Columbia University researchers have used sound waves for non-invasive opening of the blood-brain barrier, the brain’s natural “protection” that usually prevents drugs from reaching tumors. The study findings were published in Science Translational Medicine.

The technology, combined with chemotherapy, was tested on three children with diffuse intrinsic pontine glioma, a rare and highly aggressive type of brain cancer that almost always leads to death. Focused ultrasound enabled drugs to successfully reach the tumors, improving patients’ condition without side effects.

“Now that we've established the safety and feasibility of focused ultrasound in children, we've opened the door for more trials to try the technique earlier in the course of the disease,” said study leader Stergios Zacharoulis, associate professor of pediatrics at Columbia University's Vagelos College of Physicians and Surgeons.

Despite some progress in treating aggressive tumors, the prognosis for pediatric brain cancer remains extremely poor. Most patients with pontine glioma live less than a year after diagnosis. The main problem is the blood-brain barrier, which protects the brain from toxins and infections, but blocks the effect of chemotherapy.

Previous studies have shown that the barrier can be safely weakened to improve drug delivery and then restored. These results provide the basis for the new treatment method.

“Patients can rest their heads on a massage table and play on a tablet or read a book during treatment, and the procedure is entirely noninvasive and painless and only lasts for a few minutes,” said Elisa Konofagou, a professor of biomedical engineering at Columbia.

This technology has the potential to change the approach to treating pediatric cancer and improve survival for patients with hard-to-treat tumors, the researchers added.