A novel ultrasound device has shown promising results in a groundbreaking human trial, demonstrating its ability to temporarily open the blood-brain barrier (BBB) to directly deliver chemotherapy to the brain for glioblastoma treatment. This procedure, performed with patients awake, resulted in up to a six-fold increase in drug concentrations in the brain, marking a significant advancement since current treatments struggle to penetrate the BBB. Conducted by Northwestern Medicine, the trial utilized a skull-implantable device that, in a four-minute session, allowed for repeated administration of chemotherapy drugs such as paclitaxel and carboplatin, which traditionally cannot reach brain tumors. Researchers discovered that most BBB restoration occurs within 30 to 60 minutes post-sonication, creating a critical window for drug efficacy. The study represents the first quantification of ultrasound-assisted BBB opening in humans, enhancing the delivery of previously ineffective drugs for glioblastoma. Following the positive outcomes of this phase 1 trial, a phase 2 trial is underway to evaluate the combination treatment’s impact on patient survival. Dr. Adam Sonabend, lead investigator, noted that this technique could pave the way for exploring drug treatments for various brain diseases beyond cancer. The findings were published in The Lancet Oncology.
