In a phase IIa multiple-basket study (MyPathway) reported in the Journal of Clinical Oncology, Hainsworth et al found that agents targeting specific molecular alterations produced responses in tumors outside of current labeling for the agents, with high response rates being observed in some tumor types.
Study Details
In the study, between April 2014 and November 2016, 251 patients with advanced solid refractory tumors of 35 different types received targeted treatment consisting of pertuzumab (Perjeta) plus trastuzumab (Herceptin), erlotinib (Tarceva), vemurafenib (Zelboraf), or vismodegib (Erivedge) for tumors harboring alterations in HER2, EGFR, BRAF, or the Hedgehog pathway, respectively. The primary outcome measure was investigator-assessed objective response rate within each tumor-pathway cohort.
Response Rates
The efficacy population consisted of 230 treated patients evaluated for response or who discontinued treatment before evaluation. Among these, 52 patients (23%) with 14 tumor types had an objective response, including complete response in 4 patients. Responses were observed in:
Tumor-pathway cohorts with high objective response rates included HER2 amplified/overexpressing colorectal cancer (14 of 37, 38%) and BRAF V600 mutated non–small cell lung cancer (6 of 14, 43%).
The investigators concluded, “The four currently approved targeted therapy regimens in the MyPathway study produced meaningful responses when administered without chemotherapy in several refractory solid tumor types not currently labeled for these agents.”
The study was supported by Roche/Genentech.
John D. Hainsworth, MD, of Sarah Cannon Research Institute, is the corresponding author for the Journal of Clinical Oncology article.
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