biostudies-literatureUnknown28(7)Hoes LRBristol-Myers SquibbPfizerIpsen FundNOVARTIS | AlconEisaiRoche NederlandBayer, United KingdomMerck &amp; Co. | Merck Sharp and DohmeAmgen Inc | Amgen NederlandClovis OncologyBoehringer IngelheimBarcode for Life FoundationAstraZenecaKWF Kankerbestrijding<h4>Purpose</h4>Patients with rare cancers (incidence less than 6 cases per 100,000 persons per year) commonly have less treatment opportunities and are understudied at the level of genomic targets. We hypothesized that patients with rare cancer benefit from approved anticancer drugs outside their label similar to common cancers.<h4>Experimental design</h4>In the Drug Rediscovery Protocol (DRUP), patients with therapy-refractory metastatic cancers harboring an actionable molecular profile are matched to FDA/European Medicines Agency-approved targeted therapy or immunotherapy. Patients are enrolled in parallel cohorts based on the histologic tumor type, molecular profile and study drug. Primary endpoint is clinical benefit (complete response, partial response, stable disease ≥ 16 weeks).<h4Clinical cancer research : an official journal of the American Association for Cancer Research1402-1411https://www.ebi.ac.uk/biostudies/studies/S-EPMC9365364biostudies-literaturePatients with Rare Cancers in the Drug Rediscovery Protocol (DRUP) Benefit from Genomics-Guided Treatment.PMC9365364Cuppen Ede Groot JWBde Leng WJGelderblom HJansen AMLvan Herpen CMLvan Werkhoven Ede Groot DJVoest EEvan Berge Henegouwen JMErdkamp Fvan der Velden DLvan de Haar JVerheul HMWBeerepoot LVHoeben ASmit EFHoes LRvan der Wijngaart Hvan der Noort VHendriks MPHuitema ADRRoepman PLabots MZeverijn LJLolkema MPGort EHMorreau HKerver EDvan der Graaf WTAfalsePatients with Rare Cancers in the Drug Rediscovery Protocol (DRUP) Benefit from Genomics-Guided Treatment.<h4>Purpose</h4>Patients with rare cancers (incidence less than 6 cases per 100,000 persons per year) commonly have less treatment opportunities and are understudied at the level of genomic targets. We hypothesized that patients with rare cancer benefit from approved anticancer drugs outside their label similar to common cancers.<h4>Experimental design</h4>In the Drug Rediscovery Protocol (DRUP), patients with therapy-refractory metastatic cancers harboring an actionable molecular profile are matched to FDA/European Medicines Agency-approved targeted therapy or immunotherapy. Patients are enrolled in parallel cohorts based on the histologic tumor type, molecular profile and study drug. Primary endpoint is clinical benefit (complete response, partial response, stable disease ≥ 16 weeks).<h42022-01-01T00:00:00Z2022 Apr2026-07-14T21:12:18.883Z2025-04-06T14:11:07.824ZS-EPMC93653643504606210.1158/1078-0432.CCR-21-3752