Patients whose treatment was selected based on the molecular characteristics of their particular tumor had significantly better outcomes compared with patients who did not, a meta-analysis of phase 1 clinical trials that will be presented at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, IL, has shown.1

Because previous meta-analyses of phase 2 and 3 trials demonstrated improved outcomes with precision medicine approaches, researchers sought to compare patients outcomes between phase 1 trials of single agents that used precision medicine vs those that did not.

The investigators analyzed efficacy and safety data from 346 phase 1 clinical trials published between 2011 and 2013. From those, 58 treatment arms employed a biomarker-based treatment selection strategy while 293 did not. Only 1 precision medicine trial assessed chemotherapy, specifically topotecan, as opposed to a targeted agent.

Continue Reading

Results showed that tumor shrinkage rates were 30.6% in treatment arms that employed precision medicine compared with 4.9% in those that did not. Median progression-free survival was 5.7 months and 2.95 months, respectively.

“Our study suggests that, with a precision medicine approach, we can use a patient’s individual tumor biomarkers to determine whether they are likely to benefit from a particular therapy, even when that therapy is at the earliest stage of clinical development,” said lead study author Maria Schwaederle, PharmD, of the Center for Personalized Cancer Therapy, University of California-San Diego School of Medicine. “This strategy often results in good outcomes for patients, and I hope it will encourage and reassure doctors and patients considering enrollment in precision medicine-based phase I trials.”

Subgroup analyses further demonstrated similar results in the 57 trials evaluating targeted therapies. The researchers also found that selecting treatment based on DNA biomarkers was associated with higher tumor shrinkage rates (42%) vs protein biomarkers (22.4%).

RELATED: Non-small Cell Lung Cancer in Younger Patients May Be More Targetable With Precision Medicine

“Precision medicine is not the future of cancer care, it is the present. This study reinforces that the more we personalize treatment to the patient and the tumor, the better the outcomes—even in the earliest phases of research,” said Don S. Dizon, MD, FACP, ASCO spokesperson. “This is the same approach ASCO’s TAPUR trial is using, and we anticipate it will also bring new insights that lead to better therapies for patients in need.”


  1. Precision medicine yields better outcomes for patients in phase I clinical trials [news release]. Alexandria, VA: American Society of Clinical Oncology; May 18, 2016. Accessed May 18, 2016.