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Precision Medicine Shown to Boost Cancer Outcomes

Stethoscope on DNA sheet

(National Heart, Lung, and Blood Institute)

7 June 2016. An analysis of results from early-stage clinical trials shows trials using treatments for cancer aimed at specific molecular targets had higher response rates and longer survival times than studies not using these targets. The team from University of California in San Diego, led by postdoctoral researcher Maria Schwaederlé, published its findings in yesterday’s issue of the journal JAMA Oncology. The study was also presented as a poster at this year’s American Society of Clinical Oncology annual meeting.

Schwaederlé and colleagues from UC-San Diego’s Center for Personalized Cancer Therapy are seeking to document benefits to patients of using precision medicine to guide cancer treatments. Precision medicine takes advantage of recent advances in technology making it faster and less expensive to genetically analyze tumors to reveal underlying mutations, then finding treatments addressing those mutations rather than using drugs directed against cancer found in a specific part of the body.

The UC-San Diego team analyzed records of 346 early-stage clinical trials in 2011 through 2013 testing new cancer treatments. Early-stage trials, also known as phase 1 trials, mainly evaluate the safety of new therapies, but often also gather evidence of efficacy. The trials reviewed in this study enrolled a total of 13,203 adult patients in tests of single therapies. Combination therapies, as well as studies of supportive or palliative care, were excluded.

The researchers found only 58 or 17 percent of the trials tested treatments with a precision medicine approach, i.e. targeting specific biomarkers. The remaining 293 trials investigated effects of drugs on cancer in organs, with most (84%) of these trials testing drugs against solid-tumor cancers. The studies of precision medicine were more evenly divided: 25 (43%) evaluated treatments for solid tumors, while 33 (57%) tested blood-related cancer therapies.

The results show more patients in precision medicine trials receiving the test treatments — some participants received placebos — reported responding to those treatments (median of 31%) than patients in the trials receiving treatments aimed at cancer in specific organs (median of 5%). In addition, patients receiving precision medicine treatments in those trials reported longer median progression-free survival times (5.7 months) compared to participants receiving treatments aimed at cancer in specific organs (3 months).

Identifying patients for treatments using biomarkers, say the authors, also appears to improve cancer outcomes. In 234 sample groups in the reviewed studies, investigators used biomarkers to identify patients for treatments, and those patients reported median response rates of 31 percent, compared to 5 percent for participants where biomarkers were not part of the selection criteria.

“Our analysis shows that in the era of precision medicine, phase 1 clinical trials using personalized therapy with a biomarker-based approach can do more than assessing the toxicity and side effects,” says first-author Schwaederlé in a university statement. “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.”

The study’s senior author and director of the Center for Personalized Therapy Razelle Kurzrock is also a co-founder of the spin-off enterprise CureMatch Inc. in San Diego. CureMatch develops computational decision support systems for treatment strategies with precision medicine.

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2 comments to Precision Medicine Shown to Boost Cancer Outcomes

  • It s important for health systems to understand data isn t uniform in these systems, and taking into consideration how it s viewed at the ground level will affect how these key agents translate the information, she added. The only way to use EHRs as a prediction tool for precision outcomes, McMilland said, is to validate the data using evidence-based research.

  • The goal of Intermountain Cancer Genomics is to allow all patients to have access to precision medicine. Initially piloted internally, Intermountain has now made their genetically targeted therapeutic approach accessible to other providers