{"id":2978,"date":"2021-12-08T20:47:00","date_gmt":"2021-12-09T00:47:00","guid":{"rendered":"https:\/\/fusfoundation.org\/?post_type=sw_disease_condition&#038;p=2978"},"modified":"2026-04-24T13:47:32","modified_gmt":"2026-04-24T17:47:32","slug":"amyotrophic-lateral-sclerosis-als","status":"publish","type":"sw_disease_condition","link":"https:\/\/www.fusfoundation.org\/diseases-and-conditions\/amyotrophic-lateral-sclerosis-als\/","title":{"rendered":"Amyotrophic Lateral Sclerosis (ALS)"},"content":{"rendered":"\n<div class=\"wp-block-buttons is-layout-flex wp-block-buttons-is-layout-flex\">\n<div class=\"wp-block-button has-custom-width wp-block-button__width-50 is-style-fill\"><a class=\"wp-block-button__link has-background wp-element-button\" href=\"https:\/\/www.fusfoundation.org\/the-technology\/treatment-sites\/?indication=Amyotrophic%20Lateral%20Sclerosis%20%28ALS%29\" style=\"background-color:#086da8\" target=\"_blank\" rel=\"noreferrer noopener\">Find a Treatment Site or Clinical Trial<\/a><\/div>\n<\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-overview\">Overview<\/h2>\n\n\n\n<p>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.<\/p>\n\n\n\n<p><strong>How it Works<\/strong><br>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.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-benefits\">Benefits<\/h2>\n\n\n\n<p>There is no cure for ALS and current therapies focus on slowing disease progression and symptom management. However, the blood brain barrier can prevent therapies from reaching the brain in adequate concentrations. Focused ultrasound blood-brain and blood-spinal barrier opening has the potential to enhance the delivery of disease modifying therapies (DMTs) such as small molecules and gene therapy to treat ALS more effectively. Additionally, focused ultrasound neuromodulation may be able to augment nerve firing to prevent and slow muscle weakness.<\/p>\n\n\n\n<p>Advantages:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Noninvasive &#8211; no incisions, no risk of infection or bleeding, and rapid recovery<\/li>\n\n\n\n<li>Image-guided &#8211; minimal damage to surrounding tissue<\/li>\n\n\n\n<li>Safe, temporary, and repetitive opening of the blood brain barrier &#8211; enhancing the delivery of therapeutics directly to the target site within the brain<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-regulatory-authorizations\">Regulatory Authorizations<\/h2>\n\n\n\n<p>Focused ultrasound treatment for ALS is not yet approved by regulatory bodies or covered by medical insurance companies.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-clinical-trials\">Clinical Trials<\/h2>\n\n\n\n<p>A <a href=\"https:\/\/clinicaltrials.gov\/search?term=NCT07193953\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>clinical trial<\/strong><\/a> is using the next generation dome helmet with intravenous immunoglobulin (IVIg) to treat patients with ALS in Toronto, Canada.<\/p>\n\n\n\n<p>Another <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT05983211?term=nct05983211&amp;rank=1\" target=\"_blank\" rel=\"noreferrer noopener\"><strong><\/strong><strong>clinical trial<\/strong><\/a> in Toronto, Canada is using repetitive transcranial magnetic stimulation with continuous theta-burst stimulation to help patients with ALS.&nbsp;&nbsp;<\/p>\n\n\n\n<p>A <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT06681610?term=nct06681610&amp;rank=1\" target=\"_blank\" rel=\"noreferrer noopener\"><strong><\/strong><strong>clinical trial<\/strong><\/a> in in Spain is using transcranial pulse stimulation to see if that can help patients with ALS.