Research And Grants

A Phase 1/2 Study of Sonodynamic Therapy Using SONALA-001 and Exablate 4000 Type 2 in Patients With DIPG

Diffuse intrinsic pontine glioma (DIPG) is a lethal pediatric brain tumor with no effective therapy and extremely poor prognosis (average 9 months of survival after diagnosis). Because of the location of DIPG tumors within the brainstem, surgical intervention is a highly invasive and imprecise option, as accidental removal of any excess tissue in the area can cause additional brain damage. Thus, there is significant need for a method to effectively treat DIPG that is non-invasive. Preclinical studies have shown that performing non-invasive sonodynamic therapy (SDT) through MRI-Guided Focused Ultrasound (MRgFUS) combined with 5-aminolevulinic acid (ALA) can slow growth of gliomas and greatly extend survival in animal models of glioma. ALA is a metabolite, or a molecule used in metabolic processes. Because cancer cells have metabolic rates significantly higher than healthy cells, ALA is selectively taken up by cancer cells at a rate much higher than normal cells. Within tumor cells, ALA is then converted into a molecule, Protoporphyrin IX (PpIX), which causes cell death when activated by blue light – this process is called Photodynamic Therapy. SonALAsense’s SDT uses the energy of ultrasound to produce light within the brain to activate PpIX, selectively killing tumor cells.

We have shown that ALA combined with SDT (or ALA SDT) can safely and effectively kill tumor cells in a first-in-man a Phase 0/1 clinical trial at the Ivy Brain Tumor Center in recurrent high-grade glioma patients (NCT04559685) and decrease DIPG cell viability in cell cultures through a study with Dr. Javad Nazarian the DMG Research Center at University Children’s Hospital, Zurich, Switzerland. With this promising data, SonALAsense will test the combination therapy of MRgFUS and ALA in a three center, Phase 1/2 clinical trial that will establish dosing and safety for future clinical trials to improve the outcomes and survival in DIPG patients.