Timeframe: 2025 – 2027
Goal: Assess potential to induce immune response with a FLC therapeutic vaccine
Principal Investigator: Mark Yarchoan, MD and Marina Baretti, MD
Study overview: This grant allows Dr. Mark Yarchoan and Dr. Marina Baretti of Johns Hopkins University to keep JHU’s successful “peptide vaccine” clinical trial open to enrollment by fibrolamellar patients.Â
In the study, patients receive a vaccine created to train the immune system to recognize and attack cells carrying the abnormal fusion protein that causes FLC. Because nearly all FLC tumors share this same fusion protein, the same “off-the-shelf” vaccine made from a small peptide representing the junction of the fused proteins can be used for all patients. During the clinical trial, the vaccine is administered alongside two immune checkpoint inhibitors (ICIs) already approved for use in other cancers (nivolumab and ipilimumab).Â
Research published in Nature Medicine in November 2025 summarized the results of this clinical trial for the first 12 evaluable participants:
- The vaccine appears safe.
- 75% of participating patients mounted the desired immune response; all of these responders achieved some disease control (at least several months without tumor growth).
- One-third of responders (3 patients) experienced dramatic, long-lasting tumor shrinkage—and remain cancer-free.
An ultimate objective of the peptide vaccine study is to obtain FDA approval of a new, effective treatment regimen for FLC. The additional data provided by this expanded Phase 1 study will be crucial to determine the size (number of patients) and precise design of the Phase 2/3 clinical trial aimed at obtaining regulatory approval. It also will shed light on whether specific subgroups of patients are more likely to respond to the therapy.
Details of the trial and contact information can be found at: https://www.clinicaltrials.gov/ct2/show/NCT-04248569.