&nbsp;&nbsp;<\/p>\n\n\n\n<p>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 <a href=\"https:\/\/www.clinicaltrials.gov\/study\/NCT03321487?cond=ALS&amp;term=focused%20ultrasound&amp;rank=1\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>here<\/strong><\/a>, it will take you to the latest information available from <a href=\"https:\/\/www.clinicaltrials.gov\/\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>https:\/\/www.clinicaltrials.gov\/<\/strong><\/a>.&nbsp;<\/p>\n\n\n\n<p><a href=\"https:\/\/clinicaltrials.gov\/ct2\/results?cond=ALS&amp;term=focused+ultrasound&amp;cntry=&amp;state=&amp;city=&amp;dist=\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>See a full list of ALS clinical trials ><\/strong><\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"h-notable-papers\">Notable Papers<\/h2>\n\n\n\n<p><strong><em>Suggested Reading:<a href=\"https:\/\/fusf.sharepoint.com\/sites\/FUSF-Development\/Shared%20Documents\/Forms\/AllItems.aspx?id=%2Fsites%2FFUSF%2DDevelopment%2FShared%20Documents%2FFUS%5FNeurodegenerative%2Epdf&amp;parent=%2Fsites%2FFUSF%2DDevelopment%2FShared%20Documents&amp;p=true&amp;wdLOR=cF08C24CE%2D3E05%2D48D3%2D9B9C%2DEB0891A6E8D0&amp;ct=1661384679775&amp;or=Outlook-Body&amp;cid=0AF0D68D-F13B-4C1C-B5D7-83C9623A7D69&amp;ga=1\" target=\"_blank\" rel=\"noreferrer noopener\"> Focused Ultrasound: Transforming treatment of Neurodegenerative Diseases <\/a>August 2022<\/em><\/strong><\/p>\n\n\n\n<p>Martin E, Roberts M, Grigoras IF, Wright O, Nandi T, Rieger SW, Campbell J, den Boer T, Cox BT, Stagg CJ, Treeby BE. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/40913042\/\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>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&nbsp;<\/p>\n\n\n\n<p>Yang HM. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/40871062\/\" target=\"_blank\" rel=\"noreferrer noopener\"><strong>Overcoming the Blood-Brain Barrier: Advanced Strategies in Targeted Drug Delivery for Neurodegenerative Diseases<\/strong><\/a>. Pharmaceutics. 2025 Aug 11;17(8):1041. doi: 10.3390\/pharmaceutics17081041. PMID: 40871062&nbsp;<\/p>\n\n\n\n<p>Ediriweera GR, Sivaram AJ, Cowin G, Brown ML, McAlary L, Lum JS, Fletcher NL, Robinson L, Simpson JD, Chen L, Wasielewska JM, Byrne E, Finnie JW, Manavis J, White AR, Yerbury JJ, Thurecht KJ, Vine KL. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/39645085\/\" target=\"_blank\" rel=\"noreferrer noopener\" class=\"ek-link\"><strong>Lipid nanoparticles and transcranial focused ultrasound enhance the delivery of SOD1 antisense oligonucleotides to the murine brain for ALS therapy<\/strong><\/a>. J Control Release. 2024 Dec 12;378:221-235. doi: 10.1016\/j.jconrel.2024.11.074. PMID: 39645085&nbsp;<\/p>\n\n\n\n<p>Mehta RI, Ranjan M, Haut MW, Carpenter JS, Rezai AR. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/39322357\/\" target=\"_blank\" rel=\"noreferrer noopener\" class=\"ek-link\"><strong>Focused Ultrasound for Neurodegenerative Diseases<\/strong><\/a>. Magn Reson Imaging Clin N Am. 2024 Nov;32(4):681-698. doi: 10.1016\/j.mric.2024.03.001. Epub 2024 Jul 26. PMID: 39322357&nbsp;<\/p>\n\n\n\n<p>Wasielewska JM, Chaves JCS, Cabral-da-Silva MC, Pecoraro M, Viljoen SJ, Nguyen TH, Bella V, Oikari LE, Ooi L, White AR. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/39138578\/\" target=\"_blank\" rel=\"noreferrer noopener\">A patient-derived amyotrophic lateral sclerosis blood-brain barrier model for focused ultrasound-mediated anti-TDP-43 antibody delivery<\/a>. Fluids Barriers CNS. 2024 Aug 13;21(1):65. doi: 10.1186\/s12987-024-00565-1. PMID: 39138578&nbsp;<\/p>\n\n\n\n<p>Zhang J, Chen K, Chen Y, Hua L, Chen S, Chen X, Zou L, Li S, Yang X, Shen Y. