Fibrolamellar Carcinoma in the Molecular Era: From DNAJB1::PRKACA Biology to Precision Therapeutic Strategies
PURPOSE: Fibrolamellar carcinoma (FLC) is a rare primary liver cancer of adolescents and young adults that arises in a non-cirrhotic liver and was long treated as a variant of hepatocellular carcinoma (HCC). Fusion-directed immunotherapy has provided early clinical proof of concept: a phase 1 trial of a fusion peptide vaccine combined with nivolumab and ipilimumab reported an objective response rate of 19%, with fusion-specific T-cell responses in 9 of 12 patients completing the priming phase.
PURPOSE: Fibrolamellar carcinoma (FLC) is a rare primary liver cancer of adolescents and young adults that arises in a non-cirrhotic liver and was long treated as a variant of hepatocellular carcinoma (HCC). Fusion-directed immunotherapy has provided early clinical proof of concept: a phase 1 trial of a fusion peptide vaccine combined with nivolumab and ipilimumab reported an objective response rate of 19%, with fusion-specific T-cell responses in 9 of 12 patients completing the priming phase.
What the study was
- Study design
- Phase 1 clinical trial
- Population
- Patients
- Sample size
- 12
- Category
- Treatment Innovation
- Maturity
- Validated
- Journal
- Journal of gastrointestinal cancer
Why it surfaced
Matched Novel therapeutics, Precision oncology and genomic medicine; Phase 1 clinical trial; human study; score 7/10 (n=2 r=3 dq=1 pop=1)
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