Synthetic lethality in ATM-deficient RAD50-mutant tumors underlies outlier response to cancer therapy
Ataxia Telangiectasia Mutated Proteins
DNA Repair Enzymes
Strategies to effect deep and lasting responses to cancer therapy in patients with metastatic disease have remained difficult to attain, especially in early-phase clinical trials. Here, we present an in-depth genomic and functional genetic analysis identifying RAD50 hypomorphism as a contributing factor to a curative response to systemic combination therapy in a patient with recurrent, metastatic small-cell cancer.