Cerebral cavernous malformations (CCMs), also known as cavernomas, are abnormal clusters of blood vessels that can grow in the brain, spinal cord, or other parts of the body. While most cases remain asymptomatic, some individuals may experience headaches, seizures, muscle weakness, or even life-threatening complications. Treatment for CCMs often involves brain surgery, typically when there is a risk of a dangerous brain bleed, or stereotactic radiosurgery, which uses radiation to target CCMs that are too difficult for a surgeon to reach. Now, a new, incision-free technique has emerged as a promising option to treat these debilitating lesions, showing great potential in early trials by effectively halting their growth.

The new approach, developed at UVA Health’s FUS Cancer Immunotherapy Center (Charlottesville, VA, USA), could revolutionize the way CCMs are treated, according to the researchers. The method employs tiny, gas-filled "microbubbles" that are activated by focused sound waves. These sound waves open the brain's protective barrier and stop the growth of the cavernomas. In a study published in Nature Biomedical Engineering, the researchers were astounded by the success of their microbubble treatment in laboratory tests. After just one month, the treatment had stopped the growth of 94% of CCMs in lab mice. In contrast, untreated CCMs grew seven times in size over the same period. In some instances, brain tissue that was exposed to the focused ultrasound with microbubbles showed a reduced tendency to develop new CCMs in the future. If this outcome can be replicated in humans, it may offer a preventive treatment for individuals with a genetic predisposition to developing multiple new CCMs throughout their lives.

This novel technique could present an alternative to traditional treatments, avoiding the side effects commonly associated with brain surgery and stereotactic radiosurgery. For instance, conventional brain surgery carries risks inherent to the procedure and the possibility that the cavernomas could regrow after removal. Furthermore, simulations of treatment plans for patients who have received stereotactic radiosurgery show that the microbubble approach is already compatible with current technology, although clinical trials will be necessary before the U.S. Food and Drug Administration (FDA) could approve it for use in patients. One of the key advantages of this approach is that it does not involve the use of drugs. Researchers at UVA and other institutions have been exploring the use of focused ultrasound to briefly disrupt the blood-brain barrier, which could allow for targeted drug delivery for conditions like Alzheimer's disease. However, in both Alzheimer’s and now CCMs, the application of sound-propelled microbubbles has demonstrated substantial benefits even without the use of drugs, a result that scientists are still working to fully understand.

“Because the focused ultrasound treatment is relatively simple and non-invasive and the necessary clinical devices are becoming more common, if proven safe in clinical trials, I am hopeful it could eventually become a real treatment option,” said researcher Richard J. Price, PhD, co-director of UVA Health’s Focused Ultrasound Cancer Immunotherapy Center.

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