FDA Fast Tracks Investigational Drug for the Treatment of Recurrent Brain Cancer

The Food and Drug Administration (FDA) recently granted Alpheus Medical orphan drug and fast track designations for its investigational CV-01 delivery of sonodynamic therapy as a potential treatment option for patients with recurrent high-grade glioblastoma, a type of spinal cord and brain cancer, according to a news release.

Patients are currently being enrolled onto the first-in-human phase 1 trial that is being conducted across multiple cancer treatment centers. The goal of the early-phase trial, according to the release, is to investigate the safety, optimal dosage and efficacy of the investigational drug in this patient population.

The investigational form of sonodynamic therapy is a non-invasive drug-device combination that targets cancer cells via low-intensity, diffuse ultrasound, according to the release. The treatment is administered in an outpatient setting.

“The diffuse nature of glioblastomas, often across the hemisphere, makes it an extremely challenging disease to treat,” Dr. Michael Schulder, director of the Brain Tumor Center and primary investigator (PI) for the clinical trial at Northwell Health's Institute for Neurology and Neurosurgery in New York City, said in the release. “There are very few effective options, leading to poor patient outcomes, and a universally fatal disease. Alpheus' sonodynamic therapy … has the potential to change the landscape of high-grade glioma therapy and we are excited to be part of this important study.”

A fast track designation creates a system where the FDA and the manufacturer can frequently communicate throughout the development and review process. As for the orphan drug designation, the FDA grants this status to pharmaceutical companies developing investigational treatments that would address rare medical diseases or conditions that affect less than 200,000 individuals in the United States.

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