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SonALAsense Forges Ahead With First Patient Dosing for Devastating Childhood Brain Cancer

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Diffuse intrinsic pontine glioma (DIPG) is a rare and deadly pediatric brain cancer with a 2% five-year survival rate. To provide therapeutic options for patients and families, SonALAsense is partnering with clinicians at Children’s National Hospital, Ivy Brain Tumor Center at Barrow Neurological Institute, Phoenix Children’s Hospital and University of California San Francisco (UCSF) Medical Center to conduct a phase 1/2 clinical trial for sonodynamic therapy (SDT). This is the first SDT trial for children.

Roger Packer, M.D., co-leads the Children’s National clinical trial site with Lindsay Kilburn, M.D., and Hasan Syed, M.D., who treated the study’s first patient, a 5-year-old girl, two weeks ago. The trial will assess safety, overall patient health and tumor size.

“DIPG is a relentless and sadly fatal disease afflicting children, and there’s currently very little we can do to stop it,” said Dr. Packer, who directs the Brain Tumor Institute at Children’s National. “Most children survive less than a year. Sonodynamic therapy offers us an innovative and promising, noninvasive way to directly target the tumors in these children.”

“With sonodynamic therapy, we are using focused ultrasound to activate SonALAsense’s drug candidate, which is administered to the patient, and selectively killing tumor cells while leaving normal cells intact,” said treating neurosurgeon, Dr. Syed. “With this, we are hoping to see improved outcomes for both tumor regression and patient health during treatment.”

As many as 300 children are diagnosed with DIPG in the U.S. each year and there are no effective treatments. DIPG infiltrates the pons, which is located in the brainstem. The condition is difficult to diagnose and treat because tumor cells often diffuse throughout the pons. Radiation therapy is the current standard of care, but it can only delay the cancer, not stop it.

“SDT is a targeted and nonsurgical investigational approach for kids who have almost no therapeutic options,” said SonALAsense founder, Chief Medical Officer and Chief Science Officer Stuart Marcus, M.D., Ph.D. “Surgery is contraindicated, chemotherapy and directed therapies are ineffective and have significant side effects.”

SDT combines SonALAsense’s drug candidate, SONALA-001, a proprietary intravenous formulation of aminolevulinic acid (ALA), with Insightec’s magnetic resonance-guided focused ultrasound to destroy cancer cells. As the SDT pioneer, SonALAsense is the first and only company to advance SDT into clinical trials in DIPG.

“SDT is a completely novel approach to an incredibly challenging pediatric cancer,” said Dr. Sabine Mueller, Pacific Pediatric Neuro-oncology Consortium (PNOC) Lead, and co-Leader of the Pediatric Malignancies Program, UCSF Helen Diller Family Comprehensive Cancer Center.

“DIPG is a devastating disease and, right now, we have no viable treatments for it,” said patient advocate Lisa Ward, whose son Jace died from DIPG complications in 2021 at age 22. “We need to explore encouraging new treatments, like SDT, to help patients and families who are suffering with this condition.”

The trial is supported, in part, by a $2 million National Cancer Institute Small Business Innovation Research grant to SonALAsense with a subcontract to Children’s National. The trial site in Phoenix is led by Nader Sanai, M.D., at the Ivy Brain Tumor Center, who also led the first-in-human Phase 0/1 clinical trial of SDT in recurrent high-grade glioma patients. The UCSF trial site is being led by Sabine Mueller, M.D., Ph.D.