Innovative Focused Ultrasound Technique Stops Growth of Brain Cavernomas in Early Trials

Researchers at UVA Health have developed a groundbreaking, minimally invasive method using focused ultrasound combined with microbubbles to treat cerebral cavernous malformations, also known as cavernomas. These vascular anomalies, which consist of clusters of abnormally formed blood vessels in the brain, can cause symptoms like seizures, headaches, and in severe cases, neurological damage or death.

The new technique involves delivering tiny, gas-filled microbubbles into the bloodstream and directing focused sound waves at the target lesions. This process temporarily opens the blood-brain barrier, enabling the microbubbles to influence the growth and stability of the cavernomas without the need for surgery or radiation.

Early laboratory studies have yielded impressive results, with one month post-treatment showing that 94% of cavernomas in mouse models halted their growth, whereas untreated malformations grew seven times larger during the same period. Notably, even aggressive mouse models with rapidly expanding CCMs responded completely to this treatment.

The approach was discovered serendipitously while investigating focused ultrasound for drug and gene delivery to brain lesions. Dr. Richard J. Price, a lead researcher, emphasized the simplicity and noninvasiveness of the method and expressed optimism about its future application in clinical settings, pending safety and efficacy in human trials.

Currently, traditional treatments for cavernomas include surgical removal—primarily when there is a risk of bleeding—or stereotactic radiosurgery, which uses targeted radiation. However, these options carry risks such as surgical complications and recurrence.

This microbubble-focused ultrasound technique could revolutionize cavernoma treatment by offering a safer alternative that avoids the downsides of surgery and radiation. In preclinical studies, the treatment significantly reduced CCM growth and showed potential for preventative applications in genetically predisposed individuals.

Furthermore, simulations suggest that implementing this strategy with existing clinical ultrasound devices is feasible. Despite promising results, further clinical trials are essential before the approach receives regulatory approval. Interestingly, this technique does not involve any drugs, aligning with ongoing research into focused ultrasound's capacity to transiently open the blood-brain barrier for medication delivery.

The success of similar ultrasound-based therapies for Alzheimer's disease provides a hopeful outlook for broader neurological applications. Researchers aim to understand exactly how focused ultrasound halts lesion growth, which could lead to new, combined therapeutic strategies against cavernomas, including eradication approaches.

This advancement represents a significant step forward in noninvasive neuromodulation, opening new avenues for treatment and prevention of complex brain vascular disorders.