MIT Technology Review by Cassandra Willyard — Therapies to treat brain diseases share a common problem: they struggle to reach their target. The blood vessels that permeate the brain have a special lining so tightly packed with cells that only very tiny molecules can pass through. This blood-brain barrier “acts as a seal,” protecting the brain from toxins or other harmful substances, says Anne Eichmann, a molecular biologist at Yale. But it also keeps most medicines out. Researchers have been working on methods to sneak drugs past the blood-brain barrier for decades. And their hard work is finally beginning to pay off. Last week, researchers at the West Virginia University Rockefeller Neuroscience Institute reported that by using focused ultrasound to open the blood-brain barrier, they improved delivery of a new Alzheimer’s treatment and sped up clearance of the sticky plaques that are thought to contribute to some of the cognitive and memory problems in people with Alzheimer’s by 32%. For this issue of The Checkup, we’ll explore some of the ways scientists are trying to disrupt the blood-brain barrier. A patient surrounded by a medical team lays on the bed of an MRI machine with their head in a special focused ultrasound helmet
In the West Virginia study, three people with mild Alzheimer’s received monthly doses of aducanumab, a lab-made antibody that is delivered via IV. This drug, first approved in 2021, helps clear away beta-amyloid, a protein fragment that clumps up in the brains of people with Alzheimer’s disease. (The drug’s approval was controversial, and it’s still not clear whether it actually slows progression of the disease.) After the infusion, the researchers treated specific regions of the patients’ brains with focused ultrasound, but just on one side. That allowed them to use the other half of the brain as a control. PET scans revealed a greater reduction in amyloid plaques in the ultrasound-treated regions than in those same regions on the untreated side of the brain, suggesting that more of the antibody was getting into the brain on the treated side.