Pioneering Ultrasound Treatment for Deadly Brain Tumors in Children

SebastianStrong Foundation Gift Fuels Research on Ultrasound for Hard-to-treat Childhood Brain Tumors

Children with deadly, hard-to-treat brain tumors have no time to wait for the traditional pace of scientific progress. They need help today. Focused ultrasound shows promise for treating tumors non-invasively with sound waves.

A $672,000 gift from the SebastianStrong Foundation (SSF) aims to push this field forward — faster. It will enable a new research consortium to test ultrasound treatments for one of the deadliest forms of brain cancer— diffuse midline glioma (DMG). Members include:

• Children’s National Hospital Brain Tumor Institute
• Virginia Tech Fralin Biomedical Research Institute
• Columbia University Irving Medical Center

“Kids deserve so much better,” says Oscar Ortiz, who founded SSF in 2017 in memory of his son Sebastian, who died of cancer. “Collaboration between three institutions will accelerate the desired results.” This gift represents SSF’s largest investment to date.

DMG tumors typically begin in the brainstem. This makes surgery or chemotherapy difficult. Radiation therapy can prolong survival. However, the tumor eventually grows back, leading to death.

Focused ultrasound provides hope. “We have discovered promising drugs for DMG. The problem is drug distribution across the blood brain barrier,” says Javad Nazarian, Ph.D., M.Sc., our Brain Tumor Institute’s scientific director. “Focused ultrasound is a promising platform. We can effectively open the blood brain barrier for a short period of time.” In addition, focused ultrasound can facilitate targeted treatments such as immunotherapy.

The SSF gift will assemble a dream team of experts to test these possibilities. In addition to Dr. Nazarian, the team includes:

• Jennifer Munson, Ph.D., of Virginia Tech, a bioengineer with expertise in tissue engineering of 3D cultures for brain tumors
• Eli Vlaisavljevich, Ph.D., of Virginia Tech, a designer and developer of focused ultrasound devices
• Cheng-Chia Wu, M.D., Ph.D., of Columbia University, principal investigator for the world’s first clinical trial using focused ultrasound in children with relapsed DMG

Much of the research will occur at the new Children’s National Research & Innovation Campus, where both Virginia Tech and Children’s National have laboratories.

“Bringing this expertise together will advance this promising platform for treatment of pediatric brain tumors,” says Dr. Nazarian. “Individually, we progress slowly, or we may fail. Together we will be able to better understand how to treat these tumors and accelerate their delivery to the clinic.”

The team will prioritize rapid translation of its findings through the Pacific Pediatric Neuro-Oncology Consortium (PNOC). Over the last two years, Dr. Nazarian and his colleagues have launched clinical trials for two novel therapies for DMG within this network.