Focused Ultrasound Therapy
Focused ultrasound is an early-stage, non-invasive, therapeutic technology with the potential to improve the quality of life and decrease the cost of care for patients with Alzheimer’s disease. This novel technology focuses beams of ultrasonic energy precisely and accurately on targets deep in the brain without damaging surrounding normal tissue. Where the beams converge, the ultrasound produces a variety of therapeutic effects without incisions or radiation.
Currently, there is no cure for Alzheimer’s and few disease modifying therapies. Potentially promising drug and antibody therapies are often limited by their inability to efficiently cross the blood-brain barrier (BBB), a naturally occurring barrier of cells that inhibits the diffusion of drugs or toxins into the central nervous system.
Promising preclinical studies have demonstrated the potential of focused ultrasound to enhance the delivery of therapeutic drugs or antibodies to the brain. Focused ultrasound in combination with microbubbles can safely open the BBB and enable enhanced delivery of anti-amyloid antibodies to the brain. Studies suggest that this method can reduce the number of plaques and improve cognition in Alzheimer’s disease models. Furthermore, focused ultrasound and microbubbles alone (no antibodies) have had similar effects in two different Alzheimer’s models.
Based on this preclinical evidence, a human trial investigating repeated, temporary opening of the BBB in patients with mild to moderate Alzheimer’s disease was recently completed. Read the press release >
- Focused ultrasound is non-invasive – no incisions, holes in the skull, electrodes in the brain – and therefore has reduced risk for infection and blood clots.
- Precise targeting minimizes damage to non-targeted healthy brain.
- Treatment can be a complement to drug therapy, enabling enhanced delivery of disease-modifying therapies into the brain via temporary opening of the blood-brain barrier.
A clinical trial investigating the repeated opening of the BBB in Alzheimer’s patients at Sunnybrook Health Sciences Centre in Toronto, Canada, has been published in Nature Communications. The trial aimed to assess the safety, feasibility and reversibility of BBB opening with focused ultrasound as well as the reproducibility of repeated BBB opening in human patients. Read the press release >
A Phase II trial at Sunnybrook Health Sciences Centre in Toronto, Canada, has begun. This 30-patient trial will investigate the use of focused ultrasound to temporarily open the BBB in multiple regions of the brain.
A clinical trial in the US is opening the BBB with MR-guided focused ultrasound in patients with Alzheimer’s disease.
Another clinical trial in the US is opening the BBB with a neuronavigation device in patients with Alzheimer’s disease.
A third clinical trial in the US is using low-intensity focused ultrasound pulsation (LIFUP) to treat patients with early Alzheimer’s disease.
Two additional clinical trials in the US are treating patients with neurodegenerative dementia in Los Angeles, California. These studies are only recruiting patients by invitation.
A clinical trial in Paris, France, is opening the BBB in patients with Alzheimer’s disease.
Preclinical Laboratory Studies
Preclinical studies suggest focused ultrasound’s potential to reduce amyloid plaques and improve cognition in Alzheimer’s models. Active studies are assessing focused ultrasound-induced BBB opening to improve the delivery of:
- Anti-amyloid antibodies and other disease modifying drugs (Sunnybrook Health Sciences Centre; Columbia University; Queensland University – Australia; Brigham and Women’s Hospital)
- Anti-tau antibodies (Queensland University – Australia).
Suggested Reading: Focused Ultrasound for Alzheimer's Disease. March 2019.
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Lipsman N, Meng Y, Bethune AJ, Huang Y, Lam B, Masellis M, Herrmann N, Heyn C, Aubert I, Boutet A, Smith GS, Hynynen K, Black SE. Blood-brain barrier opening in Alzheimer's disease using MR-guided focused ultrasound. Nat Commun. 2018 Jul 25;9(1):2336. doi: 10.1038/s41467-018-04529-6.
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