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/39217855\/\" target=\"_blank\" rel=\"noreferrer noopener\" class=\"ek-link\">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<\/a>. Ultrasonics. 2024 Aug 29;144:107449. doi: 10.1016\/j.ultras.2024.107449. PMID: 39217855&nbsp;<\/p>\n\n\n\n<p>Shen Y, Zhang J, Xu Y, Sun S, Chen K, Chen S, Yang X, Chen X. <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/36931463\/\" target=\"_blank\" rel=\"noreferrer noopener\" class=\"ek-link\"><strong>Ultrasound-enhanced brain delivery of edaravone provides additive amelioration on disease progression in an ALS mouse model<\/strong><\/a>. Brain Stimul. 2023 Mar 16;16(2):628-641. doi: 10.1016\/j.brs.2023.03.006.<\/p>\n\n\n\n<p>Ediriweera GR, Chen L, Yerbury JJ, Thurecht KJ, Vine KL.&nbsp;<a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/34010004\/\" target=\"_blank\" rel=\"noreferrer noopener\">Non-Viral Vector-Mediated Gene Therapy for ALS: Challenges and Future Perspectives.&nbsp;<\/a>Mol Pharm. 2021 May 19. doi: 10.1021\/acs.molpharmaceut.1c00297.<\/p>\n\n\n\n<p>Abrahao A, Meng Y, Llinas M, Huang Y, Hamani C, Mainprize T, Aubert I, Heyn C, Black SE, Hynynen K, Lipsman N, Zinman L.&nbsp;<a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pubmed\/31558719\" target=\"_blank\" rel=\"noreferrer noopener\">First-in-human trial of blood-brain barrier opening in amyotrophic lateral sclerosis using MR-guided focused ultrasound.<\/a>&nbsp;Nat Commun. 2019 Sep 26;10(1):4373. doi: 10.1038\/s41467-019-12426-9.<\/p>\n\n\n\n<p>Jones RM, Hynynen K.&nbsp;<a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pubmed\/30507302\" target=\"_blank\" rel=\"noreferrer noopener\">Advances in acoustic monitoring and control of focused ultrasound-mediated increases in blood-brain barrier permeability.<\/a>&nbsp;Br J Radiol. 2018 Dec 3:20180601. doi: 10.1259\/bjr.20180601.<\/p>\n\n\n\n<p>Saunders NR, Habgood MD, M\u00f8llg\u00e5rd K, Dziegielewska KM.&nbsp;<a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pubmed\/26998242\" target=\"_blank\" rel=\"noreferrer noopener\">The biological significance of brain barrier mechanisms: help or hindrance in drug delivery to the central nervous system?&nbsp;<\/a>F1000Res. 2016 Mar 10;5. pii: F1000 Faculty Rev-313. doi: 10.12688\/f1000research.7378.1.<br><br>Konofagou EE1, Tung YS, Choi J, Deffieux T, Baseri B, Vlachos F.<a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pubmed\/22201586\" target=\"_blank\" rel=\"noreferrer noopener\">&nbsp;Ultrasound-induced blood-brain barrier opening.<\/a>&nbsp;Curr Pharm Biotechnol. 2012 Jun;13(7):1332-45.<\/p>\n\n\n\n<p><strong><a href=\"https:\/\/www.ncbi.nlm.nih.gov\/pubmed\/?term=(%22focused+ultrasound%22+OR+HIFU)+and+%22Amyotrophic+Lateral+Sclerosis%22\" target=\"_blank\" rel=\"noreferrer noopener\">Click here<\/a> for additional references from PubMed.<\/strong><\/p>\n","protected":false},"template":"","meta":{"_acf_changed":false,"_searchwp_excluded":"","_editorskit_title_hidden":false,"_editorskit_reading_time":0,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","episode_type":"","audio_file":"","podmotor_file_id":"","podmotor_episode_id":"","cover_image":"","cover_image_id":"","duration":"","filesize":"","filesize_raw":"","date_recorded":"","explicit":"","block":"","itunes_episode_number":"","itunes_title":"","itunes_season_number":"","itunes_episode_type":"","_vp_format_video_url":"","_vp_image_focal_point":[],"footnotes":""},"sw_disease_condition_cat":[6],"sw_disease_condition_stage":[20],"class_list":["post-2978","sw_disease_condition","type-sw_disease_condition","status-publish","hentry","sw_disease_condition_cat-neurological"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.2 (Yoast SEO v27.2) - 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