Alzheimer’s disease is marked by a progressive decline in memory and cognition, and it is the most common neurodegenerative disorder.1At this year’s Alzheimer’s Association International Conference (AAIC), the largest international meeting devoted to developing and sharing the latest research related to this disease,2the results of an innovative clinical trial using focused ultrasound for facilitating drug therapy in patients with Alzheimer’s disease were unveiled. This clinical trial was the first in a multi-phase proposal to test the safety and effectiveness of using focused ultrasound as a treatment intervention.1The ultrasound treatment requires no surgery and could be utilized to deliver pharmaceuticals specifically to the regions of the brain impacted by the disease, representing a potential breakthrough in the treatment of Alzheimer’s.1,2
The blood brain barrier, which separates the bloodstream from brain tissue itself, often limits potentially effective medicines and immune responses from reaching diseased areas of the brain.3The researchers involved in these studies believe that temporarily delivering antibodies through temporary openings in the blood brain barrier that reduce the amount of damaging plaques in the brain could help to improve the memory and cognitive processes typically impacted as a result of the disease.1,3
Current Treatment Strategies
Up to this point, dementia experts have not been able to isolate the exact underlying causes of Alzheimer’s. Due to this, developing medical treatments and methods for preventing Alzheimer’s has been a difficult endeavor for researchers to tackle. There is currently no cure for Alzheimer’s disease, nor is there a way to stop or slow down its progression.4 However, there are a number of treatment strategy options available to help treat the various symptoms associated with this disease.
The Food and Drug Administration (FDA) has approved two classes of medications, Cholinesterase inhibitors (Aricept, Exelon, Razadyne) and Memantine (Namenda) to treat the cognitive symptoms such as memory loss, confusion, and difficulty thinking and reasoning. Other medications, behavioral strategies, and alternative treatments (e.g. dietary supplements, herbal remedies) have also been promoted to aid in the management of cognitive, sleep, and behavioral changes.5
Phase I Safety Trial
In the phase I trial, six patients with Alzheimer’s disease (ages 50-85) underwent two separate focused ultrasound procedures targeted at safely and temporarily opening the blood brain barrier.2,3The treatment lasted about three hours, which included MRI scans in addition to the ultrasound treatment. Following the treatments the patients were in good spirits according to the press release and discharged to their homes within the same day.3
No therapeutic drugs were administered in this initial safety trial. Rather than delivering medications, microscopic air bubbles were injected into the bloodstream.1When the air bubbles reach the area of the brain being targeted and are exposed to the ultrasound, the blood brain barrier has been successfully opened during each of the trials thus far.3,6The researchers hypothesize that in the future the focused ultrasound treatments could be utilized to temporarily open the blood brain barrier for precisely targeted drug delivery in therapeutic concentrations.1-3
Phase II and the Future
Phase II of the clinical trials is currently underway at the West Virginia University Rockefeller Neuroscience Institute, which will evaluate whether focused ultrasound reduces the amount of plaque and cognitive decline in patients with Alzheimer’s. On October 16, 2018 the first phase II patient, a 61-year-old female nurse with early-stage Alzheimer’s, underwent the focused ultrasound treatment procedure.3
According to researchers, the potential benefits of the focused ultrasound treatments will likely take several years to evaluate.3However, they are hopeful that this could become a valuable treatment option for patients who are dealing with the challenges associated with this difficult, prevalent disease.
- Lipsman N, Meng Y, Bethune AJ, et al. Blood–brain barrier opening in Alzheimer’s disease using MR-guided focused ultrasound. Nature Communications. 2018;9(1). doi:10.1038/s41467-018-04529-6
- Kassell NF. Potential new treatment for Alzheimer’s disease. Focusedultrasoundfoundation.org. https://www.fusfoundation.org/the-foundation/news-media/blog/potential-new-treatment-for-alzheimers-disease. Published July 25, 2018. Accessed February 19, 2019.
- West Virginia University Rockefeller Neuroscience Institute Pioneers Promising New Alzheimer’s Therapy. Wvumedicine.org. https://wvumedicine.org/news/article/west-virginia-university-rockefeller-neuroscience-institute-pioneers-promising-new-alzheimer-s-thera/. Published October 17, 2018. Accessed February 19, 2019.
- Causes and risk factors. Alz.org. https://www.alz.org/alzheimers-dementia/what-is-alzheimers/causes-and-risk-factors. Accessed February 19, 2019.
- Treatments. Alz.org. https://www.alz.org/alzheimers-dementia/treatments. Accessed February 19, 2019.
- Rohman M. Researchers successfully open blood-brain barrier with focused ultrasound in Alzheimer’s patient.Healthimaging.com. https://www.healthimaging.com/topics/advanced-visualization/researchers-open-blood-brain-barrier-focused-ultrasound. Published October 29, 2018. Accessed February 19, 2019.