Focused Ultrasound Therapy
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 brain tumors. This novel technology focuses beams of ultrasonic energy precisely and accurately on targets deep in the brain without damaging surrounding normal tissue.
How it Works
Where the beams converge, the ultrasound produces a variety of therapeutic effects enabling treatment without incisions or radiation. These include temporary opening of the blood-brain barrier, designed to improve levels in the brain of therapeutic agents; thermal ablation; activation of sono-sensitive agents at the target; and combining with radiation therapy for a more pronounce effect. There is also a study using focused ultrasound prior to surgery to help mark the boundary of the tumor extension.
Advantages
Current treatments for brain tumors include surgery, radiation therapy, and chemotherapy, all of which have limitations and side effects.
Focused ultrasound, used alone or in combination with conventional therapies, has the potential for several advantages.
- Focused ultrasound is non-invasive, so it does not carry added concerns like surgical wound healing or infection.
- Focused ultrasound can reach the desired target without damaging surrounding tissue.
- Focused ultrasound does not include the use of ionizing radiation
- Enhanced chemotherapy dose for the target, with less impact to the rest of the patient.
- It can be repeated, if necessary.
Clinical Trials
Extracellular Impact of Ultrasound-induced Blood-brain Barrier Disruption
This study of BBBD is looking at the impact of the treatment on other therapeutic and pharmacodynamic substances that may also be in the area when this is accomplished.
An Ultrasound-Based Blood Brain Barrier Opening Clinical Trial Using Albumin Bound Paclitaxel to Treat Recurrent Glioblastoma
This invasive procedure that is similar to focused ultrasound uses an implanted device to treat patients with recurrent glioblastoma.
Blood-Brain Barrier Disruption (BBBD) for Liquid Biopsy in Subjects With Glioblastoma Brain Tumors
A clinical trial for patients with Glioblastoma for liquid biopsy has also begun recruiting patients.
The following studies concern focused ultrasound and sonodynamic therapy.
Sonodynamic therapy in patients with recurrent glioblastoma.
This study used oral gleolan (5 ALA) which becomes altered in the presence of focused ultrasound and becomes locally toxic to the tumor.
A study of sonodynamic therapy for patient with high grade glioma
A new clinical trial in the US is using sound activated drugs to treat patients with recurrent high grade glioma, including glioblastoma.
A study of sonodynamic therapy in patients with newly diagnosed glioblastoma
This study will use low frequency focused ultrasound to activate drugs in patients with newly diagnosed glioblastoma.
Blood-Brain Barrier Disruption (BBBD) for Liquid Biopsy in Subjects With Glioblastoma Brain Tumors
A clinical trial for patients with glioblastoma for liquid biopsy has also begun recruiting patients.
The following studies concern tumors in pediatric patients.
Phase 1/2 Study of Sonodynamic therapy to treat Type 2 Patients with DIPG
A clinical trial is using sonodynamic therapy to treat pediatric patients who are five years and older with DIPG (diffuse intrinsic pontine glioma).
Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric DIPG
A clinical trial using doxorubicin to treat DIPG has begun in the US.
Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric DIPG
A clinical trial using doxorubicin to treat DIPG has begun in Canada.
A Feasibility Safety Study of Benign Centrally-Located Intracranial Tumors in Pediatric and Young Adult Subjects
Centrally located intracranial benign tumors that require intervention in pediatric and young adult patients. This study has been completed.
A clinical trial by Carthera is recruiting pediatric patients with recurrent malignant brain tumors in France.
The following studies concern treatment of tumors from the body that have metastasized to the brain.
A clinical trial for patients with breast cancer that has metastasized to their brain is enrolling patients in Canada. The team will us focused ultrasound to open the blood-brain barrier to allow therapeutic medications to more effectively reach the tumor in their brain. This study is only open to Canadian citizens. A summary of the first four patients has been published.
A clinical trial for patients with lung cancer that has metastasized to the brain has begun recruiting patients in the US.
