Revolutionizing Childhood Brain Cancer Treatment: Focused Ultrasound's Promise
The Challenge:
For decades, the prognosis for brain cancer has remained bleak, especially for children. Survival rates for those with midline glioma, a rare and aggressive brain cancer, are typically less than a year post-diagnosis. A major hurdle in improving these outcomes is the blood-brain barrier, which protects the brain from harmful substances but also prevents most chemotherapy drugs from reaching brain tumors in sufficient concentrations.
A Breakthrough:
Columbia University researchers have made a groundbreaking discovery. They've shown that focused ultrasound, a non-invasive technique using sound waves, can safely and effectively open the blood-brain barrier in children with brain cancer. This innovation, developed by Columbia engineers, was tested in combination with chemotherapy in three children with diffuse midline glioma, a universally fatal brain cancer.
The Results:
The study found that focused ultrasound successfully opened the blood-brain barrier in all three patients, allowing the chemotherapy drug to reach the tumors. This led to some improvement in patient mobility. While all three patients eventually died from their disease or complications from COVID, the study demonstrates the potential of focused ultrasound as a promising treatment option.
Looking Ahead:
"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 and with lower systemic but higher brain doses," says Stergios Zacharoulis, associate professor of pediatrics at Columbia University's Vagelos College of Physicians and Surgeons and a pediatric oncologist at New York-Presbyterian Morgan Stanley Children's Hospital. "Our hope is that the technology will improve survival for children with brain cancer."
Columbia has already launched a follow-up trial using focused ultrasound with etoposide, an FDA-approved chemotherapy drug that has shown activity against brain cancer cells. The technique's potential to transform treatment is significant, as many cancer drugs are capable of killing brain cancer cells.
How Focused Ultrasound Works:
Focused ultrasound opens the blood-brain barrier by using sound waves to vibrate tiny lipid-coated gas bubbles within the barrier. As the vibrating bubbles expand and contract, they pry open pathways in the barrier that drugs can pass through. Most focused ultrasound techniques require the use of MRI machines to guide the sound waves to specific locations in the brain.
Overcoming Challenges:
"MRI scans can be stressful experiences for some, especially for children who find it difficult to stay still during the procedure," says Elisa Konofagou, a professor of biomedical engineering at Columbia. "At Columbia, our innovation was to move the focused ultrasound treatment outside of the MRI machine and perform the treatment in a friendly environment for the child with their family in the room during the procedure."
With the Columbia-designed device, MRI images for navigation planning are obtained before the focused ultrasound treatment with a hand-guided device. Patients can rest their heads on a massage table and play on a tablet or read a book during treatment, making the procedure entirely non-invasive, painless, and lasting only a few minutes.
