The Fibrolamellar Cancer Foundation

Cycle 1

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Fibrolamellar carcinoma (FLC), also known as fibrolamellar hepatocellular carcinoma, is a rare liver cancer that primarily occurs in adolescents and young adults who have no history of liver disease. In the early stages of the disease, affected patients often have no symptoms, so by the time the cancer is found, it may have already spread beyond the liver. When symptoms develop, they may include abdominal pain, weight loss, and malaise.

Last updated 04/30/2026

Clinical

Disease Class

Abdominal surgical diseases

Cancer

Gastroenterological diseases

Hepatic diseases

Body Systems

Digestive

Metabolic

Organs

Bones

Brain

Liver

Lungs

Lymph fluid, nodes, ducts, vessels

Known Genetic Link

No, the disease is not known to have a genetic basis

Causative Genes

None specified / unknown

Contributory Genes

None specified / unknown

Type of Inheritance

Not specified / unknown

Newborn Screening

Disease Mechanism(s)

Gene fusion

Age of Onset

Adolescence (12-17)

Adulthood (age 18-64)

Middle childhood (6-11)

Average Age at Diagnosis

Adolescence (12-17)

Adulthood (age 18-64)

Life Expectancy

Adolescence (12-17)

Adulthood (age 18-64)

Affected Sex(es)

Female

Intersex

Male

National Prevalence

101-1000

Global Prevalence

1001-10000

National Incidence

Less than 10

Global Incidence

Less than 10

Symptoms / Phenotypes

abdominal swelling

bruising susceptibility

gynecomastia

hemorrhage/bleeding

jaundice

malaise

mass / neoplasm

pain, abdominal

vomiting / nausea

weight loss

Biomarkers

Diagnostic

· DNAJB1-PRKACA

Monitoring

· procalcitonin

Prognostic

· procalcitonin

Existing Therapies

None

Therapies in Development

Antisense oligonucleotide (ASO) therapy

Cellular therapies (stem cell transplants, CAR-T therapies, etc.)

· TCR-T to gene fusion (several HLA types)

Immunotherapy

· Peptide vaccine

RNA interference (RNAi) therapy (siRNA, etc.)

· to DNAJB1-PRKACA

Repurposed drug

Small molecule therapy (novel small molecule drugs)

· several identified by FCF-driven DEL screen

Surgical & interventional

Therapeutic Development Stages

IND/CTA submitted but not approved

In clinical trials (Phase I, II, III, or IV)

In preclinical development

In research/exploratory phase

Therapeutic Development Role

Data analysis

Data sharing

Focus group participation or coordination

Funding

Meetings with regulators (e.g., FDA listening sessions, PFDD meetings)

Other

· Serving as PI for several efforts

Recruitment and outreach to patients

Recruitment and outreach to trial sites / physicians

Results dissemination (including publications)

Sample provision

Study material design and/or review (not protocol) — includes patient-facing materials such as informed consent

Study protocol design and/or review (includes selection of outcome measures)

Organizational & Research

Cell Lines

Cancer/tumor cells

Cell Lines, Institution

Dana-Farber Cancer Institute

Massachusetts General Hospital

Cell Lines, Involvement

Consulted

Funded

Cell Lines, share

All our cell lines are freely available

Disease Model

Mouse

Organoids

Other

Disease Model, Involvement

Consulted

Funded

Disease Model, share

All our disease models are freely available

Organizational Challenges

Coordination and understanding of regulatory requirements to proceed to registration

Clinical Trial Role

Data analysis

Data sharing

Focus group

Funding

Meeting with regulators

Outcome measures, development

Recruitment and outreach, patients

Recruitment and outreach, trial sites/physicians

Results dissemination, publication

Study material design, review (not protocol)

Study protocol design, review

Travel coordination

Clinical Trial Types

Phase 1

Phase 2

Biobank, Institution

Massachusetts General Hospital

Biobank, Involvement

Designed

Funded

Own

Center of Excellence, Institution

None

Registry

Yes, we have collaborated on a registry

Data Collected, Registry

Clinical data

Longitudinal natural history data

Medication usage

Patient contact info

Patient-reported data

Data Entered by, Registry

Patients

Platform, Registry

REDCap

Natural History Study

Yes, we have collaborated on a natural history study

Data Collected, Natural History Study

Electronic health records/electronic medical records

Medication usage

Patient-reported outcomes

Prospective data

Retrospective data

Platform, Natural History Study

REDCap

xCures

FDA Patient Listening Session

Yes

FDA Patient-Focused Drug Development (PFDD) Program

ICD Codes

Yes, we have an ICD-11 code specific to our exact disease

Diagnostic Guidelines

Yes, we have guidance available on our website

In the process of creating formal diagnostic guidelines for publication in a peer-reviewed journal

Science Advisory Board Policies

Yes, willing to share SAB policies

Research Network Policies

Has CRN and willing to share policies

Patient Priority Survey

Research Roadmap

Yes we have a Research Roadmap, and will share policies

None