The following study concerns a wide variety of brain tumors.
This clinical trial is for a wide variety of brain tumors that are planned to be biopsied. This study is only open to Canadian citizens.
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, it will take you to the latest information available from https://www.clinicaltrials.gov/.
See a list of treatment sites >
See a list of clinical trials sites >
See a list of laboratory research sites >
Regulatory Approval and Reimbursement
Focused ultrasound is not approved by any regulatory bodies worldwide as a treatment for brain tumors, nor is the treatment reimbursed by medical insurance providers.
Preclinical Laboratory Studies
Preclinical studies are underway to investigate the use of various mechanisms of focused ultrasound in the treatment of brain tumors. Examples of these studies include:
- Focused ultrasound to temporarily disrupt the BBB and deliver a variety of chemotherapy or immunotherapy drugs, including the dosing and timing (e.g. frequency) of drug administration.
- Focused ultrasound to induce an immune response, including a multi-site study investigating the type of immune response elicited by different “modes” of energy delivery.
- Focused ultrasound to enable targeted delivery and/or activation of drugs via carrier vehicles (e.g. microbubbles, nanoparticles, liposomes) to enable delivery of high concentrations in the tumor with minimal systemic side effects.
- Non-thermal mechanical destruction of tumor using a type of focused ultrasound called histotripsy.
Additional Resources
There are many government bodies and patient groups dedicated to brain tumors, including the following:
- Medline Plus: A service of the U.S. National Library of Medicine and NIH
- National Cancer Institute’s Brain Tumor Page
- American Brain Tumor Association
- National Brain Tumor Society
Notable Papers
Suggested Reading: Focused Ultrasound for Glioblastoma (PDF), June 2021.
Beylerli O, Gareev I, Musaev E, Roumiantsev S, Chekhonin V, Ahmad A, Chao Y, Yang G. New approaches to targeted drug therapy of intracranial tumors. Cell Death Discov. 2025 Mar 20;11(1):111. doi: 10.1038/s41420-025-02358-3. PMID: 40113789
De Maio A, Huang Y, Lin FH, Stefanovic B, Stanisz GJ, O’Reilly MA. Evaluation of focused ultrasound modulation of the blood-brain barrier in gray and white matter. J Control Release. 2025 Mar 15;381:113631. doi: 10.1016/j.jconrel.2025.113631. PMID: 40096865
Koruk H, Payne C, Cressey P, Thanou M, Pouliopoulos AN. Delivering Gd-Labeled IgG Antibodies Into the Mouse Brain Following Focused Ultrasound Treatment. Ultrasound Med Biol. 2025 Mar 13:S0301-5629(25)00067-5. doi: 10.1016/j.ultrasmedbio.2025.02.015. PMID: 40087107
Tang Z, Niu H, Wu Y, Zhang Y, Zhang F, Wang C, Zhang S, Song X, Wang Y, Du L, Jin Y. Ultrasonic head-mounted device spatiotemporal opening blood-brain barrier enhances the brain permeation of drugs for treatment of radiation-induced brain injury. Int J Pharm. 2025 Mar 11;674:125430. doi: 10.1016/j.ijpharm.2025.125430. PMID: 40081430
