ESMO22: Phase III trial shows targeting Notch pathway ‘DeFi’s desmoid tumor progression

A Phase III trial of a Notch inhibitor to treat desmoid tumors has reported impressive results at the ESMO Congress 2022 (9–13 September, Paris, France). DeFi, led by Bernd Kasper, Mannheim Cancer Center (Germany), has found that the gamma secretase inhibitor nirogacestat improved progression in patients with desmoid tumors, which are benign but aggressive soft tissue tumors that can cause pain and disfigurement. In the randomized controlled trial, 142 patients were recruited from 37 centers around the world and treated with either nirogacestat or a placebo. On average, treatment with nirogacestat led to a 71% lower risk of disease progression...