UM Cure 2020 – A consortium of European experts in uveal melanoma to identify new therapies for patients with metastatic disease

Abstract

PurposeUveal melanoma (UM) is a rare intraocular tumour with an incidence of 5 cases per million individuals per year. Up to 30% of UM patients develop metastases, most often in the liver, and there is no therapy to either prevent or treat these metastases. Despite new discoveries in the genetic and molecular background of the primary tumour, little is known about the metastatic disease.In UM Cure 2020, funded by European Union's Horizon 2020 programme, we will identify and validate at the preclinical level novel therapeutic approaches for the treatment of UM metastases (www.umcure2020.org). The Consortium brings together major EU experts in clinical, translational and basic research on UM, as well as patient representatives and innovative biotech companies.MethodsAn ambitious multidisciplinary approach is proposed to move from patient tissue characterisation to preclinical evaluation of single or combinations of drugs. We will characterise the genetic landscape of metastatic UM and its microenvironment, perform proteomic studies to address signalling pathway deregulation and establish novel relevant in vitro and in vivo UM models. Underpinning this will be our virtual sample registry, linking existing biobanks into a harmonised network, which will prospectively collect primary and metastatic UM samples.ResultsIn parallel, we are already evaluating in the first phase of the project the efficacy of a series of active compounds using partners’ available models. In addition to the initiation of UM‐dedicated clinical trials, dissemination of results includes initiatives to increase patient information and disease awareness, in particular by supporting the formation of a European UM patient network.ConclusionsThe UM Cure 2020 Consortium holds great potential to make significant advances in the treatment of metastatic UM, at present an incurable disease.