Blood-brain barrier opening in Alzheimer's disease using MR-guided focused ultrasound

Nat Commun. 2018 Jul 25;9(1):2336. doi: 10.1038/s41467-018-04529-6.

Abstract

Magnetic resonance-guided focused ultrasound in combination with intravenously injected microbubbles has been shown to transiently open the blood-brain barrier, and reduce beta-amyloid and tau pathology in animal models of Alzheimer's disease. Here, we used focused ultrasound to open the blood-brain barrier in five patients with early to moderate Alzheimer's disease in a phase I safety trial. In all patients, the blood-brain barrier within the target volume was safely, reversibly, and repeatedly opened. Opening the blood-brain barrier did not result in serious clinical or radiographic adverse events, as well as no clinically significant worsening on cognitive scores at three months compared to baseline. Beta-amyloid levels were measured before treatment using [18F]-florbetaben PET to confirm amyloid deposition at the target site. Exploratory analysis suggested no group-wise changes in amyloid post-sonication. The results of this safety and feasibility study support the continued investigation of focused ultrasound as a potential novel treatment and delivery strategy for patients with Alzheimer's disease.

Publication types

  • Clinical Trial, Phase I
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Aged, 80 and over
  • Alzheimer Disease / diagnostic imaging*
  • Amyloid beta-Peptides / chemistry
  • Aniline Compounds / chemistry
  • Biological Transport
  • Blood-Brain Barrier*
  • Brain / diagnostic imaging*
  • Female
  • Humans
  • Magnetic Resonance Imaging*
  • Male
  • Microbubbles
  • Middle Aged
  • Positron Emission Tomography Computed Tomography
  • Prospective Studies
  • Psychometrics
  • Stilbenes / chemistry
  • Ultrasonography*

Substances

  • Amyloid beta-Peptides
  • Aniline Compounds
  • Stilbenes
  • 4-(N-methylamino)-4'-(2-(2-(2-fluoroethoxy)ethoxy)ethoxy)stilbene