ASCO-Precision Medicine Getting Better

James Radke, PhD

Targeted therapies are good but not great for patients with cancer. A new study is hoping to make most targeted therapies (aka precision medicine) great.
Preliminary results for the Phase 2 MyPathway study being presented at the 2016 ASCO Annual Meeting in Chicago show the benefit of precision medicine in cancer patients.  The researchers are matching patients with molecular abnormalities in their tumors to corresponding targeted treatments.

About the Study

MyPathway is an ongoing non-randomized, open-label trial that evaluates 4 treatment regimens in patients with advanced cancer for whom no beneficial treatment is available. This is a nationwide study with 39 currently participating sites.
Patients in the study must have had previous molecular studies of the cancer showing abnormalities in the HER2, BRAF, Hedgehog or EGFR pathways. Patients were then matched with drugs targeting those abnormalities. Patients received a combination of trastuzumab (Herceptin) and pertuzumab (Perjeta) if they had HER2 abnormalities (amplification, overexpression, or mutation); vemurafenib (Zelboraf) for BRAF mutations; vismodegib (Erivedge) for Hedgehog pathway mutations; and erlotinib (Tarceva) for EGFR mutations. Patients had a median of 3 prior lines of therapy before starting the trial.

Preliminary Results

As of December 2015, 129 patients had been evaluated, including those with HER2 (n = 82; 53 amplifications, 23 mutations, 5 both, 1 RBMS-NRG1 fusion), BRAF (n = 33; 18 V600E, 15 other), Hh (n = 8; 7 PTCH1, 1 SMO), or EGFR (n = 6). Of the 129 patients, 118 had sufficient data for follow-up and among these poorly responding patients, 22 responded to targeted therapies.

In a news release, lead study author John D. Hainsworth, MD, senior investigator at Sarah Cannon Research Institute in Nashville, TN said, “Although it is still early to draw conclusions, our findings suggest that, for example, HER2-targeted therapy could be expanded beyond the current indications of HER2-positive breast and gastric cancers. Our study gives strong early signals for activity of HER2-targeted therapy in HER2-amplified colorectal cancers (those with extra copies of HER2 gene), and possibly other HER2-positive cancers.”


Hainsworth JD, Meric-Bernstam F, Swanton C, et al. Targeted therapy for advanced solid tumors based on molecular profiles: Early results from MyPathway, an open-label, phase IIa umbrella basket study. J Clin Oncol 2016;34 (suppl; abstr LBA11511).
Printer Printing...