Genomics-guided off-label cancer therapy shows benefit in defined subgroups (DRUP trial)
In the Drug Rediscovery Protocol (DRUP), 1,610 patients with advanced solid tumors were treated with 37 off-label drugs matched to actionable genomic alterations. Of 1,363 evaluable patients, the clinical benefit rate was 34.9% and the objective response rate was 15.7%, with median progression-free and overall survival of 3.4 months and 8.2 months, respectively; grade 3+ treatment-related adverse events occurred in 28.4%, leading to discontinuation in 9.4%. Exceptional responders (7.0%) appeared across molecular subgroups, and some results supported Dutch reimbursement. The authors emphasize meaningful benefit in defined molecular contexts despite overall modest activity, advocating structured, data-generating frameworks, earlier intervention, biomarker refinement, and international collaboration to guide safe use and potential label expansion.