A meta-analysis of hundreds of cancer clinical trials revealed that therapeutic approaches using precision medicine had improved responses and longer disease remission, even in phase I trials, a study published in JAMA Oncology reports.1

Precision medicine uses genetic information from individual patients to guide and refine cancer treatment. This study examined 346 phase I clinical trials involving 13 203 patients. In trial arms in which precision medicine approaches were used, 30.6% of patients responded to treatment. Only 4.9% of patients in trial arms in which precision medicine was not used responded to treatment.

In addition, patients treated with precision medicine experienced longer progression-free survival (PFS), with a median of 5.7 months compared to 2.95 months before the disease progressed.

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“Our analysis shows that in the era of precision medicine, phase I clinical trials using personalized therapy with a biomarker-based approach can do more than assessing the toxicity and side effects,” said Maria Schwaederlé, PharmD, a researcher at University of California San Diego Moores Cancer Center and at the Center for Personalized Cancer Therapy, San Diego, California, and lead author of the study.

“These early trials can result in improved outcomes for patients, even among people whose disease is resistant to standard treatments, by selecting patients who will respond best using a personalized approach from the start.”

Of the clinical trials examined, 58 arms used precision medicine and 293 arms did not. Improvements in response rate and PFS occurred across tumor type when precision medicine was used. The use of personalized medicine correlated with a higher response rate in patients with solid tumors (24.5% vs 4.5%) and in patients with blood cancers (24.5% vs 13.5%).

In both tumor types, the use of precision medicine associated with longer PFS (4.1 months vs 2.8 months in patients with solid tumors and 13.6 months vs 4 months in patients with hematologic tumors).

Pairing targeted therapy with a biomarker can be important. An additional analysis of 234 arms testing targeted drugs revealed that the use of biomarkers to assign patients to treatment arms correlated with a response rate of 31.3% vs 5.1% in the arms that did not use biomarkers.

An additional analysis examined the use of protein and genomic biomarkers in the trials that used precision medicine, revealing genomic biomarkers as performing better than protein biomarkers. Targeting genomic elements associated with a 42% response rate vs a 22.4% response rate when protein biomarkers were targeted.


1. Schwaederle M, Zhao M, Lee JJ, et al. Association of biomarker-based treatment strategies with response rates and progression-free survival in refractory malignant neoplasms: a meta-analysis [published online June 6, 2016]. JAMA Oncol. doi:10.1001/jamaoncol.2016.2129.