Using high-throughput multigene sequencing panel tests with tumor samples to inform decision-making about targeted therapies is associated with prolonged progression-free survival (PFS) rates among previously-treated patients with 12 different, difficult-to-treat types of metastatic cancer, according to results from the prospective Molecular Screening for Cancer Treatment Optimization (MOSCATO 01; Identifier: NCT02613962) clinical trial. The findings were published in Cancer Discovery.1

“The identification of genomic drivers of cancers and the development of targeted therapies have led to the hypothesis that testing for a large number of genes across all tumor types could improve outcomes for patients with advanced cancers,” explained study coauthor Fabrice André, MD, PhD, of INSERM Unit U981 and the Intitut Gustave Roussy in Villejuif, France, in an American Association for Cancer Research (AACR) press release.2 The authors assessed the ratio of PFS from patients’ targeted treatments and their prior (first) lines of therapy (PFS2/PFS1).

The study team successfully sequenced 843 patients’ tumor samples, identifying at least 1 actionable gene target in 411 patients’ tumors, 199 of whom received targeted cancer therapy matched to their tumor mutations.

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The most frequent cancer types were digestive tumors (21% of sequenced patients’ tumor samples), lung (18%), urological (16%), breast (14%), and head and neck tumors (12%).

Across tumor types, the most frequently administered classes of targeted therapy were PI3K-AKT-mTOR pathway drugs (59 patients), NOTCH (25), ERBB2 (26), and FGFR (24). EGFR-targeting and MAPK-targeting drugs were each administered to 12 patients, and MET targets in 11 patients.

RELATED: More Work Needed Before Multigene Mutation Testing Ready for Routine Clinical Practice

Overall response rate (ORR) was 11%. Only 2 patients had a complete tumor response to targeted therapy and 20 had partial responses; 100 patients had stable disease and 33 saw disease progression.

The study was not randomized.


  1. Massard C, Michiels S, Ferté C, et al. High-throughput genomics and clinical outcome in hard-to-treat advanced cancers: results of the MOSCATO 01 trial. Cancer Discov. 2017 Apr 1. doi: 10.1158/2159-8290.CD-16-1396 [Epub ahead of print]
  2. Targeted therapies selected based on multigene panel improved outcomes for patients with hard-to-treat cancers [news release]. Philadelphia, PA: American Association for Cancer Research; April 1, 2017. Accessed April 1, 2017.