A new breakthrough technique could revolutionize the treatment of glioblastoma, a deadly brain cancer with a survival rate of only 6.8 percent within the first five years of diagnosis. The new technique involves the use of sound waves to permeate the blood-brain barrier, a line of defense that prevents toxins and pathogens from entering the brain, and allows chemotherapy drugs to reach the neurological tissues where the cancer can grow.

The technique was tested in a phase 1 in-human clinical trial with 17 patients who underwent surgery to remove their tumors and had an ultrasound device implanted. The device, a novel skull-implantable grid of nine ultrasound emitters made by French biotech company Carthera, repeatedly uses sound waves to permeate the barrier and reach the brain tumor. The chemotherapy drugs paclitaxel and carboplatin, which are typically unable to cross the blood-brain barrier, were then able to reach the brain.

The results of the trial, published in the journal The Lancet Oncology on May 2, demonstrated the safety and efficacy of the new technique. The blood-brain barrier was opened in a volume of the brain nine times larger than with the small single-ultrasound emitter implants originally used, allowing for the treatment of a larger region of the brain next to the cavity that remains after glioblastoma tumors are removed. The procedure to open the blood-brain barrier only takes four minutes and is performed while the patient is awake.

The study also showed how quickly the blood-brain barrier closes after being opened by the ultrasound, with the communication closing in the first 30 to 60 minutes. This will help scientists optimize the order of drug delivery to allow for better penetration of the brain.

According to co-author and Northwestern University neurosurgeon Adam Sonabend, “This is potentially a huge advance for glioblastoma patients.” The technique could offer hope to the thousands of patients in the US suffering from glioblastoma, as well as opening up possibilities for the treatment of other brain diseases.

The technique builds on earlier research from 2014, which showed that sound waves could be used to permeate the blood-brain barrier. This new study demonstrates the safety and efficacy of the technique, and its potential for wider use in the treatment of brain diseases.

Sonabend notes that the current chemotherapy used for glioblastoma, Temozolomide, does cross the blood-brain barrier but is weak. Previous studies that injected paclitaxel directly into the brains of patients with these tumors had promising signs of efficacy, but the direct injection was associated with toxicity such as brain irritation and meningitis. The new technique avoids these risks and could lead to more effective treatment options for glioblastoma patients.

A phase 2 clinical trial is already underway, and the researchers hope to continue refining the technique to optimize drug delivery and improve outcomes for patients. As Sonabend notes, “while we have focused on brain cancer, this opens the door to investigate novel drug-based treatments for millions of patients who suffer from various brain diseases.”

The new technique could offer hope to patients with a range of brain diseases, as well as offering new possibilities for drug delivery in other parts of the body. The sound wave technique represents a major breakthrough in the treatment of glioblastoma, and a promising avenue for future research in the fight against cancer and other diseases.

By Impact Lab