FCF is pleased to announce that two of its research partners – Mark Yarchoan, MD of the Johns Hopkins Kimmel Cancer Center and Paul Thomas, PhD of St. Jude Children’s Medical Center have received significant funding from the National Cancer Institute (NCI) for a collaborative fibrolamellar research project. Their initiative “Neoantigen-specific T cell responses for Fibrolamellar Hepatocellular Carcinoma” aims to lay the groundwork for the next generation clinical trials for FLC, including adoptive cell therapies with specificity for the DNAJB1-PRKACA fusion. In the study, the team will conduct a clinical trial in patients with unresectable FLC of a vaccine targeting the DNAJB1-PRKACA chimera, in combination with the checkpoint inhibitors (ICIs) nivolumab and ipilimumab. As part of that effort, they will determine if the vaccine combined with ICIs increases the number of T cells targeting the DNAJB1-PRKACA chimera in the tumor. After identifying T cell receptors (TCRs) specific for the FLC fusion protein, they will treat humanized mouse models with cells transduced with the identified TCRs to understand their relative targeting efficiency. The hope is that the research effort will identify optimal peptide and TCR combinations for targeting the DNAJB1-PRKACA fusion.

This project is funded under NCI’s Investigator-Initiated Early Phase Clinical Trials for Cancer Treatment and Diagnosis funding opportunity, which provides funding for research projects that implement early phase (Phase 0, I, and II) investigator-initiated clinical trials focused on cancer-targeted diagnostic and therapeutic interventions. The five-year grant will provide the combined Johns Hopkins/St. Jude effort $733,308 in funding for fiscal year 2022.

This exciting project is a great example of how “seed” funding, insightful initial research efforts and multi-institutional collaboration can lead to significant increases in financial support for FLC research.