Liquid Biopsy Blood Tests Predict Breast Cancer Immunotherapy Response in Vanderbilt Study
Key Insights
Researchers at Vanderbilt-Ingram Cancer Center demonstrated that liquid biopsies using repeated blood sampling can effectively predict patient response to immunotherapy in high-risk, early-stage breast cancer (search).
The study analyzed 546 blood samples from 160 patients with HER2 (search)-negative stage 2 and 3 breast cancers, using RNA sequencing to study T cell activation gene expression patterns.
Molecular signals in blood samples successfully predicted patient response to the immunotherapy drug Pembrolizumab, offering a minimally invasive alternative to traditional tissue biopsies.
Researchers at the Vanderbilt-Ingram Cancer Center have identified a breakthrough approach to improving outcomes in high-risk, early-stage breast cancer (search), demonstrating that repeated blood sampling—known as liquid biopsy—can help assess and predict patient response to immunotherapy. Published in Science Translational Medicine, the study highlights a minimally invasive and cost-effective alternative to traditional tissue biopsies, offering a new pathway toward personalized cancer treatment.
Study Design and Patient Population
Led by Justin Balko, professor of Medicine and Pathology, Microbiology and Immunology at Vanderbilt Health, the research analyzed 546 blood samples from 160 patients with stage 2 and 3 breast cancers that are negative for human epidermal growth factor receptor 2 (HER2 (search)). Patients were undergoing treatment with chemotherapy alone or in combination with immunotherapy.
The study was conducted in collaboration with investigators from the I-SPY2 clinical trial, a nationwide initiative exploring targeted therapies based on cancer biomarkers. Co-author Laura Esserman, director of the Breast Care Center at the University of California, San Francisco, serves as the principal investigator of the trial.
Molecular Analysis and Key Findings
The team conducted RNA sequencing to study gene expression linked to the activation of T cells (search)—key components of the body's antitumor immune response. Findings revealed that these molecular signals in blood samples could effectively predict patient response to the immunotherapy drug Pembrolizumab.
The liquid biopsy approach allows clinicians to monitor immune response in real time and adjust therapies accordingly. By analyzing blood samples taken over time, researchers could monitor the changes in the immune response as the therapy progressed, offering a non-invasive way to gain insights into how well a patient is responding to treatment.
Clinical Implications and Future Applications
Researchers emphasized that combining molecular profiles from tissue samples with circulating tumor DNA and transcriptome data could significantly enhance treatment precision. The study underscores the growing importance of non-invasive diagnostics in oncology, paving the way for more adaptive, patient-centric cancer care.
While further validation is needed, the findings suggest this approach could guide immunotherapy decisions, enable personalized treatment strategies, and advance precision oncology across multiple cancer types. "This method offers an accessible tool for tailoring treatment strategies in breast cancer (search)," the researchers noted, adding that it may also be applicable to other solid tumors.
The study highlights the need for new biomarkers to enhance the effectiveness of immunotherapy, which, despite becoming a standard treatment, has shown limited success in shrinking tumors. This approach offers potential for more tailored and effective therapeutic strategies in breast cancer (search) treatment.