The Drug Rediscovery Protocol: approved cancer drugs used outside their licence, inside a national trial

Large-scale genetic profiling of tumours finds actionable variants for which approved drugs exist, but when patients are treated with those drugs outside their label the successes and failures are not systematically collected or shared. The Drug Rediscovery Protocol is an adaptive precision-oncology trial that treats such patients, who must have exhausted or declined standard therapy, in cohorts defined by tumour type and molecular variant, to find signals of activity.

The abstract reports an overall clinical benefit rate, defined as complete or partial response or stable disease beyond 16 weeks, of 34 per cent in 215 treated patients, 136 of whom received targeted therapies and 79 immunotherapy. The median duration of clinical benefit was 9 months (95 per cent confidence interval 8 to 11), with 26 patients still benefiting at data cut-off. A cohort of patients with microsatellite-unstable tumours given nivolumab had a clinical benefit rate of 63 per cent, while patients with colorectal cancer with a relatively low mutational load gained only limited benefit from immunotherapy.