Key Points
- ASCO 2023 featured updates from an ongoing brain cancer liquid biopsy trial and a pediatric sonodynamic therapy trial.
- Nina Theresa Yoh, MD, received a prestigious Young Investigator Award for her work using focused ultrasound to treat childhood brain tumors.
The annual meeting of the American Society of Clinical Oncology (ASCO) was held June 2–6, 2023, in Chicago. In the brain cancer sessions, there were two oral presentations describing ongoing studies using focused ultrasound for brain tumors. One trial is investigating focused ultrasound–enhanced liquid biopsy for glioblastoma (GBM) in adults, and the other presented information on an ongoing sonodynamic trial for DIPG.
On behalf of the clinical trial’s 10 enrollment sites and investigators, neuro-oncologist Manmeet Ahluwalia, MD, MBA, FASCO, the chief scientific officer, chief of medical oncology, deputy director and Fernandez family endowed chair at Baptist Health Miami Cancer Institute, presented the methodology as well as primary and secondary aims from Insightec’s LIBERATE study. LIBERATE stands for “Liquid Biopsy with Low Intensity Ultrasound in Brain Tumors.” The poster (#TPS2084) was titled Low-intensity focused ultrasound with systemic microbubble oscillators for blood-brain barrier disruption for liquid biopsy in glioblastoma (LIBERATE). The poster was presented in the Central Nervous System Tumors session as part of the Central Nervous System Tumors track and Primary CNS Tumors – Glioma subtrack.
“LIBERATE has now enrolled at least 17 of 57 participants with GBM,” said Dr. Ahluwalia. “We plan to present early data at the European Association of Neuro-Oncology (EANO) meeting in September.”
When asked about why he is doing this study, Dr. Ahluwalia said, “Liquid biopsies have been transformational in altering the standard of care in other cancers like lung cancer and how research has been conducted…but one of the stumbling blocks for brain tumors has been an inability to reliably capture cell-free DNA in the blood with a high sensitivity – that is where the power of focused ultrasound technology lies.”
Dr. Ahluwalia continued, “There is just so much that needs to be done for patients with brain tumors. These are normal people with no risk factors. These patients are in the prime of their life, in their 40s, 50s, and early 60s. Life is going great until the day they get the diagnosis, the biggest shock of their life – and their family’s life. I will be thrilled if I can do anything to help move the needle for these patients.”
Enrollment sites for the LIBERATE clinical trial, which is funded by Insightec, currently include:
- University of California, San Francisco (San Francisco, CA)
- Miami Cancer Institute at Baptist Health (Miami, FL)
- University of Maryland (Baltimore, MD)
- Johns Hopkins University (Baltimore, MD)
- Mayo Clinic (Rochester, MN)
- NYU Grossman School of Medicine (New York, NY)
- University of Texas, Southwestern (Dallas, TX)
- University of Texas MD Anderson Cancer Center (Houston, TX)
- West Virginia University Rockefeller Neuroscience Center (Morgantown, WV)
- Sunnybrook Research Institute (Toronto, Ontario, Canada)
To describe progress to date for the diffuse intrinsic pontine glioma (DIPG) sonodynamic therapy clinical trial at Children’s National Hospital in Washington, DC (Abstract # TPS10070), Hasan Syed, MD, displayed a poster, Phase 1/2, first-in-child study of sonodynamic therapy (SDT) using low intensity focused ultrasound and 5-aminolevulinic acid (ALA) for patients with diffuse intrinsic pontine glioma. The first cohort has completed enrollment for this DIPG study, which also uses the Insightec Exablate Type 2 device. Beyond evaluating safety and tolerability, the investigators are seeking to determine the maximum tolerated dose of the focused ultrasound energy plus ALA. Patients at least 5 years of age with newly diagnosed, radiographically typical DIPG who have completed 4 to 24 weeks of standard radiotherapy are eligible for the trial. The poster was presented in the Pediatric Oncology session as part of the Pediatric Oncology track and Pediatric Solid Tumors subtrack.
Nina Theresa Yoh, MD, a neurosurgery resident at Columbia University Irving Medical Center, received a prestigious 2023 Conquer Cancer Sontag Foundation Young Investigator Award for her work using focused ultrasound to treat childhood brain tumors. With mentorship provided by Cheng-Chia Wu, MD, PhD, and Elisa Konofagou, PhD, Dr. Yoh is developing focused ultrasound liquid biopsy to diagnose and monitor diffuse midline gliomas.
The Foundation thanks Dr. Ahluwalia and Ahmad Ozair, MBBS, postdoctoral research fellow in neuro-oncology at the Miami Cancer Institute at Baptist Health, for assistance with this meeting report.