Key Points
- Preliminary data from a clinical trial using focused ultrasound to address deadly pediatric brain tumors have been presented.
- Most participants experienced tumor reduction or stable disease, and no serious adverse events were reported.
Preliminary data from the first participants enrolled in the clinical trial using focused ultrasound to address deadly pediatric brain tumors called diffuse intrinsic pontine gliomas (DIPG) were presented at the 28th Annual Meeting and Education Day of the Society for Neuro-Oncology, which was held November 15–19, 2023 in Vancouver, Canada.
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 is investigating the safety and efficacy of using sonodynamic therapy (SDT) to target and destroy brain tumor tissue. With SDT, focused ultrasound is used to activate a chemical substance that then causes cell death only within the targeted region. The noninvasive drug-device combination used in this trial is SonALAsense’s proprietary aminolevulinic acid HCl (ALA) formulation, called SONALA-001, in combination with Insightec’s Exablate Neuro focused ultrasound device.
According to SonALAsense’s press release, of the first five participants, two experienced at least a 25% reduction in tumor size through eight weeks post treatment, and two other participants experienced stable disease. Four participants have exceeded the median survival of 9–11 months post diagnosis. There have been no dose limiting toxicities or grade 3 adverse events related to the treatment.
“We are pleased with the early results and the safety profile of SONALA-001 SDT,” said Hasan R. Syed, MD, pediatric neurosurgeon at Children’s National Hospital who is co-leading the trial along with principal investigator Roger Packer, MD. “Sonodynamic therapy is part of a larger trend toward less invasive treatment paradigms in neurosurgery. For families of children with DIPG, this trial offers hope and a chance to potentially alter the course of this aggressive disease.”
The US Food and Drug Administration (FDA) recently granted Fast Track Designation for the development of this SDT therapy for the treatment of patients with DIPGs.
Read SonALAsense’s Press Release
For Patients
If you are interested in learning more about this clinical trial, please contact one of the two sites below.
University of California, San Francisco
San Francisco, California, USA
Contact: Karina Wong at 415-298-9434 or Karina.Wong@ucsf.edu
Contact: Marina Khamhaengwong at 415-476-1823 or Marina.Khamhaengwong@ucsf.edu
Children’s National Hospital/Children’s Research Institute
Washington, District of Columbia, USA
Contact: Elizabeth Paronett at eparonett@childrensnational.org