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 ALS. 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, focused ultrasound produces temporary opening of the blood-brain barrier (BBB) that may allow therapeutic agents to gain access to the brain in sufficient quantities to treat ALS. The BBB closes shortly after the completion of the sonication.
Advantages
The primary conventional options for treatment of ALS include medication and supportive care. For certain patients, focused ultrasound could provide a noninvasive, effective alternative for this very difficult disease. Focused ultrasound may be a more successful treatment than current therapy as it may include the passage of drugs that otherwise could not cross the BBB. It can also be repeated, if necessary.
Clinical Trials
A clinical trial will be starting soon, using this technology to temporarily open the BBB in patients with ALS. This trial is jointly funded by the ALS Association and the Focused Ultrasound Foundation.
See a full list of ALS clinical trials >
Regulatory Approval and Reimbursement
Focused ultrasound treatment for ALS is not yet approved by regulatory bodies or covered by medical insurance companies.
Notable Papers
Suggested Reading: Focused Ultrasound: Transforming treatment of Neurodegenerative Diseases August 2022
Wasielewska JM, Chaves JCS, Cabral-da-Silva MC, Pecoraro M, Viljoen SJ, Nguyen TH, Bella V, Oikari LE, Ooi L, White AR. A patient-derived amyotrophic lateral sclerosis blood-brain barrier model for focused ultrasound-mediated anti-TDP-43 antibody delivery. Fluids Barriers CNS. 2024 Aug 13;21(1):65. doi: 10.1186/s12987-024-00565-1. PMID: 39138578
Zhang J, Chen K, Chen Y, Hua L, Chen S, Chen X, Zou L, Li S, Yang X, Shen Y. Pathology reduction and motor behavior improvement associated with ultrasound-mediated delivery of arctiin to the motor cortex in a mutant SOD1 mouse model of amyotrophic lateral sclerosis. Ultrasonics. 2024 Aug 29;144:107449. doi: 10.1016/j.ultras.2024.107449. PMID: 39217855
Shen Y, Zhang J, Xu Y, Sun S, Chen K, Chen S, Yang X, Chen X. Ultrasound-enhanced brain delivery of edaravone provides additive amelioration on disease progression in an ALS mouse model. Brain Stimul. 2023 Mar 16;16(2):628-641. doi: 10.1016/j.brs.2023.03.006.
Ediriweera GR, Chen L, Yerbury JJ, Thurecht KJ, Vine KL. Non-Viral Vector-Mediated Gene Therapy for ALS: Challenges and Future Perspectives. Mol Pharm. 2021 May 19. doi: 10.1021/acs.molpharmaceut.1c00297.
Abrahao A, Meng Y, Llinas M, Huang Y, Hamani C, Mainprize T, Aubert I, Heyn C, Black SE, Hynynen K, Lipsman N, Zinman L. First-in-human trial of blood-brain barrier opening in amyotrophic lateral sclerosis using MR-guided focused ultrasound. Nat Commun. 2019 Sep 26;10(1):4373. doi: 10.1038/s41467-019-12426-9.
Jones RM, Hynynen K. Advances in acoustic monitoring and control of focused ultrasound-mediated increases in blood-brain barrier permeability. Br J Radiol. 2018 Dec 3:20180601. doi: 10.1259/bjr.20180601.
Saunders NR, Habgood MD, Møllgård K, Dziegielewska KM. The biological significance of brain barrier mechanisms: help or hindrance in drug delivery to the central nervous system? F1000Res. 2016 Mar 10;5. pii: F1000 Faculty Rev-313. doi: 10.12688/f1000research.7378.1.
Konofagou EE1, Tung YS, Choi J, Deffieux T, Baseri B, Vlachos F. Ultrasound-induced blood-brain barrier opening. Curr Pharm Biotechnol. 2012 Jun;13(7):1332-45.
Click here for additional references from PubMed.
