Evaluation of Small-Animal PET Outcome Measures to Detect Disease Modification Induced by BACE Inhibition in a Transgenic Mouse Model of Alzheimer Disease

J Nucl Med. 2017 Dec;58(12):1977-1983. doi: 10.2967/jnumed.116.187625. Epub 2017 Jun 13.

Abstract

In this study, we investigated the effects of chronic administration of an inhibitor of the β-site amyloid precursor protein-cleaving enzyme 1 (BACE1) on Alzheimer-related pathology by multitracer PET imaging in transgenic APPPS1-21 (TG) mice. Methods: Wild-type (WT) and TG mice received vehicle or BACE inhibitor (60 mg/kg) starting at 7 wk of age. Outcome measures of brain metabolism, neuroinflammation, and amyloid-β pathology were obtained through small-animal PET imaging with 18F-FDG, 18F-peripheral benzodiazepine receptor (18F-PBR), and 18F-florbetapir (18F-AV45), respectively. Baseline scans were acquired at 6-7 wk of age and follow-up scans at 4, 7, and 12 mo. 18F-AV45 uptake was measured at 8 and 13 mo of age. After the final scans, histologic measures of amyloid-β (4G8), microglia (ionized calcium binding adaptor molecule 1), astrocytes (glial fibrillary acidic protein), and neuronal nuclei were performed. Results: TG mice demonstrated significant age-associated increases in 18F-AV45 uptake. An effect of treatment was observed in the cortex (P = 0.0014), hippocampus (P = 0.0005), and thalamus (P < 0.0001). Histology confirmed reduction of amyloid-β pathology in TG-BACE mice. Regardless of treatment, TG mice demonstrated significantly lower 18F-FDG uptake than WT mice in the thalamus (P = 0.0004) and hippocampus (P = 0.0332). Neuronal nucleus staining was lower in both TG groups in the thalamus and cortex. 18F-PBR111 detected a significant age-related increase in TG mice (P < 0.0001) but did not detect the treatment-induced reduction in activated microglia as demonstrated by histology. Conclusion: Although 18F-FDG, 18F-PBR111, and 18F-AV45 all detected pathologic alterations between TG and WT mice, only 18F-AV45 could detect an effect of BACE inhibitor treatment. However, changes in WT binding of 18F-AV45 undermine the specificity of this effect.

Keywords: Alzheimer’s disease; BACE inhibitor; longitudinal; small animal PET.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aging
  • Alzheimer Disease / diagnostic imaging*
  • Alzheimer Disease / drug therapy*
  • Alzheimer Disease / pathology
  • Amyloid Neuropathies / pathology
  • Amyloid Precursor Protein Secretases / antagonists & inhibitors*
  • Amyloid beta-Peptides / metabolism
  • Aniline Compounds
  • Animals
  • Aspartic Acid Endopeptidases / antagonists & inhibitors*
  • Brain / diagnostic imaging
  • Brain / pathology
  • Brain Chemistry
  • Enzyme Inhibitors / therapeutic use
  • Ethylene Glycols
  • Fluorodeoxyglucose F18
  • Humans
  • Inflammation / pathology
  • Mice
  • Mice, Transgenic
  • Positron-Emission Tomography
  • Radiopharmaceuticals
  • Treatment Outcome

Substances

  • Amyloid beta-Peptides
  • Aniline Compounds
  • Enzyme Inhibitors
  • Ethylene Glycols
  • Radiopharmaceuticals
  • Fluorodeoxyglucose F18
  • florbetapir
  • Amyloid Precursor Protein Secretases
  • Aspartic Acid Endopeptidases
  • BACE1 protein, human