Ziel
Cholangiocarcinoma (CCA) is one of the deadliest cancers comprising a heterogeneous cluster of malignancies that are distinct to hepatocellular carcinoma (HCC) and that can emerge at any point in the biliary tree. While not very common, CCA is responsible for 2% of cancer-related deaths worldwide annually and rising. The incidence in European countries is increasing rapidly ranging from 1 to more than 4 cases/100,000. CCA is mostly diagnosed very late resulting in aggressive disease progression, poor treatment response, and dismal prognosis with a median survival of less than 2 years. Curative hepatic resection is an option in 10-30% cases and is mostly linked to recurrence within 12 months in over 50% of patients. For most patients, diagnosis will occur when the disease is already too advanced, and the only options are chemotherapies or palliative care. Current standard-of-care for CCA is unsatisfactory with very limited efficacy and adverse effects. Recently approved second-line therapeutics slightly improve outcome but are only suited to a small subset of CCA patients. Thus, there is a major unmet medical need for novel therapeutic strategies to improve patient outcome. Based on a robust data package obtained in a panel of CCA models within ERC HEPCIR, here we will explore the use of a novel class of compounds exploiting a unique and differentiated first-in-class mechanism-of-action and validate their efficacy in vivo as a first-line therapeutic for CCA. Proof-of-concept studies will be performed in state-of-the-art patient-derived models combined with scRNASeq and proteomics. The data obtained will serve as a pre-IND data package for this indication. An established collaboration with a pharma partner will ensure fast-track completion of the preclinical development towards IND. Collectively, ERC PoC CANDY will deliver completed proof-of-concept for a novel therapeutic approach for CCA which has the potential to transform patient care.
Wissenschaftliches Gebiet
CORDIS klassifiziert Projekte mit EuroSciVoc, einer mehrsprachigen Taxonomie der Wissenschaftsbereiche, durch einen halbautomatischen Prozess, der auf Verfahren der Verarbeitung natürlicher Sprache beruht.
CORDIS klassifiziert Projekte mit EuroSciVoc, einer mehrsprachigen Taxonomie der Wissenschaftsbereiche, durch einen halbautomatischen Prozess, der auf Verfahren der Verarbeitung natürlicher Sprache beruht.
Programm/Programme
- HORIZON.1.1 - European Research Council (ERC) Main Programme
Thema/Themen
Finanzierungsplan
HORIZON-AG-LS - HORIZON Lump Sum GrantGastgebende Einrichtung
75654 Paris
Frankreich