Focused Ultrasound Could Offer New Approach to Treating Deadly Brain Tumor

Pairing a new drug with focused ultrasound could introduce a much-needed treatment for glioblastoma, the deadliest type of brain tumor for which there are currently few therapeutic options.

Investigators from the University of Virginia School of Medicine are in the early phases of testing this combination both with mouse and human cells, but the results, so far, show their method can kill the number of living cancer cells in a tumor by nearly 50 percent. Their results, published in two recent studies in the Journal of Neuro-Oncology, indicate this oncologic strategy could be effective against other difficult-to-treat cancers, such as lung, breast, and skin cancers.

“Sonodynamic therapy with focused ultrasound offers a new therapeutic approach to treating patients with malignant brain tumors,” said Jason Sheehan, M.D., Ph.D., a neurosurgeon with UVA Health. “This approach combines two approved options, [the drug] 5-ALA and focused ultrasound, to produce a powerful tumoricidal effect on several different types of glioblastomas.”

Simply put, Sheehan’s team developed custom equipment to deliver 5-ALA, a drug that makes tumors sensitive to sound waves, before hitting the tumors with focused ultrasound. The ultrasound creates bubbles inside the tumor cells, killing them. Both the drug and the focused ultrasound can kill some tumor cells on their own, 5 percent and 16 percent, respectively. Together, though, they can kill 47 percent.

And, the team’s strategy is unique because it destroys the cancer cells without creating any heat.

These tests are early, Sheehan pointed out, but they are part of a larger effort at the University of Virginia to uncover the efficacy of focused ultrasound against a wide variety of diseases, such as epilepsy and breast cancer. Sheehan is also launching a separate glioblastoma clinical trial in people using a different focused ultrasound approach that determine whether the approach can be used to temporarily open the brain’s protective barrier so doctors can deliver tumor treatments they are currently unable to use.

“Focused ultrasound has the potential to improve outcomes for patients with complex brain tumors and other neurosurgical pathologies,” Sheehan said. “We may be at the tip of the iceberg in terms of intracranial indications for focused ultrasound.”