Unmet NeedFibrolamellar carcinoma (FLC) is an extremely rare form of cancer. It affects both men and women in approximately 1 in 5,000,000 people with greater frequency among young adults with a median age of diagnosis of 25. Unfortunately, there is no standard of care therapy for this cancer, and patients with this disease have an expected median survival of approximately 12 months.
Technology OverviewHopkins researchers have designed a vaccine targeting a unique fibrolamellar carcinoma oncogenic fusion protein, involving a kinase, which was recently identified as the signature genetic event initiating FLC. The researchers developed a novel vaccine for the treatment for fibrolamellar hepatocellular carcinoma (FL-HCC). The vaccine is based upon a peptide encoded by the fusion gene and a concomitant adjuvant. Having tested their FL-HCC vaccine in mice, in vivo data demonstrated a marked delay in tumor growth, as well as, significantly reduced tumor volume and growth rate.
This fusion protein has also been identified in other cancers, including pancreatic and biliary cancers. If proven clinically successful, this could be developed further as a new therapeutic approach to this lethal form of liver malignancy. This fusion kinase target may be an expanded opportunity for neoantigen-specific immunotherapy in other indications.
Stage of DevelopmentA human clinical trial has been fully funded by the Fibrolamellar Cancer Foundation and Bristol-Meyer-Squibb with patient recruitment beginning soon.
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