Amyotrophic Lateral Sclerosis (ALS)

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Focused Ultrasound Therapy

Focused ultrasound is a rapidly evolving, 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.

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

clinical trial for temporarily opening the BBB for ALS patients is about to start recruiting more patients in Canada. This study is ONLY open to Canadian citizens. The initial report of the first 4 patients shows that the BBB of the motor region (target) can be reversibly opened by focused ultrasound without untoward side effects. This study opens the door for combining focused ultrasound BBB opening with several possible therapeutic agents.

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

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.

Clinical Trials