MORGANTOWN, W.Va. – The WVU Rockefeller Neuroscience Institute (RNI) is advancing the treatment of high-grade gliomas, one of the most aggressive and challenging forms of brain cancer. A multicenter clinical trial, now published in The Lancet Oncology, demonstrates that microbubble-enhanced transcranial focused ultrasound (MB-FUS) can safely and temporarily open the blood–brain barrier (BBB), allowing standard chemotherapy to reach tumor tissue more effectively.
The research was conducted across leading North American academic medical centers, including the RNI and collaborators at the WVU Cancer Institute. Together, these teams bring nationally recognized expertise in Neurosurgery, Neuro-Oncology, Neuroradiology and clinical trials to advance care for patients with complex brain tumors.
Christopher P. Cifarelli, M.D., Ph.D., director of the WVU Comprehensive Brain Tumor Program, served as the site principal investigator on the trial.
“These early outcomes offer real promise for patients facing one of the most challenging cancers in neurosurgery,” Dr. Cifarelli said. “Focused ultrasound may give us a new pathway to deliver chemotherapy directly where it’s needed, potentially improving treatment effectiveness while minimizing systemic exposure.”
The trial enrolled 34 participants and used MRI-guided, low-intensity focused ultrasound to target peritumoral regions — areas where invasive tumor cells often remain shielded behind an intact blood–brain barrier.
Across all treatments, researchers consistently achieved successful and well-controlled opening of the barrier, with most reported effects mild and temporary. Early signals also suggest improved progression-free and overall survival when compared with external matched controls.
The study further outlines potential for a “sono-liquid biopsy” approach, in which plasma biomarkers enhanced by MB-FUS may offer a non-invasive method for monitoring tumor behavior and response to therapy.
Ali Rezai, M.D., executive chair of the RNI and co-author of the study, is a pioneer in focused ultrasound and blood–brain barrier opening innovation.
“This collaborative effort represents a significant advance to improve the targeted delivery of chemotherapy to brain tumors,” Dr. Rezai said. “The blood-brain barrier is a major obstacle for delivery of drugs to the brain as more than 98 percent of drugs have limitations in crossing the BBB. Focused ultrasound technology allows for a safe, targeted and reproducible opening of the BBB to allow for enhanced drug delivery to the brain.”
This publication reinforces WVU Medicine‘s growing leadership in advancing innovative therapies for brain cancer — driven by the close collaboration between the RNI, the Cancer Institute and national collaborators.
“The WVU Cancer Institute continues to engage in cutting-edge research that translates to expanded treatment options and enhances patient care in West Virginia and beyond,” Hannah Hazard-Jenkins, M.D., executive chair and director of the WVU Cancer Institute, said.
“These types of studies further reinforce our commitment to innovative research, treatment and patient care. We can do this through collaboration across a vast spectrum of expertise that allows us to further important research and clinical care to provide patients with treatment options under the care of compassionate healthcare teams,” Hazard-Jenkins said.
For more information on the WVU Rockefeller Neuroscience Institute, visit WVUMedicine.org/RNI.