Dai H, Li W, Wang Q, Cheng B. Multiple Instance Learning-Based Prediction of Blood-Brain Barrier Opening Outcomes Induced by Focused Ultrasound. IEEE Trans Biomed Eng. 2025 Apr;72(4):1465-1472. doi: 10.1109/TBME.2024.3509533. Epub 2025 Mar 21. PMID: 40030539
Ponomarchuk E, Tsysar S, Kadrev A, Kvashennikova A, Chupova D, Pestova P, Papikyan L, Karzova M, Danilova N, Malkov P, Chernyaev A, Buravkov S, Sapozhnikov O, Khokhlova V. Boiling Histotripsy in Ex Vivo Human Brain: Proof-of-concept. Ultrasound Med Biol. 2025 Feb;51(2):312-320. doi: 10.1016/j.ultrasmedbio.2024.10.006. Epub 2024 Oct 30. PMID: 39482208
Manuel TJ, Bancel T, Tiennot T, Didier M, Santin M, Daniel M, Attali D, Tanter M, Lehéricy S, Pyatigorskaya N, Aubry JF. Ultra-short time-echo based ray tracing for transcranial focused ultrasound aberration correction in human calvaria. Phys Med Biol. 2025 Mar 21;70(7). doi: 10.1088/1361-6560/ad4f44. PMID: 38776944
Gong Y, Xu K, Ye D, Yang Y, Miller MJ, Feng Z, Hu S, Chen H. In vivo two-photon microscopy imaging of focused ultrasound-mediated glymphatic transport in the mouse brain. J Cereb Blood Flow Metab. 2025 Feb 22:271678X251323369. doi: 10.1177/0271678X251323369. PMID: 39985197
Atkinson-Clement C, Kaiser M. Optimizing Transcranial Focused Ultrasound Stimulation: An Open-source Tool for Precise Targeting. Neuromodulation. 2025 Jan;28(1):185-187. doi: 10.1016/j.neurom.2024.06.496. Epub 2024 Jul 31. PMID: 39093260
Bawiec CR, Hollender PJ, Ornellas SB, Schachtner JN, Dahill-Fuchel JF, Konecky SD, Allen JJB. A Wearable, Steerable, Transcranial Low-Intensity Focused Ultrasound System. J Ultrasound Med. 2025 Feb;44(2):239-261. doi: 10.1002/jum.16600. Epub 2024 Oct 24. PMID: 39449176
Shah BR, Tanabe J, Jordan JE, Kern D, Harward SC, Feltrin FS, O’Suilliebhain P, Sharma VD, Maldjian JA, Boutet A, Mattay R, Sugrue LP, Narsinh K, Hetts S, Shah LM, Druzgal J, Lehman VT, Lee K, Khanpara S, Lad S, Kaufmann TJ. State of Practice on Transcranial MR-Guided Focused Ultrasound: A Report from the ASNR Standards and Guidelines Committee and ACR Commission on Neuroradiology Workgroup. AJNR Am J Neuroradiol. 2025 Jan 8;46(1):2-10. doi: 10.3174/ajnr.A8405. PMID: 39572202
Kaovasia TP, Duclos S, Gupta D, Kalayeh K, Fabiilli M, Noll DC, Sukovich J, Pandey A, Xu Z, Hall TL. A pre-clinical MRI-guided all-in-one focused ultrasound system for murine brain studies. Sci Rep. 2025 Jan 2;15(1):144. doi: 10.1038/s41598-024-84078-9. PMID: 39747938
Young JS, Semonche A, Morshed RA, Al-Adli NN, Haddad AF, Gerritsen JKW, Saggi S, Narsinh K, de Groot J, Aghi MK. Focused ultrasound therapy as a strategy for improving glioma treatment. J Neurosurg. 2025 Jan 24:1-10. doi: 10.3171/2024.9.JNS24721. PMID: 39854698
Arsiwala TA, Blethen KE, Wolford CP, Pecar GL, Panchal DM, Kielkowski BN, Wang P, Ranjan M, Carpenter JS, Finomore V, Rezai A, Lockman PR. Single Exposure to Low-Intensity Focused Ultrasound Causes Biphasic Opening of the Blood-Brain Barrier Through Secondary Mechanisms. Pharmaceutics. 2025 Jan 8;17(1):75. doi: 10.3390/pharmaceutics17010075. PMID: 39861723
