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.
A 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.
Regulatory Approval and Reimbursement
Focused ultrasound treatment for ALS is not yet approved by regulatory bodies or covered by medical insurance companies.
Suggested Reading: Focused Ultrasound: Transforming treatment of Neurodegenerative Diseases August 2022
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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.
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