Precision Medicine and Cancer Outcomes

Improved response rates and extended periods of disease remission found with precision medicine.

Precision medicine demonstrated improved response rates and extended periods of disease remission, even during phase 1 trials.

In a meta-analysis published in JAMA Oncology, researchers reviewed 346 phase 1 clinical trials involving a total of 13,203 patients. The results of the analysis showed that more than 30% of patients treated with precision medicine therapy responded to treatment compared with 4.9% patients in the non-personalized medicine group.

Furthermore, patients in the precision medicine group experienced longer progression-free survival with a median of 5.7 months before the disease worsened, compared with 2.95 months in the other group.

“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,” said lead study author Maria Schwaederlé, PharmD. “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.”

There were 58 precision medicine treatment arms and 293 non-personalized arms. Precision medicine was associated with a higher response rate of 24.5% in patients with solid tumors compared with 4.5% in non-personalized approaches. Blood cancers had a 24.5% response rate for personalized medicine compared with 13.5%.

Precision medicine gave patients longer progression-free survival in both tumor types, at 4.1 months versus 2.8 months in solid tumors and 13.6 versus 4 months in hematologic tumors.

A sub-analysis of 234 arms that tested targeted drugs revealed that the use of biomarkers for determining a course of treatment led to a 31.1% response rate compared with 5.1% for those who did not.

An additional sub-analysis of the precision medicine trials showed that the use of genomic and protein biomarkers both improved outcomes, but that genomic biomarkers performed better.

Targeted genomic alterations had a 42% response rate compared to 22.4% if the biomarker was directed at the overexpression of a protein.

“What we observed is that phase 1 trials can serve both to inform us on the effectiveness of new therapies as well as identify patients likely to benefit most if a personalized approach is employed,” said senior study author Razelle Kurzrock, MD. “Another important point is that targeted drugs in and of themselves are often quite useless if not combined with a patient's individual tumor biomarkers to determine whether they are likely to benefit from a particular therapy.”