We are pleased to announce that FCF has awarded Dr. Juliane Walz of the University Hospital Tübingen a grant to develop and launch a new clinical trial for FLC patients. This phase I study will assess the safety and efficacy of a peptide vaccine (Fusion-VAC-XS15) against FLC’s driver protein in a different clinical context than has been examined to date.

Because of the high frequency of recurrence of FLC, patients whose tumors have been completely resected would experience great value from a treatment that prevents disease relapse. While a minority of FLC patients appear to be cured by surgery, many more relapse, sometimes years after the initial treatment. A recent case report documented recurrence in a fibrolamellar patient two decades after the apparently successful resection of the initial tumor.

This clinical trial will assess the utility of a peptide vaccine as “adjuvant” therapy in FLC patients who, after surgery and/or other treatment, are free of cancer as judged by radiological scans. The goal is to significantly delay or entirely prevent the return of the cancer. The vaccine will be given alone, without additional immunotherapy or any other systemic agent.

If successful, this new study could:

• Deliver a new treatment to prevent recurrence of FLC. This would address a major unmet need of the patient community, especially for individuals who achieve complete remission status, yet still carry a population of FLC cancer cells that could lead to relapse of their disease.
• Confirm that a targeted therapeutic vaccine alone can effectively stimulate the expansion of anti-FLC T cells, without exposing the patients to immune checkpoint inhibitors that could cause autoimmune side-effects.

FCF is thrilled to support this new effort from a world-class research team. Dr. Walz has attained a position of international leadership in the development of peptide-based immunotherapies for both infectious diseases and cancer. She and her team also have extensive experience with FLC. In 2022, they published an article on the potential of DP-targeted immunotherapy to treat FLC (Nature Communications, October 2022) that described the response achieved by a single FLC patient who had suffered several relapses of the disease before receiving a peptide vaccine against the DP fusion protein. Remarkably, that patient experienced no further relapses of FLC, and remains free of measurable disease 4 years after the treatment. Last year, the Walz team also launched a clinical trial at the University of Tübingen for patients with advanced or metastatic FLC, that tests the Fusion-VAC-XS15 vaccine in combination with an immune checkpoint inhibitor (ICI).

The new FusionVAC22_02 study will be directed from University Hospital Tübingen, Baden-Würtemberg, Germany. It will aim to enroll 20 subjects with FLC who have achieved complete remission status according to RECIST 1.1.

More information on the trial will be available after its approval by regulatory authorities.