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 the disease. 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. “We are incredibly excited to reopen the peptide vaccine trial and welcome additional patients,” said Dr. Baretti. Dr. Yarchoan added, “We are deeply grateful to the Fibrolamellar Cancer Foundation for their continued partnership — this kind of collaboration between researchers, advocacy groups, and patients is vital to driving meaningful progress and bringing new hope to those affected by this rare cancer.”

In this clinical trial, 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, as shown by the presence of circulating immune cells that can recognize the fusion peptide. 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 from their cancer. 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.

Below is a brief video of Dr. Baretti discussing preliminary results of the trial with The Video Journal of Oncology (VJOncology).

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), or contact:

• Marina Baretti, MD – mbarett1@jhu.edu, or
• Colleen Apostol, RN – GIClinicalTrials@jhmi.edu.