Focused ultrasound is a noninvasive, therapeutic technology with the potential to improve the quality of life and decrease the cost of care for patients with cerebral cavernous malformations (CCMs), which are also called \u201ccavernomas\u201d. This novel technology focuses beams of ultrasound energy precisely and accurately on targets deep in the brain without damaging surrounding normal tissue.<\/p>\n\n\n\n
How it Works<\/strong> Advantages<\/strong> For certain patients, focused ultrasound could provide a noninvasive alternative to surgery with less risk of complications \u2013 such as surgical wound healing or infection \u2013 at a lower cost. It can also be repeated, if necessary.<\/p>\n\n\n\n At the present time, there are no clinical trials recruiting patients for focused ultrasound treatment of CCMs.<\/p>\n\n\n\n The Foundation updates these pages regularly, but with the increasing number of clinical trials, we want to be sure that our audience has the latest information available. Therefore, we also added the website search information for the above trials. If you click here<\/strong><\/a>, it will take you to the latest information available from https:\/\/www.clinicaltrials.gov\/<\/strong><\/a>. <\/p>\n\n\n\n Focused ultrasound treatment for CCM treatment is not yet approved by regulatory bodies or covered by medical insurance companies.<\/p>\n\n\n\n Martin E, Roberts M, Grigoras IF, Wright O, Nandi T, Rieger SW, Campbell J, den Boer T, Cox BT, Stagg CJ, Treeby BE. Ultrasound\u202fsystem\u202ffor\u202fprecise\u202fneuromodulation\u202fof\u202fhuman\u202fdeep\u202fbrain\u202fcircuits<\/strong><\/a>. Nat Commun. 2025 Sep 5;16(1):8024. doi: 10.1038\/s41467-025-63020-1. PMID:\u202f40913042\u202f\u00a0<\/p>\n\n\n\n Arrieta VA, Gould A, Kim KS, Habashy KJ, Dmello C, V\u00e1zquez-Cervantes GI, Palac\u00edn-Aliana I, McManus G, Amidei C, Gomez C, Dhiantravan S, Chen L, Zhang DY, Saganty R, Cholak ME, Pandey S, McCord M, McCortney K, Castro B, Ward R, Muzzio M, Bouchoux G, Desseaux C, Canney M, Carpentier A, Zhang B, Miska JM, Lesniak MS, Horbinski CM, Lukas RV, Stupp R, Lee-Chang C, Sonabend AM. Ultrasound-mediated\u202fdelivery\u202fof\u202fdoxorubicin\u202fto the\u202fbrain\u202fresults in immune modulation and improved responses to PD-1 blockade in gliomas<\/strong><\/a>. Nat Commun. 2024 Jun 6;15(1):4698. doi: 10.1038\/s41467-024-48326-w. PMID:\u202f38844770\u00a0<\/p>\n\n\n\n Fisher DG, Hoch MR, Gorick CM, Huchthausen C, Breza VR, Sharifi KA, Tvrdik P, Miller GW, Price RJ. Focused\u202fultrasound\u202faugments the delivery and penetration of model therapeutics into cerebral cavernous malformations.<\/a><\/strong> J Control Release. 2025 Jul 10;383:113861. doi: 10.1016\/j.jconrel.2025.113861. Epub 2025 May 16. PMID:\u202f40383158<\/p>\n\n\n\n Fisher DG, Cruz T, Hoch MR, Sharifi KA, Shah IM, Gorick CM, Breza VR, Debski AC, Samuels JD, Sheehan JP, Schlesinger D, Moore D, Mandell JW, Lukens JR, Miller GW, Tvrdik P, Price RJ. <\/strong>Focused<\/strong>\u202f<\/strong>ultrasound<\/strong>-m<\/strong>i<\/strong>crobubble treatment arrests the growth and formation of cerebral cavernous malformations.<\/strong><\/a> Nat Biomed Eng. 2025 May 13. doi: 10.1038\/s41551-025-01390-z. Online ahead of print. PMID:\u202f40360762 <\/p>\n\n\n\n Fisher DG, Hoch MR, Gorick CM, Huchthausen C, Breza VR, Sharifi KA, Tvrdik P, Miller GW, Price RJ. Focused\u202fUltrasound\u202fAugments the Delivery and Penetration of Model Therapeutics into Cerebral Cavernous Malformations.<\/strong><\/a> bioRxiv [Preprint]. 2025 Jan 23:2024.08.27.609060. doi: 10.1101\/2024.08.27.609060. Update in:<\/strong>\u202fJ Control Release. 2025 Jul 10;383:113861. doi: 10.1016\/j.jconrel.2025.113861.<\/strong><\/a> PMID:\u202f39253521 <\/p>\n\n\n\n Fisher DG, Sharifi KA, Shah IM, Gorick CM, Breza VR, Debski AC, Hoch MR, Cruz T, Samuels JD, Sheehan JP, Schlesinger D, Moore D, Lukens JR, Miller GW, Tvrdik P, Price RJ. Focused\u202fUltrasound\u202fBlood-Brain Barrier Opening Arrests the Growth and Formation of Cerebral Cavernous Malformations.<\/strong><\/a> bioRxiv [Preprint]. 2024 Feb 4:2024.01.31.577810. doi: 10.1101\/2024.01.31.577810. Update in:<\/strong>\u202fNat Biomed Eng. 2025 May 13. doi: 10.1038\/s41551-025-01390-z.<\/strong><\/a> PMID:\u202f38352349 <\/p>\n\n\n\n Gorick CM, Mathew AS, Garrison WJ, Thim EA, Fisher DG, Copeland CA, Song J, Klibanov AL, Miller GW, Price RJ. <\/strong><\/strong>Sonoselective<\/strong> transfection of cerebral vasculature without blood-brain barrier <\/strong>disruption.<\/strong><\/a> Proc Natl Acad Sci U S A. 2020 Mar 17;117(11):5644-5654. doi: 10.1073\/pnas.1914595117. Epub 2020 Mar 2. PMID:\u202f32123081\u202f <\/p>\n\n\n\n Pruitt R, Gamble A, Black K, Schulder M, Mehta AD. Complication avoidance in laser interstitial thermal therapy: lessons learned.<\/strong><\/a> J Neurosurg. 2017 Apr;126(4):1238-1245. doi: 10.3171\/2016.3.JNS152147. Epub 2016 Jun 3.<\/p>\n\n\n\n
Where the beams converge, focused ultrasound produces precise ablation (thermal destruction of tissue), which may enable CCMs to be treated without surgery. In addition, the use of low power focused ultrasound to temporarily disrupt the blood brain barrier (BBB) has been shown to dramatically decrease the size of existing CCMs in a mouse model. In follow up work, the delivery of small molecules to the CCM when accompanied by temporary BBB opening was increased both in the lesion core (4.6-fold) and perilesional space (6.7-fold) in mouse model. Additional preclinical work was showed the cessation of growth of 94% of the CCMs, compared to 7 fold growth of untreated CCMs, and there was a dramatic reduction in the de novo growth of new CCMs.<\/p>\n\n\n\n
The primary options for treatment of CCMs include surgery or observation. However, for deep brain CCMs, open surgery is likely to result in neural injury due to the retraction required to gain access to the CCM. The use of a non-invasive approach like focused ultrasound may enable patients to treat or control the growth of CCMs without the risks of open surgery.<\/p>\n\n\n\nClinical Trials<\/h2>\n\n\n\n
Regulatory Approval and Reimbursement<\/h2>\n\n\n\n
Notable Papers<\/h2>\n\n\n\n