An experimental treatment approach for Alzheimer’s disease that uses microscopic gas bubbles excited by ultrasound energy to temporarily breach the blood-brain barrier and break up amyloid plaques is set to be tested in humans for the first time. According to a press release by the Queensland Brain Institute (QBI), a $10 million (ASD) government grant combined with public donations has provided nearly all the funding needed for a phase 1 trial. They anticipate to enroll and begin treating a small group of patients late next year.
Earlier this year, a University of Queensland research team published study results showing that their non-invasive technique successfully reversed dementia symptoms and lowered the number of plaques in elderly mice when combined with an antibody drug agent. Past investigations had demonstrated that the excited microbubbles alone are effective at removing plagues in a rodent model of Alzheimer’s.
"All brains change with age, potentially making them more fragile. As Alzheimer’s is an age-related disease, we wanted to investigate whether our ultrasound technology is safe for use on older brains," investigator Dr Gerhard Leinenga said in a statement in February. "The mice we treated in this study would be the human equivalent of 80 to 90 years old. With age, they are known to have cerebral amyloid angiopathy (CAA) – the build-up of toxic amyloid in blood vessels.”
“The technology temporarily opens the blood-brain-barrier to remove toxic plaques from the brain and has successfully reversed Alzheimer’s symptoms and restored memory function in animal models,” QBI Director Professor Pankaj Sah said in today’s statement.
The blood-brain barrier (BBB) is a highly selective shielding, made of specialized flattened cells, that wraps around all the blood vessels in the central nervous system. It serves to protect neural tissue from pathogens and toxins circulating in the bloodstream while letting in respiratory gases, sugars, and small signaling molecules like hormones. And though the BBB does a good job at preventing dangerous elements from entering the brain, it also blocks the entry of most drugs and limits movement and activation of immune cells.