Central Nervous System (CNS) diseases affect millions of patients as well as their caregivers worldwide. More than any other CNS disease, the prevalence of Alzheimer’s (AD) is 5.2 million patients in the U.S. only, while it is predicted to affect 1 in 85 people globally by 2050.
Currently, there is no treatment for AD; therapies are limited to symptomatic relief and the main impediment for the treatment of AD is the blood-brain barrier (BBB). The BBB is a physical and metabolic barrier that protects the brain’s microenvironment and excludes 98% of small molecule drugs and 100% of large-molecule drugs from reaching the brain parenchyma. Focused Ultrasound in conjunction with systemically-administered microbubbles has been shown to be the only technique thus far to non-invasively, locally and temporarily open the BBB and induce trans-BBB diffusion of molecules of various sizes, and as large as up to 50 nm in hydrodynamic diameter.
Given the urgent need for treatment of CNS diseases, and AD in particular, and having demonstrated feasibility and efficacy for BBB opening in normal as well as disease-model mice and NHPs over the past decade, our group is ready to develop and test a clinical FUS BBB opening system for the treatment of AD in humans. The objectives of this proposed study are thus to develop a human prototype and establish its safety and efficacy in NHP and safety in humans. Our multidisciplinary team encompasses all critical specialty areas such as ultrasound engineering, Alzheimer’s pathophysiology in animals and humans, neuroscience, histological and behavioral studies as well as MR and microscopy imaging expertise