Alpheus Medical, Inc. has announced encouraging results from its Phase 1/2 clinical trial evaluating sonodynamic therapy (SDT) for patients with recurrent or refractory high-grade gliomas. The study, presented at the 2024 Society of Neuro-Oncology (SNO) Annual Meeting, demonstrated a favorable safety profile and notable improvements in survival outcomes compared to historical benchmarks.
The open-label, multicenter, duration-escalation study (NCT05362409) enrolled twelve patients across three cohorts, with treatment durations escalating to 60, 90, and 120 minutes per monthly session. The SDT approach combines low-intensity diffuse ultrasound (LIDU™) with oral 5-aminolevulinic acid (5-ALA) to selectively target and destroy cancer cells throughout the brain. This non-invasive method avoids the need for imaging or sedation, enabling outpatient administration.
Survival Benefits and Safety Profile
Key findings from the Phase 1/2 trial include a median overall survival (OS) of 15.7 months, significantly longer than the historical OS of approximately 6-8 months for patients with recurrent high-grade gliomas. The median progression-free survival (PFS) was 5.5 months, compared to a historical PFS of 1.8 months. Importantly, the treatment demonstrated a strong safety profile, with no treatment-related deaths, serious adverse events (SAEs), or duration-limited toxicities (DuLTs) reported.
Expert Commentary
"Glioblastomas are the most common and aggressive primary brain cancer, presenting a devasting diagnosis for patients and their familes," said David Reardon, MD, Clinical Director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute, and member of the Alpheus Medical Scientific Advisory Board. "Current treatment options are limited and often ineffective due to the diffuse spread of the disease across the blood-brain barrier and often across the entire hemisphere, making it universally fatal with a rapid timeline. The early clinical results of Alpheus's therapy are promising, offering hope for this new approach. I look forward to further exploring the potential benefits of their SDT therapy for this patient population who is in critical need of an effective solution."
Future Directions
Alpheus Medical plans to initiate a randomized, controlled trial at multiple centers across the U.S. in 2025 to further evaluate the efficacy of its SDT platform. This trial will provide more definitive evidence of the treatment's benefits and help establish its role in the management of recurrent high-grade gliomas. The company's SDT therapy offers a potential new treatment option for patients with this challenging disease, addressing the unmet need for effective and well-tolerated therapies.
