A new clinical trial has brought hope to people with glioblastoma, the most aggressive form of brain cancer.
Researchers at the University of Maryland School of Medicine (UMSOM) found that using MRI-guided focused ultrasound to open the blood-brain barrier, combined with standard chemotherapy, helped patients live longer.
The trial included 34 patients and showed that those who received the focused ultrasound treatment lived, on average, over 30 months. This is compared to just 19 months for patients who only received chemotherapy. Their cancer also took longer to return—about 14 months versus 8 months.
This is the first time focused ultrasound has shown a survival benefit in brain cancer patients. The technique helps more of the chemotherapy drug temozolomide reach the brain by temporarily opening the blood-brain barrier. Normally, less than 20% of this drug reaches the brain because the barrier blocks it.
Glioblastoma is the deadliest brain tumor. It affects thousands of people every year in the U.S., and most patients survive only about 14 to 16 months after diagnosis, even with surgery, radiation, and chemotherapy. This cancer usually comes back because some cancer cells remain hidden in the brain.
The blood-brain barrier acts like a security system to protect the brain. Researchers used a safe method to open it: they injected tiny bubbles into the bloodstream and used focused ultrasound guided by MRI to target specific brain areas. The ultrasound causes the bubbles to vibrate, which gently opens the barrier for a short time.
This trial is the result of more than ten years of research. It was led by Dr. Graeme Woodworth and took place at UMSOM and four other U.S. and Canadian hospitals.
Dr. Woodworth and his team also showed that opening the blood-brain barrier allowed for a new type of blood test called a ‘liquid biopsy.’ This test checks for cancer-related proteins and DNA in the blood, which could help doctors monitor brain cancer without surgery.
The liquid biopsy results matched closely with how patients were doing, including how long they lived and how long their cancer stayed away. This shows that it might become a useful, non-invasive way to track brain cancer.
Future trials may test this technique with other drugs that normally can’t cross the blood-brain barrier. This could lead to even better treatments for glioblastoma and other brain diseases.
The study is a major step forward. It proves that opening the blood-brain barrier is safe and may help improve survival for patients with very few treatment options. Leaders at UMSOM and the University of Maryland Medical Center say this success is only the beginning, and it brings new energy to brain cancer research.
A new trial called LIBERATE is already underway to explore even more uses for focused ultrasound. This trial is part of a larger effort called ReFOCUSED, which includes researchers from over 20 sites in North America. Their goal is to use focused ultrasound to improve treatment and diagnosis for brain diseases.
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The study is published in The Lancet Oncology.
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