Drug Repurposing in Pancreatic Cancer: A Multi-Stakeholder Perspective to Improve Treatment Options for Pancreatic Cancer Patients

Evelyn Hewitt,1 Gauthier Bouche,2 Alexandre Costa Alencar,3 Stephen J Bigelsen,4 Raluca Radu,5 Violeta Stoyanova-Beninska,6 Alfredo Carrato,7,8 Federica Valsecchi,9 Alicia Soler Cantón,10 Helene G van der Meer,11 María Laura García Bermejo,12 Alfredo Budillon,13 Luca Cardone,14,15 Ilse Rooman,2,16 Hans Platteeuw,17 Judit Baijet,18 Claudia Fuchs18 1Beacon: for Rare Disease, Cambridge, UK; 2The Anticancer Fund, Meise, Belgium; 3Rising Tide Foundation for Clinical Cancer Research, Schaffhausen, Switzerland; 4Patient Advocate, Department of Allergy, Asthma and Immunology, Rutgers New Jersey Medical School, Newark, NJ, USA; 5Medicines for Europe, Brussels, Belgium; 6College ter Beoordeling van Geneesmiddelen, Utrecht, the Netherlands; 7Department of Medical Oncology, Alcalá University, Instituto Ramón Y Cajal de Investigación Sanitaria (IRYCIS), Centro de Investigación Biomédica En Red de Cáncer (CIBERONC), Ramon Y Cajal University Hospital, Madrid, Spain; 8Pancreatic Cancer Europe, Brussels, Belgium; 9Fondazione Nadia Valsecchi, Brescia, Italy; 10EATRIS ERIC, European Infrastructure for Translational Medicine, Amsterdam, the Netherlands; 11ZonMw, The Hague, the Netherlands; 12Biomarkers and Therapeutic Targets Group, Instituto Ramón Y Cajal de Investigación Sanitaria (IRYCIS), Ramon Y Cajal University Hospital, Madrid, Spain; 13Scientific Directorate, Istituto Nazionale Tumori – IRCCS - Fondazione G. Pascale, Naples, Italy; 14Institute of BiOChemistry and Cell Biology (IBBC), CNR c/o Campus Internazionale “a.buzzati-Traverso”, Monterotondo Scalo Roma (Rome), Italy; 15Department of Tumour Immunology and Immunotherapy, IRCCS Regina Elena National Cancer Institute, Rome, Italy; 16Translational Oncology Research Center (TORC), Laboratory of Medical and Molecular Oncology, (LMMO) Vrije Universiteit Brussel, Brussels, Belgium; 17Galenicap, Brussels, Belgium; 18EURORDIS - Rare Diseases Europe, Paris, FranceCorrespondence: Claudia Fuchs, EURORDIS - Rare Diseases Europe, Rare Diseases Platform, 96 rue Didot, Paris, 75014, France, Email [email protected] Evelyn Hewitt, Beacon for Rare Diseases, 66 Devonshire Road, Cambridge, CB1 2BL, United Kingdom, Email [email protected]: Pancreatic cancer (PC) remains one of the most challenging malignancies to treat. Current therapeutic options are unsatisfactory, and there is an urgent need for more effective and less toxic drugs to improve the dismal prognosis of PC. In recent years, drug repurposing (DR) has emerged as an attractive strategy to identify novel treatments for PC by leveraging existing drugs approved for other indications. Through the use of electronic medical records, Artificial Intelligence, study of metabolic pathways, signalling pathways, and many other approaches, it has become much easier in recent years to identify potential novel uses for old drugs. Although policy, funding and research attention in this area are steadily growing, major challenges to efficient and effective patient-centric DR in PC need to be addressed. These include but are not limited to regulatory, financial and funding barriers and the lack of coordination and collaboration among several sectors and stakeholders. To explore the opportunities and challenges associated with DR in PC, a one-day multi-stakeholder meeting was held on 14th of November 2024 in Brussels, Belgium as part of the REMEDi4ALL project. This meeting provided a platform for researchers, clinicians, industry representatives, funders, regulatory experts, and patient advocates to discuss and propose actions to optimize and accelerate DR in PC. Insights from this meeting support the potential of DR to enhance PC treatment options while highlighting the importance of systemic and supportive changes in the regulatory, policy and funding landscapes, interdisciplinary collaboration, data sharing, and patient involvement in driving therapeutic innovation. This summary highlights key outcomes and recommendations from the meeting in informing future efforts to advance DR initiatives in the context of PC.Keywords: drug repurposing, pancreatic cancer, multi-stakeholder discussion, collaboration, patient centricity