Non-Invasive Ultrasound Halts Brain Lesion Growth

A new, non-invasive method developed at UVA Health to treat cerebral cavernous malformations (CCMs) has shown promising results in early laboratory tests. The approach halts the growth of these brain lesions, which can cause severe neurological problems.

A novel approach to treat CCMs

The technique uses tiny gas-filled microbubbles that are propelled by focused ultrasound waves to temporarily open the blood-brain barrier, the protective layer surrounding the brain. This method inhibits the progression of CCMs, clusters of abnormal blood vessels that can develop in the brain, spinal cord or other parts of the body.

Richard J. Price, PhD, co-director of UVA Health’s Focused Ultrasound Cancer Immunotherapy Center, explained that the treatment was discovered somewhat unexpectedly during safety studies of focused ultrasound for drug and gene delivery.

“This is a clear example of serendipity in science. We were looking for something else – performing long-term safety studies of focused ultrasound as a tool for drug and gene delivery to CCMs – when we noticed that CCMs exposed to just focused ultrasound with microbubbles were being stabilized," said Price.

Because focused ultrasound is non-invasive and the necessary devices are increasingly available in clinical settings, this technique could offer a simpler alternative to current CCM treatments, pending successful clinical trials.

Current treatment options and challenges

CCMs often do not produce symptoms, but in some cases they can cause headaches, seizures, muscle weakness or fatal brain hemorrhages. Treatments currently include brain surgery, usually reserved for lesions at risk of bleeding, and stereotactic radiosurgery, which targets hard-to-reach lesions with radiation.

Both surgical and radiosurgical interventions carry risks. Surgery can lead to complications, and removed CCMs may regrow. The new ultrasound approach offers a potential alternative that could avoid these side effects.

Laboratory results and future potential

In mouse models, the ultrasound with microbubble treatment stopped the growth of 94% of CCM lesions after one month. Untreated lesions in the same time period increased in size seven-fold. These mouse models present a more aggressive form of CCM than typically seen in humans, suggesting the treatment might be effective even in severe cases.

Additionally, treated brain tissue appeared less likely to develop new CCMs, suggesting a possible preventive effect. This could benefit patients with familial CCM, who are genetically prone to developing multiple lesions over time.

Simulated treatment plans using existing stereotactic radiosurgery patient data suggest that this method is already feasible with current technology. However, clinical trials will be needed before regulatory approval can be sought.

Mechanism and next steps

The technique does not use drugs. Focused ultrasound with microbubbles has previously been investigated to transiently open the blood-brain barrier to improve drug delivery in diseases such as Alzheimer’s. In both Alzheimer’s and CCM, the ultrasound-microbubble interaction alone shows therapeutic effects that remain unexplained.

Ongoing research aims to clarify how focused ultrasound inhibits CCM growth and to explore combining this baseline effect with drug or gene therapies to potentially eliminate lesions completely.

The development of this approach builds on UVA’s long-term investment in focused ultrasound technology and its extensive expertise and infrastructure.

Reference: Fisher DG, Cruz T, Hoch MR, et al. Focused ultrasound-microbubble treatment arrests the growth and formation of cerebral cavernous malformations. Nat Biomed Eng. 2025. doi: 10.1038/s41551-025-01390-z

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