a chance to raise vital awareness and funds to improve the detection and treatment of these diseases, as well as show support for those who have heard the terrifying words - "you have liver cancer".

As we observe Liver Cancer Awareness Month, the Fibrolamellar Cancer Foundation stands with other advocacy organizations in our commitment to improving the lives of all those affected by liver cancer. We thank all the patients, caregivers, advocates, clinicians, and researchers who dedicate their efforts towards finding effective treatments and cures for these terrible diseases.

https://fibrofoundation.org/?p=15967

September 30 is Rare Cancer Day. Spearheaded by the National Organization of Rare Disorders' (NORD) Rare Cancer Coalition, Rare Cancer Day is observed on September 30 each year to highlight the challenges facing rare cancer patients and the need for greater research funding.

The Fibrolamellar Cancer Foundation and other organizations use this day to shine a spotlight on rare cancers and unify all voices in hope of a cure. The hope is that increased media and public attention on rare cancers will motivate research funding and therefore accelerate the development of effective treatments for all rare cancers, including fibrolamellar carcinoma.
https://fibrofoundation.org/?p=15966

Support Current Research by donating tumor tissue:

Fibrolamellar tumor tissue is critical to help cancer researchers learn what drives this disease and how to treat it. Studies often need large numbers of samples from different patients to adequately answer their research questions.

With a few easy steps, you can support current research by donating tumor tissue to the Fibrolamellar Cancer Foundation (FCF) Biobank.

Donate Tumor Tissue Your Surgery or Fluid Drain Can Advance FLC Research Fibrolamellar tumor tissue is critical to help cancer researchers learn what drives this disease and how to treat it. Studies often need large numbers of samples from different patients to adequately answer their research questions. With a few eas...

Another FCF Patient & Family Gathering in the books!
What a wonderful weekend spending time with old friends and making new friends.
Our time at Lake Mansfield Trout Club is magical…so much love, so much hope, so much fun….
Thank you to all those that were able to attend….great to see you all.
Until next year….Keep On Tuckin’
💚💚💚💚💚❤️❤️❤️❤️❤️