Metal Protein-Attenuating Compound for PET Neuroimaging: Synthesis and Preclinical Evaluation of [11C]PBT2

Mol Pharm. 2018 Feb 5;15(2):695-702. doi: 10.1021/acs.molpharmaceut.7b00936. Epub 2018 Jan 22.

Abstract

Dyshomeostasis or abnormal accumulation of metal ions such as copper, zinc, and iron have been linked to the pathogenesis of multiple neurodegenerative disorders including Alzheimer's disease (AD) and Huntington's disease (HD). 5,7-Dichloro-2-((dimethylamino)methyl)quinolin-8-ol, PBT2, is a second generation metal protein-attenuating compound that has recently advanced in Phase II clinical trials for the treatment of AD and HD based on promising preclinical efficacy data. Herein, we report the first radiosynthesis and preclinical positron emission tomography (PET) neuroimaging evaluation of [11C]PBT2 in rodents and nonhuman primates. Carbon-11 labeled PBT2 was synthesized in 4.8 ± 0.5% (nondecay corrected) radiochemical yield (RCY) at end-of-synthesis, based upon [11C]CH3I (n = 6), with >99% radiochemical purity and 80-90 GBq/μmol molar activity (Am) from the corresponding normethyl precursor. In the nonhuman primate brain, [11C]PBT2 uptake was extensive with peak concentration SUVpeak of 3.2-5.2 within 2.5-4.5 min postinjection in all cortical and subcortical gray matter regions (putamen > caudate > cortex ≫ white matter) followed by rapid washout from normal brain tissues. Furthermore, it is shown that [11C]PBT2 binds specifically in AD human brain tissue in vitro. The results presented here, combined with the clinical data available for PBT2, warrant the evaluation of [11C]PBT2 as an exploratory PET radiotracer in humans.

Keywords: 11C-labeled PBT2; PBT2; PET; amyloid-β plaques; carbon-11; metal hypothesis of Alzheimer’s disease; neuroimaging; positron emission tomography.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Alzheimer Disease / pathology
  • Animals
  • Autoradiography
  • Brain / diagnostic imaging
  • Brain / metabolism
  • Brain / pathology
  • Carbon Radioisotopes*
  • Clioquinol / administration & dosage
  • Clioquinol / analogs & derivatives*
  • Clioquinol / chemical synthesis
  • Clioquinol / pharmacokinetics
  • Drug Evaluation, Preclinical
  • Female
  • Humans
  • Male
  • Mice, Inbred BALB C
  • Neuroimaging / methods*
  • Papio anubis
  • Positron-Emission Tomography / methods*
  • Radiopharmaceuticals / administration & dosage*
  • Radiopharmaceuticals / chemical synthesis
  • Radiopharmaceuticals / pharmacokinetics

Substances

  • Carbon Radioisotopes
  • Carbon-11
  • PBT2 compound
  • Radiopharmaceuticals
  • Clioquinol