FDA Grants Orphan Drug and Fast Track Designations to Sonodynamic Therapy in Brain Cancers

An orphan drug designation and a fast track designation have been granted by the FDA to a novel sonodynamic therapy platform as a potential treatment for patients with recurrent glioblastoma and other malignant gliomas.

The FDA has granted both an orphan drug designation and a fast track designation to a novel sonodynamic therapy platform as a potential treatment for patients with recurrent glioblastoma and other malignant gliomas, according to Alpheus™ Medical, Inc.1

A multi-center, first-in-human, phase 1 clinical trial (NCT05362409) evaluating the safety, optimal dosage, and efficacy of the sonodynamic therapy platform for patients with recurrent high-grade glioma is currently enrolling patients.

"The diffuse nature of glioblastomas, often across the hemisphere, makes it an extremely challenging disease to treat. There are very few effective options, leading to poor patient outcomes, and a universally fatal disease," commented Michael Schulder, MD, director of the Brain Tumor Center and primary investigator for the clinical trial at Northwell Health's Institute for Neurology and Neurosurgery, stated in the press release. "Alpheus' sonodynamic therapy enables non-invasive, diffuse treatment across the hemisphere. It has the potential to change the landscape of high-grade glioma therapy and we are excited to be part of this important study."

Within the phase 1 clinical trial, investigators will evaluate the safety and tolerability of 5-aminolevulinic acid (5-ALA) in combination with CV01 delivery of ultrasound for the sonodynamic therapy platform in patients with recurrent high-grade glioma.

This study will evaluate escalating durations of ultrasound delivery with CV01, which is a device that will deliver a non-ablative, low-intensity ultrasound to deep regions of the brain to induce apoptosis of cancer cells in the enrolled patients.2

An estimated 33 participants will be enrolled and administered 5-ALA as a sonosensitizer before the delivery of a CV01 ultrasound. Following this, 5-ALA will be re-administered every 4 weeks prior to the delivery of the CV01 ultrasound.

Those eligible for enrollment in the trial are patients aged 18 years and older with a previous confirmed diagnosis of high-grade glioma and radiographic evidence of recurrence after prior therapy with radiotherapy, including astrocytoma that is WHO grade 3 or 4, and oligodendroglioma that is WHO grade 3.

Additional requirements include having a WHO performance status of ≤ 2 at the time of screening, unifocal or multifocal tumor confined to the supratentorial compartment, adequate bone marrow, organ function, and adequate coagulation function defined as prothrombin time/partial thromboplastin time within normal institutional values. Further, any toxicities found in patients attributable to prior anti-cancer therapy must be resolved to the patient's baseline level or ≤ grade 1, excluding alopecia.

Primary end points of the trial include the incidence of adverse events, safety, and tolerability, and to determine the maximum tolerated dose. Secondary end points consist of assessment of overall response rate, duration of response, overall survival, and progression-free survival, all at a time frame of 12 months.

"The FDA fast track and orphan drug designations are significant milestones and highlight the importance of innovation within the field of brain cancer," added Vijay Agarwal, MD, chief executive officer and founder of Alpheus Medical and a practicing brain tumor surgeon, in the press release. "Built on a very successful pre-clinical program, we believe our proprietary sonodynamic therapy platform is a game changer and has the potential to significantly advance the treatment of gliomas."

  1. FDA grants alpheus medical orphan drug and fast track designations for novel sonodynamic therapy for brain cancer. News release. Alpheus Medical, Inc. July 13, 2022. Accessed July 13, 2022. https://prn.to/3yuSbbK
  2. Study to evaluate 5-ALA combined With CV01 delivery of ultrasound in recurrent high-grade glioma. ClinicalTrials.gov. Updated July 8, 2022. Accessed July 13, 2022. https://www.clinicaltrials.gov/ct2/show/NCT05362409?term=NCT05362409&draw=1&rank=1