Key Points
- Insightec has launched a clinical trial assessing focused ultrasound to temporarily disrupt the blood-brain barrier to enable liquid biopsies in patients with glioblastoma.
- Liquid biopsies are an emerging method of analyzing the molecular profile of tumors through a simple blood test.
- Researchers are investigating whether focused ultrasound can enhance the levels of DNA biomarkers circulating in the bloodstream.
An international, multisite clinical trial is assessing the safety and efficacy of using Insightec’s focused ultrasound platform to temporarily disrupt the blood-brain barrier (BBB) to enable liquid biopsies in patients with glioblastoma (GBM).
GBMs are the most common primary malignant brain tumors in adults, and they have devastating prognoses. These tumors are difficult to treat because conventional biopsies may not reflect the tumor heterogeneity, contributing to the high resistance to many treatments. In addition, if they can even be performed due to the location of the tumor, conventional biopsies are invasive, need to be performed in a sterile operating suite, and carry a risk of bleeding, infection, and neurologic deficits. Therefore, liquid biopsies could represent a noninvasive platform to better define the heterogeneity of glioblastomas, monitor response and resistance to treatment, and direct targeted therapies.
Liquid biopsies analyze the molecular profile of tumors through a simple blood test. Many solid tumors throughout the body (excluding the brain) shed biomarkers that circulate throughout the bloodstream and can be detected in liquid biopsies. However, brain tumors present a challenge for the liquid biopsy techniques due to the BBB, a protective layer of tightly joined cells that line the blood vessels in the brain. The BBB typically prevents harmful substances from entering the brain and, unfortunately, also prevents medications from reaching brain tumors. Importantly, the BBB also inhibits the movement of tumor markers from the brain into the bloodstream.
In this study – called LIBERATE – for “Liquid Biopsy with Low Intensity Ultrasound in Brain Tumors,” blood samples will be collected before and after focused ultrasound is used to disrupt the BBB. Researchers will investigate whether the focused ultrasound can increase the amount of circulating cell-free DNA (cfDNA) and whether there are mutational profiles in the released cfDNA, specific for the brain tumor.
This trial builds on preliminary studies that have shown that focused ultrasound can be used to amplify the presence of brain tumor biomarkers, making them easier to identify and measure in the bloodstream.
“Liquid biopsies are a cutting-edge tool for the future of cancer diagnosis and care,” said Suzanne LeBlang, MD, the Foundation’s director of clinical relationships. “The information we could glean from a simple blood draw could potentially be used to make an initial diagnosis, guide precision therapies, monitor treatment response, and provide longitudinal follow-up for possible recurrence. We are excited about this study and to learn more about the role focused ultrasound can play in this developing field.”
The first participant in the trial was enrolled at the Mayo Clinic in Rochester, Minnesota. Other sites taking part in the trial include the University of Maryland, NYU Grossman School of Medicine, and Sunnybrook Health Sciences Centre in Toronto. Additional sites in Florida and Maryland are expected to begin enrolling soon.
Insightec is sponsoring this clinical trial.
For Patients
To learn more about the clinical trial, visit clinicaltrials.gov or contact the site nearest you.
University of Maryland, Baltimore & The University of Maryland Medical System
Baltimore, Maryland, United States
Contact: Kaitlyn Henry (410-328-0939, khenry@som.umaryland.edu)
Contact: Charlie Klontz (410-328-0939, CMKlontz@som.umaryland.edu)
Mayo Clinic
Rochester, Minnesota, United States
Contact: Adam Loudermilk (507-422-0718, loudermilk.adam@mayo.edu)
NYU Grossman School of Medicine
New York, New York, United States
Contact: Cathryn Lapierre (646-501-2762, cathryn.lapierre@nyulangone.org)
Contact: Shannon Ciprut (212-263-4076, shannon.ciprut@nyulangone.org)
Sunnybrook Health Sciences Centre
Toronto, Ontario, Canada
Contact: Maheleth Llinas (416-480-6100 ext 2476, maheleth.llinas@sunnybrook.ca)