A unique clinical trial approach may lead to more effective personalized treatments for patients with breast cancer.

The I-SPY 2 trial is a multicenter phase 2 platform trial that evaluates novel neoadjuvant (chemotherapy given before surgery) therapy for early stage breast cancer at high risk of recurrence. A traditional clinical trial investigates one therapy for one disease. In contrast, a platform trial evaluates multiple new drugs in combination with chemotherapy in parallel to figure out which therapies work best.

The I-SPY 2 trial is also an adaptive study, which allows patient results to be monitored in real time so that therapies can be refined and inform the care of new patients. This allows for quicker results with fewer patients than traditional trials.

“About 25% of patients with early stage breast cancer who receive neoadjuvant chemotherapy will experience a recurrence within five years. The goal of the I-SPY 2 trial is to improve the care of early stage breast cancer patients by finding new effective therapies,” said Dr. Heather Han, a medical oncologist in the Department of Breast Oncology at Moffitt Cancer Center. Han is also an investigator on the I-SPY 2 trial.

New results published in JAMA Oncology show that giving the immune checkpoint inhibitor pembrolizumab with standard neoadjuvant chemotherapy more than doubled the pathological complete response in patients with both hormone receptor-positive/HER2-negative and triple-negative breast cancers. Pathological complete response refers to the absence of invasive cancer in breast and lymph nodes at the time of surgery following neoadjuvant therapy.

“This has been also validated in a large randomized phase 3 trial for patients with triple-negative breast cancer published in The New England Journal of Medicine.  These results confirm that addition of immunotherapy to neoadjuvant chemotherapy is definitely a promising approach and it could lead to a new FDA-approved therapy for these patients,” said Han.