Under the coordination of Institut Curie in Paris, PEP-Therapy and 11 other partners have received 6 million euros of funding from the European Union's Horizon 2020 research and innovation programme to identify new therapeutic approaches to treat metastatic uveal melanoma in a 5-year project.
Although rare, uveal melanoma (UM) is the most common primary eye cancer in adults. While the primary tumour can often be treated effectively, up to 30 % of patients develop metastases most often in the liver, for whom no effective therapy is available. Metastatic UM patients require tailored therapies for their disease.
A project placing the patient central in therapy development
The UM Cure 2020 Consortium has gathered around a project targeted at identifying and validating at the preclinical level novel therapeutic approaches for the treatment of UM metastases. Dr Sergio Roman-Roman, Head of Translational Research department at Institut Curie and Coordinator of UM Cure 2020 says: “Our ambition is to significantly increase the survival of metastatic UM patients, a parameter that has remained unchanged in the last decades. Although we are aware this is an enormous challenge, we believe that we can improve the outcome of UM patients through the identification of new therapies and the initiation of UM-dedicated clinical trials.” The objective is to achieve the initiation of trials sponsored by academia or pharma from 2018.
To achieve this goal, it proposes an innovative concept placing the patient central in the therapy development process. While the pharmaceutical industry mainly focuses on developing drugs that may work in as many tumour types as possible, this approach only rarely results in drugs that work in rare tumours such as UM. The centre of the UM Cure 2020 approach is to characterise tumour tissue from UM patients with metastases, in order to define actionable targets. This includes the characterisation of the genetic landscape of metastatic UM and its microenvironment, and proteomic studies to address signalling pathway deregulation. Novel relevant in vitro and in vivo preclinical models of the metastatic disease will be used to evaluate single or combinations of drugs.
The project also aims to validate accurate biomarkers to evaluate therapies and detect metastases as early as possible. Underpinning this will be the UM Cure 2020 virtual biobank registry, linking biobanks in four referral centres involved into a harmonised network. The centres, in Paris (Institut Curie), Leiden (Leiden University Medical Centre), Liverpool (University of Liverpool) and Krakow (Jagiellonian University), will prospectively collect primary and metastatic UM samples, in order to gather a sufficient number of samples from this rare disease for its thorough characterisation. Single drugs and/or combinations showing promising preclinical data will then be tested in clinical trials, outside the frame of the project.
Dissemination of results under the lead of the Champalimaud Foundation (Lisbon, Portugal), will include our support to the formation of a European UM patients and patient advocates network with partner organisation the Melanoma Patient Network Europe (Uppsala, Sweden), as well as a dedicated UM patient and caregiver’s interface as part of the project website (www.umcure2020.org), in order to increase patient information and disease awareness. Patients are therefore central in each of the research steps up until the dissemination and implementation of the results.
The UM Cure 2020 Consortium, excellence gathered in the fight against metastatic UM
Maximising resources and coordinating research efforts through a Consortium like UM Cure 2020 are mandatory to succeed in this rare disease field. There has been no such previous cooperation of scientists with the aim of performing a UM research project of this amplitude in the EU or the rest of the world.
The UM Cure 2020 consortium is made of twelve partner institutions, which provide complementary expertise starting by an important track record in UM research and/or clinical management (Cancer Research UK Manchester Institute, UK, in addition to the four reference centres listed above), but also preclinical studies for cancers in general (Leiden University, The Netherlands, University of Trento, Italy), early clinical development (PEP-Therapy, France) and biomarker development (PamGene, The Netherlands), as well as patient advocacy (Melanoma Patient Network Europe), dissemination (The Champalimaud Foundation) and project management of multi-partners collaborative projects (seeding science, France).
The role of project partners is described in further details in the project website www.umcure2020.org.
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