Timeframe: 2025 – 2027 Goal: Investigate ways to therapeutically target DNAJ-PKAc Principal Investigators: John Scott, PhD Study overview: Before new drugs can move forward into clinical development, scientists must confirm that they work as intended. This effort focuses on enabling this critical step for fibrolamellar carcinoma — validating and understanding how potential drugs interact with …
Timeframe: 2025 – 2027 Goal: Investigate ways to therapeutically target DNAJ-PKAc Principal Investigators: John Gordan, MD, PhD Study overview: Testing drugs in cells and living organisms is a critical step in developing new treatments. For a drug to work, it must move through complex cell structures and, in animals and eventually humans, overcome challenges like …
Timeframe: 2025 – 2027 Goal: Investigate ways to therapeutically target DNAJ-PKAc Principal Investigators: Rafael Couñago, PhD and David Drewry, PhD Study overview: Small-molecule inhibitors of protein kinases have transformed cancer research by improving survival rates and enabling more targeted therapies. This progress stems from the fact that many cancers exhibit dysregulated protein kinases. When these …
Timeframe: 2025 – 2027 Goal: Model development Principal Investigator: Nabeel Bardeesy, PhD Study overview: Typically, cancers requires activation of a “driver” of uncontrolled growth, such as the DNAJB1-PRKACA (DP) fusion in FLC, coupled with the loss of a “tumor suppressor” function. Tumor suppressors act as “brakes” on cancers, by causing cells that have gone out of control to undergo programmed suicide. Surprisingly, studies …
Timeframe: 2025 – 2026 Goal: Model development Principal Investigator: Nabeel Bardeesy, PhD Study overview: Cell line models and patient-derived xenographs (PDXs) are important tools that researchers can use to study disease. For FLC research to move forward, stable, reliable,patient-derived cell lines that truly represent the human disease are needed. While multiple FLC models have recently …
Timeframe: 2025 – 2027 Goals: Assess the potential of a FLC therapeutic vaccine to prevent disease recurrence Principal Investigators: Jens Bauer, PhD Study Details: This study, led by Dr. Jens Bauer of the University Hospital Tübingen, aims to develop a new approach to improve the effectiveness of the anti-FLC vaccines that are under development in …
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. …
Timeframe: 2025 – 2027 Goal: Determine if targeting SLC16A14 could be a useful treatment approach for FLC. Principal Investigator: Sean Ronnekleiv-Kelly, MD Study overview: Recently, Dr. Ronnekleiv-Kelly used sophisticated genetic engineering to create a highly promising mouse model of FLC and other cancers driven by the DNAJB1-PRKACA (DP) gene fusion. Dr. Ronnekleiv-Kelly’s first goal in …
Timeframe: 2025 – 2027 Goal: Investigate alternate means of therapeutically targeting DNAJ-PKAc Principal Investigator: Yasemin Sancak, PhD Study overview: This effort, led by Yasemin Sancak of the University of Washington, aims to study mitochondrial abnormalities in FLC. It has long been known that FLC cancer cells are filled with abnormal mitochondria, but the causes and implications of those mitochondrial changes are …