Key Points
- In October 2021, the Foundation hosted a workshop to explore the use of focused ultrasound to treat DIPG, a fatal pediatric brain tumor.
- The presentations and discussions have now been summarized in a white paper.
- Workshop videos are available on the Foundation’s YouTube channel.
On Wednesday, October 27, the Foundation hosted a virtual workshop on focused ultrasound for diffuse intrinsic pontine glioma (DIPG).
DIPGs are highly aggressive and devastating brain tumors that affect young children, typically aged 5-9. Only 10 percent of children survive two years after diagnosis, because the current therapies are highly ineffective.
A synopsis of the prerecorded presentations and live panel discussions from the four-hour virtual workshop is now available in the workshop’s white paper, and the video recordings are available on YouTube. The virtual meeting convened 50 leaders in the field, including oncologists, radiologists, pediatricians, immunologists, focused ultrasound experts, and representatives from industry, the Foundation, and patient advocacy organizations.
The objectives of the workshop were to provide an overview of current knowledge, identify gaps in knowledge, and outline best practices for studying using focused ultrasound to address this devastating disease.
“DIPG occurs in a deep area of the brain where surgical resection is not an option; however, focused ultrasound can reach these areas to increase targeted drug delivery,” said Suzanne LeBlang, MD, the Foundation’s Director of Clinical Relationships. “With an innovative clinical trial already underway at Columbia University and several other centers to soon begin additional trials, we have seen immense progress in this critical indication, but there is more work to be done.”
The group built the following roadmap for moving forward:
- Preclinical researchers will form collaborations, develop a data repository, create a centralized histopathology resource, and share data, biospecimens, and experiences.
- Future preclinical studies will address the heterogeneity of DIPG tumors, determine the degree to which the BBB must be opened, devise ways to expand the treatment envelope, test the pharmacokinetics of various agents to improve the translation of preclinical studies to effective clinical trials, develop microanalyses for assessing drug penetrates, and correlate preclinical data with clinical studies.
- Industry representatives will seek to advance focused ultrasound devices in ways that improve pediatric focused ultrasound treatments in the brain. These advances may include designing frameless systems that do not require head shaving, providing spot control for BBB opening, expanding the treatment envelope, introducing a radiolabeled drug with focal BBB opening, and innovating treatments that could be conducted outside the MRI environment.
- Clinical researchers will develop collaborations with neuropharmacologists, ensure that all patients have access to clinical trials, improve measures to determine the specificity and sensitivity of drugs that are delivered across the BBB, and harmonize clinical trial protocols.
- The Foundation will convene a DIPG working group, continue to develop and implement the ideas suggested in the roadmap, and fund clinical trials to advance focused ultrasound for the treatment of this devastating disease.
“I am thrilled with the turnout and outcomes of this very important workshop,” said the Foundation’s Associate Chief Medical Officer, Lauren Powlovich, MD. “We now have a clear path forward to advance the field in this area, including the creation of preclinical and clinical working groups to enhance collaboration and information sharing amongst focused ultrasound DIPG researchers. DIPG is a devastating cancer, and I am hopeful that focused ultrasound will one day lessen the burden of this disease on pediatric patients and their families.”
Dr. Powlovich added, “We have already launched the preclinical and clinical working groups to enhance collaboration and information sharing amongst focused ultrasound DIPG researchers. As new clinical trials are approved, we will take an active role in funding and supporting them.”
The following educational content was provided to attendees ahead of the meeting:
- Noninvasive Focused Ultrasound and Panobinostat for Children by Stergios Zacharoulis, MD
- Repeated 5 Aminolevulinic Acid mediated Sonodynamic Therapy in Rat Brain Tumor Models by Sheng Kai Wu, PhD
- Current State of the Field of Preclinical FUS DIPG Research by Javad Nazarian, PhD, and Mehmet Ozdas, PhD
- Focused Ultrasound–Enhanced Drug Delivery for DIPG by Hong Chen, PhD
- Immune Modulation and Immunotherapy for DMG DIPG by Hideho Okada, MD, PhD
- Technology Requirements for the Pediatric Patient by Elisa Konofagou, PhD
- Sonodynamic Therapy for DIPG by Stuart Marcus, MD, PhD
- Liquid Biopsy for DIPG by Carl Koschmann, MD
A recording of the workshop is now available on the Foundation’s YouTube channel.