Key Points
- Researchers in Taiwan completed an early-stage trial showing focused ultrasound–mediated blood-brain barrier opening plus chemotherapy is safe in patients with GBM.
- The investigators are now recruiting participants for a larger clinical trial with the same device – the NaviFUS system – at two sites.
Results from a pilot clinical trial evaluating the safety and feasibility of repeated focused ultrasound–induced blood–brain barrier (BBB) opening combined with the monoclonal antibody drug Avastin (bevacizumab) in patients with recurrent glioblastoma were published in Neurosurgery. With the success of the pilot, the study’s next phase – the pivotal trial – is now enrolling participants.
The prospective, single-arm pilot study (NCT04446416) enrolled six participants who received biweekly BBB opening treatments plus standard-of-care Avastin using the NaviFUS low-frequency focused ultrasound device. As a monoclonal antibody, Avastin binds to the glioblastoma cells that form new blood vessels, slowing their growth. The NaviFUS device uses real-time neuronavigation guidance to direct focused ultrasound energy precisely and noninvasively through the skull to select brain tissues.
Participants were followed to assess progression using standardized radiologic criteria. The median progression-free survival was 11 months, with a 6-month progression-free survival rate of 66.7%, and treatment was well tolerated, with only transient, low-grade scalp heating reported.
Researchers also analyzed the participants’ blood samples for biomarkers and found significant post-treatment increases in total and tumor-related cell-free DNA (cfDNA). This means that opening the BBB allowed the DNA to move from the brain into the blood stream and confirms findings from previous clinical studies (Meng 2021, Leuthardt 2023). It has also been suggested that cfDNA levels may serve as a surrogate to measure and confirm BBB opening.
The authors concluded that repeated BBB opening with Avastin is safe and feasible. They support further randomized studies to determine its therapeutic benefit and to validate the associated imaging and liquid biopsy biomarker data.
The trial took place at Linkou Chang Gung Memorial Hospital in Taiwan and was led by neurosurgeon Kuo-Chen Wei, MD. The Foundation funded part of this clinical study along with the National Science Technology Council, the National Health Research Institutes, and NaviFUS.
Next Steps
NaviFUS is now expanding upon these positive results with a new multicenter phase III clinical trial (NCT06496971). Two sites in Taiwan are currently recruiting adults with recurrent glioblastoma to assess the safety and efficacy of Avastin with and without focused ultrasound–enhanced BBB opening.
This randomized, controlled study will divide up to 32 participants into two cohorts. One group will receive Avastin alone every two weeks as the current standard of care. The other group will receive Avastin followed by focused ultrasound at specified intervals.
For Patients
To learn more about the pivotal study, visit the trial page or contact
Sheang-Tze Fung, PhD
02-25860560 ext 167
[email protected]
Arthur Lung, PhD
[email protected]
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