Key Points
- The half-day virtual event addressed challenges, opportunities, and the role of focused ultrasound in the field.
- A white paper summarizing the event will be available soon.
News — On March 27, 2025, the Foundation hosted a virtual roundtable discussion on the topic of focused ultrasound and cell therapy for brain tumors. Thirty-eight people attended the half-day event.
The program was divided into two sessions. The first session reviewed challenges and opportunities associated with gene therapy for brain tumors and was moderated by Hideho Okada, MD, PhD. This session featured the following speakers and topics:
- Neuroimmunology and Cell Therapies by Richard Ransohoff, MD
- Central Nervous System (CNS) Inflammation and Lymphocytes by Robyn Klein MD, PhD
- Clinical Trial Design Considerations by John de Groot, MD
The second session addressed the role of focused ultrasound in the field and was moderated by Natasha Sheybani, PhD. The topics covered in this session included:
- Ultrasound-Based Blood-Brain Barrier Opening in Humans: Pharmacokinetics and Immune Modulation by Adam Sonabend, MD
- Focused Ultrasound Combined with Immunotherapy: A New Horizon in Targeting CNS Malignancies by Marco Gallus, MD
- Focused Ultrasound-Mediated Control of Immune Cell Trafficking and Function by Costas Arvanitis, PhD
“This roundtable discussion helped galvanize the focused ultrasound community around the challenges of gene and cell therapy and built on relationships that began at our ,” said Frédéric Padilla, PhD, the Foundation’s director of the Gene and Cell Therapy Program. “Our overarching mission at the Foundation is to expedite patient access to innovative focused ultrasound treatments. This event will help advance the field toward clinical treatments by informing our strategic funding decisions based on current opportunities and key roadblocks.”
Both sessions concluded with an open discussion among attendees. The group explored the potential of focused ultrasound to enhance cellular therapies for brain tumors, particularly T-cells and CAR-T cells. They discussed various delivery methods—including intravenous, intraventricular and intra-cavitary administration—and the challenges of crossing the blood-brain barrier. The importance of understanding CNS immunity was emphasized, including the fate of administered cells and the role of resident memory T-cells. Discussions also touched on clinical trial design, the potential for window-of-opportunity trials, and the challenge of identifying biomarkers to assess the persistence and function of administered cells.
Dr. Padilla added, “I’d like to thank our steering committee members—Costas Arvanitis, PhD, Catherine Bollard, MD, Marco Gallus, MD, Hideho Okada, MD, PhD, and Bob Smith—for their guidance and support in shaping this event.”
A white paper summarizing the meeting and its outcomes will be available soon.
RSS