Giantini-Larsen AM, Pandey A, Garton ALA, Rampichini M, Winston G, Goldberg JL, Magge R, Stieg PE, Souweidane MM, Ramakrishna R. Therapeutic manipulation and bypass of the blood-brain barrier: powerful tools in glioma treatment. Neurooncol Adv. 2025 Jan 15;7(1):vdae201. doi: 10.1093/noajnl/vdae201. eCollection 2025. PMID: 39877748
Gould A, Luan Y, Hou Y, Korobova FV, Chen L, Arrieta VA, Amidei C, Ward R, Gomez C, Castro B, Habashy K, Zhang D, Youngblood M, Dmello C, Bebawy J, Bouchoux G, Stupp R, Canney M, Yue F, Iruela-Arispe ML, Sonabend AM. Endothelial Response to Blood-Brain Barrier Disruption in the Human Brain. JCI Insight. 2024 Dec 26:e187328. doi: 10.1172/jci.insight.187328. PMID: 39724015
Erickson NJ, Stavarache M, Ibrahim T, Kaplitt MG, Markert JM. Herpes Simplex Oncolytic Viral Therapy for Malignant Glioma and Mechanisms of Delivery. World Neurosurg. 2024 Dec 20:123595. doi: 10.1016/j.wneu.2024.123595. PMID: 39710201
Mekers V, de Visser M, Suijkerbuijk K, Bos C, Moonen C, Deckers R, Adema G. Mechanical HIFU and immune checkpoint inhibition: toward clinical implementation. Int J Hyperthermia. 2024;41(1):2430333. doi: 10.1080/02656736.2024.2430333. Epub 2024 Nov 20.PMID: 39566471
Huang Q, Zhou Y, Pan L, Chen Y, Wang N, Li K, Bai J, Ji X. Experimental Evaluation of an Ultrasound-Guided High-Intensity-Focused Ultrasound Probe for Sonication of Artery. J Ultrasound Med. 2024 Dec;43(12):2357-2373. doi: 10.1002/jum.16571. Epub 2024 Sep 6. PMID: 39240034
Chen H, Anastasiadis P, Woodworth GF. MR Imaging-Guided Focused Ultrasound-Clinical Applications in Managing Malignant Gliomas. Magn Reson Imaging Clin N Am. 2024 Nov;32(4):673-679. doi: 10.1016/j.mric.2024.05.006. Epub 2024 Jun 22. PMID: 39322356
Martinez PJ, Song JJ, Castillo JI, DeSisto J, Song KH, Green AL, Borden M. Effect of Microbubble Size, Composition, and Multiple Sonication Points on Sterile Inflammatory Response in Focused Ultrasound-Mediated Blood-Brain Barrier Opening. ACS Biomater Sci Eng. 2024 Nov 5. doi: 10.1021/acsbiomaterials.4c00777. PMID: 39497639
Epstein JE, Pople CB, Meng Y, Lipsman N. An update on the role of focused ultrasound in neuro-oncology. Curr Opin Neurol. 2024 Dec 1;37(6):682-692. doi: 10.1097/WCO.0000000000001314. Epub 2024 Oct 4. PMID: 39498847
Gupta D, Kaovasia TP, Komaiha M, Nielsen JF, Allen SP, Hall TL, Noll DC, Xu Z. Transcranial MRI-guided Histotripsy Targeting Using MR-thermometry and MR-ARFI. Ultrasound Med Biol. 2024 Nov 25:S0301-5629(24)00406-X. doi: 10.1016/j.ultrasmedbio.2024.10.010. PMID: 39592380
Xu S, Meng L, Hu Q, Li F, Zhang J, Kong N, Xing Z, Hong G, Zhu X. Closed-Loop Control of Macrophage Engineering Enabled by Focused-Ultrasound Responsive Mechanoluminescence Nanoplatform for Precise Cancer Immunotherapy. Small. 2024 Nov;20(46):e2401398. doi: 10.1002/smll.202401398. Epub 2024 Aug 5. PMID: 39101277
Ahmed AK, Woodworth GF, Gandhi D. Transcranial Focused Ultrasound: A History of Our Future. Magn Reson Imaging Clin N Am. 2024 Nov;32(4):585-592. doi: 10.1016/j.mric.2024.04.002. Epub 2024 Jul 25. PMID: 39322349
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