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2019

A Pilot Study of a DNAJB1-PRKACA fusion kinase vaccine combined with nivolumab and ipilimumab for patients with fibrolamellar carcinoma (FLC)

Goal: Assess potential to induce immune response with a FLC therapeutic vaccine Principal Investigator: Mark Yarchoan, MD Grant length: Two years Study overview: Many patients with FLC currently receive an immune checkpoint inhibitor off-label. These drugs “take the brakes off” the immune system, enhancing its ability to fight cancer. However, clinical experience suggests that they …

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Retinoic acid-induced loss of DNAJB1-PRKACA fusion protein expression

Goal: Investigate the potential of retinoic acid therapy Principal Investigators: Andrew Yen, PhD and Praveen Sethupathy, PhD Grant length: One year Study overview: FLC is driven by the DNAJ-PKAc fusion protein. A potential therapeutic strategy would be to induce loss of this key driver protein. One approach to substantially alter gene expression in cancer cells …

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Molecular therapies for fibrolamellar carcinoma (FLC)

Goal: Investigate the potential of heat shock protein 70 (Hsp70) and mitogen-activated protein kinases (MAPKs) as therapeutic options for FLC Principal Investigator: John Scott, PhD Grant length: Two years Study overview: A main goal of this project was to determine how the abnormal form of protein kinase A (PKA) present in fibrolamellar carcinoma (FLC) cells …

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Targeting DNAJB1-PRKACA driven signaling dependencies in FLC

Goals: Investigate the potential of AURKA inhibitors for FLC treatment Principal Investigators: John Gordan, MD, PhD (UCSF) and Nabeel Bardeesy, PhD (MGH) Grant length: Two years Study overview: Even though the DNAJB1-PRKACA gene fusion is sufficient to trigger fibrolamellar liver cancer (FLC), no treatments directed at this target are clinically available. Most FLC patients receive …

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