Researchers at Sunnybrook Health Sciences Centre in Canada are paving the way for a new treatment using focused ultrasound to treat ALS.
Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease, is a devastating and fatal neurological disorder. As the disease progresses people with ALS become paralyzed as the brain can no longer send messages and communicate with their muscles. Over time, due to the muscles breaking down in the body, ALS results in patients losing the ability to walk, talk, eat, swallow, and eventually breathe. There is no cure for ALS.
The disease affects approximately 3,000 Canadians per year, with 250 new patients diagnosed every year. However, there are still gaps in understanding the physical changes associated with the disease and current interventions only slightly slow down the disease progression. Also, current treatments to deliver therapeutics to the brain require invasive surgical procedures and can have serious complications.
Researchers in Canada are taking ALS treatment to the next level with a potential new approach. The results of this world-first trial using focused ultrasound to treat ALS was published recently in Nature Communications. The innovative and novel approach is a direct and non-invasive approach to access the brain and test promising therapeutics targeted to slow and hopefully stop the disease from progressing.
Focused ultrasound therapy harnesses the power of up to one thousand sound waves. In the study, the therapy was used at a low frequency to target a specific area of the brain. Microbubbles, which are injected into the bloodstream begin to vibrate due to the ultrasound. This gentle process results in the blood-brain barrier being opened temporarily. The blood-brain barrier protects the brain from toxins and bacteria. However, it also inhibits medications and therapeutics which could benefit patients such as those with ALS.
The first phase of this study was to show the feasibility and safety of this novel approach to access the brain through the blood-brain barrier in humans. The study found that the focused ultrasound approach, which causes microbubbles to vibrate, could successfully and safely open the blood-brain barrier of patients. This process was reversible, and no subjects were found to have any serious adverse events following the procedure.
The next phase of the study – coming in the next few months – is to begin delivering ALS therapeutics and investigate the novel, direct-to-brain interaction. The study demonstrated that focused ultrasound could potentially be a step in the right direction to treat ALS patients.
Written by Lacey Hizartzidis, PhD
Sunnybrook research helps take future treatment of ALS patients to a new level. Sunnybrook Health Sciences Centre website https://sunnybrook.ca/media/item.asp?c=1&i=1979&f=als-research-new-level. Accessed September 27, 2019.
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.
Image by Raman Oza from Pixabay