Key Points
- The Foundation partnered with the Sontag Foundation to host a workshop on focused ultrasound–induced blood-brain barrier opening for glioblastoma.
- Attendees addressed 10 burning questions related to the field, including the potential roles of drug delivery and liquid biopsy.
The Focused Ultrasound Foundation recently partnered with the Sontag Foundation to host a two-day workshop on focused ultrasound–induced blood-brain barrier opening (FUS BBBO) for glioblastoma (GBM). More than 30 researchers, clinicians, industry representatives, government employees, and others attended the meeting in Arlington, Virginia, March 6–7, 2024.
The group participated in panel discussions, each centered around a burning question pertaining to the field of FUS BBBO for GBM. The first day of the workshop explored drug delivery while the second day included panels on using FUS BBBO to assist in liquid biopsy.
The 10 burning questions discussed were:
- Which FUS BBBO parameters should be reported/aligned to unify language and allow for comparison across devices and enable more effective communication between researchers, industry, and clinicians?
- How do we measure BBBO and therapy delivery beyond gadolinium enhancement?
- How can we optimize efficacy and synergy by combining therapeutics with FUS BBBO parameters?
- What new therapeutics should be tested for combination with FUS BBBO? Which type would you predict would be most successful and why? Which will have high potential in future to impact outcomes?
- What FUS BBBO–related toxicities should be defined, monitored, and reported?
- Which clinical settings would be most appropriate to test safety and/or effectiveness of FUS BBBO? Which situation would provide more data regarding therapeutic effects?
- What biomarkers should be collected to understand the biology of what is happening during FUS BBBO?
- What analytes should be obtained that may help us understand tumor biology and possibly determine response, recurrence, or residual active disease? Should these analytes be tailored to the specific drug mechanisms of action to measure downstream effects?
- How should we study the tumor barcode? High level or specific mutations?
- What are the key clinical needs that could be addressed with liquid biopsy, and how does this fit with clinical workflow as opposed to other methods such as cerebrospinal fluid sampling without focused ultrasound?
“The passion among the multidisciplinary group of experts was clear in wanting to engage focused ultrasound as a potential new therapeutic platform to help patients with GBMs,” said Suzanne LeBlang, MD, the Foundation’s director of clinical relationships. “The attendees engaged in thoughtful and interactive discussions while answering the burning questions. Ideas quickly germinated from these discussions on how to move the field forward in a meaningful and expeditious manner. We are grateful to the Sontag Foundation for helping to support such an important meeting.”
Established in 2002, the Sontag Foundation is one the largest private funders of brain cancer research in North America. Frederick and Susan Sontag founded the organization following Susan’s diagnosis with a lethal form of brain cancer and, to date, the Sontag Foundation has granted over $65 million in funding to support brain cancer research.
Several important action items taken from the meeting include:
- Form a working group to publish a guidelines paper on reporting standards for FUS-enhanced liquid biopsy.
- Develop a one- to two-year roadmap of projects that address the burning questions and would help achieve regulatory approval and reimbursement of FUS BBBO for GBM.
- Promote continued collaborations that were formed at the workshop and facilitate the advancement of FUS BBBO for GBM as rapidly and safely as possible
A white paper documenting the workshop’s discussion and results will be made available soon.