Focused Ultrasound Shows Promise in Delivering Chemotherapy to Children with Brain Cancer
In a groundbreaking first, researchers at Columbia University have successfully demonstrated the safe application of focused ultrasound to enhance drug delivery to the brains of children undergoing treatment for brain cancer. The study, recently published in science Translational Medicine, marks a significant step forward in developing new therapeutic strategies for thes challenging cases.
focused ultrasound is a non-invasive technique utilizing sound waves to temporarily and controllably open the blood-brain barrier – a protective structure that unluckily also hinders the passage of most chemotherapy drugs to brain tumors. The Columbia team applied this method to facilitate chemotherapy penetration in three children diagnosed with diffuse midline glioma (DMG), a rare, aggressive cancer with a particularly poor prognosis and an average survival rate of less than one year.
The procedure,combined with chemotherapy management,successfully opened the blood-brain barrier in all three patients,allowing the drug to reach the tumor. Researchers observed some improvements in the children’s mobility following the treatment. While the children ultimately succumbed to cancer or complications related to COVID-19, the study’s primary goal was to establish the safety and feasibility of the technique in a clinical setting.
The Columbia team’s innovation extends beyond simply demonstrating safety. Their focused ultrasound device operates independently of a traditional MRI machine, unlike existing methods that require constant MRI guidance. Instead, necessary images are captured beforehand, allowing the procedure to be performed with a handheld device, creating a less stressful experience for young patients who can comfortably sit, read, or play during the brief, painless treatment.
The technique relies on the use of gaseous microbubbles coated in lipids, injected into cerebral vessels. Ultrasound waves cause these microbubbles to expand and contract, creating temporary microscopic openings that allow drugs to reach tumor tissue. Importantly, laboratory studies have shown the blood-brain barrier can be safely relaxed and then restored to its normal function.
Researchers believe that establishing the safety of this procedure paves the way for future trials utilizing earlier stages of the disease and lower systemic drug doses, while maximizing concentration within the brain. Columbia University has already initiated a follow-up study combining focused ultrasound with etoposide, a chemotherapy drug already approved for treating brain cancer.
The ability to overcome the blood-brain barrier is particularly significant, as many anti-tumor drugs have proven effective against glioma cells in laboratory settings. Future results from ongoing studies will be crucial in determining whether this technology can ultimately improve survival rates for children battling brain tumors - a field where advancements have been limited in recent decades.
