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Targeted Therapy for Advanced Solid Tumors on the Basis of Molecular Profiles: Results From MyPathway, an Open-Label,
John D Hainsworth1, Funda Meric-Bernstam1, Charles Swanton1
1John D. Hainsworth, David R. Spigel, and Howard Burris, Sarah Cannon Research Institute; Tennessee Oncology, Nashville, TN; Funda Meric-Bernstam, University of Texas MD Anderson Cancer Center, Houston, TX; Charles Swanton, Francis Crick Institute, London, United Kingdom; Herbert Hurwitz, Duke University Medical Center, Durham, NC; Christopher Sweeney, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA; Ron Bose, Washington University School of Medicine, St Louis, MO; Bongin Yoo, Alisha Stein, and Mary Beattie, Genentech, South San Francisco; and Razelle Kurzrock, Moores Cancer Center, University of California San Diego, San Diego, CA.
Abstract:
Purpose Detection of specific molecular alterations in tumors guides the selection of effective targeted treatment of patients with several types of cancer. These molecular alterations may occur in other tumor types for which the efficacy of targeted therapy remains unclear. The MyPathway study evaluates the efficacy and safety of selected targeted therapies in tumor types that harbor relevant genetic alterations but are outside of current labeling for these treatments. Methods MyPathway ( ClinicalTrials.gov identifier: NCT02091141) is a multicenter, nonrandomized, phase IIa multiple basket study. Patients with advanced refractory solid tumors harboring molecular alterations in human epidermal growth factor receptor-2, epidermal growth factor receptor, v-raf murine sarcoma viral oncogene homolog B1, or the Hedgehog pathway are treated with pertuzumab plus trastuzumab, erlotinib, vemurafenib, or vismodegib, respectively. The primary end point is investigator-assessed objective response rate within each tumor-pathway cohort. Results Between April 1, 2014 and November 1, 2016, 251 patients with 35 different tumor types received study treatment. The efficacy population contains 230 treated patients who were evaluated for response or discontinued treatment before evaluation. Fifty-two patients (23%) with 14 different tumor types had objective responses (complete, n = 4; partial, n = 48). Tumor-pathway cohorts with notable objective response rates included human epidermal growth factor receptor-2-amplified/overexpressing colorectal (38% [14 of 37]; 95% CI, 23% to 55%) and v-raf murine sarcoma viral oncogene homolog B1 V600-mutated non-small-cell lung cancer (43% [six of 14]; 95% CI, 18% to 71%). Conclusion 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.
Insights
Targeted therapies showed efficacy in various refractory solid tumors beyond their approved indications. The MyPathway study found meaningful objective responses in patients with specific molecular alterations, expanding treatment options.
Area of Science:
- Oncology
- Molecular Biology
- Clinical Trials
Background:
- Molecular alterations in tumors guide targeted cancer therapy.
- Efficacy of targeted therapies in unapproved tumor types with relevant alterations is often unclear.
Purpose of the Study:
- To evaluate the efficacy and safety of targeted therapies in tumor types with specific genetic alterations outside current labeling.
- To assess targeted treatments in the MyPathway study (NCT02091141) for advanced refractory solid tumors.
Main Methods:
- A multicenter, nonrandomized, phase IIa multiple basket study design.
- Patients with advanced refractory solid tumors harboring alterations in HER2, EGFR, BRAF, or Hedgehog pathways received specific targeted therapies.
- Primary endpoint: investigator-assessed objective response rate per cohort.
Main Results:
- 251 patients with 35 tumor types received treatment; 230 were evaluated for efficacy.
- Objective responses (complete or partial) were observed in 52 patients (23%) across 14 tumor types.
- Notable response rates included HER2-amplified/overexpressing colorectal cancer (38%) and BRAF V600-mutated non-small-cell lung cancer (43%).
Conclusions:
- Four approved targeted therapy regimens demonstrated meaningful responses in refractory solid tumors not currently indicated for these agents.
- The MyPathway study supports the use of targeted therapies in molecularly selected tumor types beyond their current labeling.
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