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Celebrating the 10th Anniversary

A decade of discoveries in Cancer Discovery. For the community. By the Community.


Early Promise for Genome-Driven Therapy

As high-throughput genomic analyses became more clinically feasible in the last decade, whether genomic profiling of tumors could actually provide clinical benefit remained unclear. In 2017, Massard, André, Soria, and colleagues presented results from the Molecular Screening for Cancer Treatment Optimization (MOSCATO) 01 trial, one of the first prospective clinical trials designed to address this question. Of 1,035 adult patients with advanced cancer enrolled, 843 had both a successful tumor biopsy and molecular profiling completed. An actionable target for which an approved therapy did not yet exist was detected in 411 of these patients, and ultimately 199 patients (24% of those whose tumors were profiled) received a matched therapy as part of ongoing phase I/II trials. In MOSCATO, patients served as their own control: efficacy was determined by whether the progression-free survival achieved on the immediate prior line of therapy (PFS1) was exceeded by the PFS on the matched therapy (PFS2) by 30% or more. MOSCATO met its primary endpoint in showing that this PFS2/PFS1 ratio threshold was surpassed in 33% of patients, a rate that was likely affected by some patients receiving subtherapeutic doses of experimental agents in phase I dose-escalation studies and by the exclusion of patients for whom an approved therapy existed for their genomic alteration and tumor type. Though the number of patients who were eligible for matched therapy was relatively low due to limitations inherent to different molecular profiling approaches, incomplete annotation of gene variants, and clinical availability of therapies targeted to a specific alteration—issues the precision medicine field still grapples with today—this landmark trial provided the first evidence for the utility of genome-driven therapy as a treatment paradigm and inspiration for matched targeted therapy trials that followed in the years to come.

Read 2017 article by Massard and colleagues


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