New Trial of Focused Ultrasound for Pediatric Brain Cancer


Key Points

  • Researchers at Children’s National Hospital have begun a clinical trial to address deadly pediatric brain tumors called diffuse intrinsic pontine gliomas (DIPG).
  • The trial will use focused ultrasound—induced sonodynamic therapy to kill tumor cells.
  • Another clinical trial for DIPG using focused ultrasound to disrupt the blood-brain barrier is being planned for later this year at Children’s National.

Researchers at Children’s National Hospital in Washington, DC, have begun a clinical trial to address deadly pediatric brain tumors called diffuse intrinsic pontine gliomas (DIPG).

DIPGs are highly aggressive and devastating brain tumors that affect young children, typically aged 5–9. Only 10 percent of children survive past the two-year mark after diagnosis because current therapies are highly ineffective.

This clinical trial will investigate the safety and efficacy of using sonodynamic therapy (SDT) to target and destroy brain tumor tissue. SDT involves using focused ultrasound to activate a chemical substance that then causes cell death only within the tumor. The noninvasive drug-device combination that will be used in this trial was developed by SonALAsense in collaboration with Insightec.

Roger Packer, MD, senior vice president of the Center for Neurosciences and Behavioral Medicine at Children’s National, is leading the study.

“Focused ultrasound gives us a way to potentially transiently open up the barrier, so we can deliver novel therapy directly to the tumor and improve the likelihood of survival,” said Dr. Packer. “It is the greatest breakthrough we’ve potentially had in the past 50 years or more for the management of these tumors. We made great strides in our understanding of molecular genetics and the molecular drivers of tumors, but we have not yet translated that knowledge into better therapies; this may be our most effective mechanism to overcome the barrier.”

Researchers aim to enroll 18 patients in the trial. Two additional sites in the US are planned to join the study but are not yet enrolling.

A similar study for adult patients is ongoing in Phoenix, Arizona.

Upcoming Trial Will Take Alternate Approach
Another clinical trial is being developed by the team at Children’s National to address DIPG by disrupting the blood-brain barrier (BBB).

The BBB is a protective layer of tightly joined cells that lines the blood vessels in the brain and prevents harmful substances, such as toxins and infectious agents, from diffusing into the surrounding brain tissue. It can also prevent therapeutic agents from getting into the brain, which is why research has centered on using focused ultrasound to safely and temporarily disrupt this barrier.

This trial is expected to begin later this year, and we will report on updates as they become available.

“Focused ultrasound could have a tremendous impact on young patients suffering from a range of conditions,” said the Foundation’s Associate Chief Medical Officer, Lauren Powlovich, MD. “The noninvasive nature of this technology makes it especially attractive as a treatment option for the pediatric population as it has fewer side effects than more traditional treatments. The Foundation is closely watching how the technology could impact devastating neurological diseases like DIPG, and we are planning to fund research in this area.”

In 2020, Children’s National was designated the Foundation’s first pediatric Center of Excellence. Last year, the Foundation hosted a DIPG Workshop, and 22 organizations – including the Children’s National team – participated to develop a roadmap for how focused ultrasound can impact these patients.

For Patients
If you are interested in learning more about the SDT trial at Children’s National, please contact Sophie Kopec at