Polyethylene Glycol-Poly-Lactide-co-Glycolide Block Copolymer-Based Nanoparticles as a Potential Tool for Off-Label Use of N-Acetylcysteine in the Treatment of Diastrophic Dysplasia

J Pharm Sci. 2017 Dec;106(12):3631-3641. doi: 10.1016/j.xphs.2017.08.004. Epub 2017 Aug 19.

Abstract

Potential off-label therapeutic role of N-acetylcysteine (N-Ac) was recently demonstrated in the treatment of diastrophic dysplasia (DTD) using mutant mice; its main drawback is the rapid clearance from blood due to the liver metabolism. Our goal was to investigate the potential of polyethylene glycol polylactide-co-glycolide block copolymer (PLGA-PEG)-based nanoparticles (NPs) in order to improve in vivo biodistribution performances and N-Ac pharmacokinetic profile after subcutaneous administration in mice. Results suggest that N-Ac can be effectively loaded into NPs (about 99 μg/mg NPs) using a suitably optimized nanoprecipitation method. Thanks to the good physical characteristics (mean diameter <100 nm, zeta potential about -8 mV) NPs can reach skeletal tissue in particular femoral head and proximal tibia epiphysis at the sixth hour after injection, remaining in the tissues till 24 h. Furthermore, pharmacokinetic study revealed a sustained N-Ac concentration in plasma with a peak concentration of 2.48 ± 1.72 μM at the 24th hour after injection. Overall, results highlight the actual interest of N-Ac-loaded PLGA-PEG NPs as useful platform for N-Ac parenteral administration.

Keywords: PLGA; biodegradable polymers; freeze-drying/lyophilization; nanoparticles; nanotechnology; polymeric drug delivery systems.

MeSH terms

  • Acetylcysteine / chemistry*
  • Acetylcysteine / pharmacology*
  • Animals
  • Drug Carriers / chemistry
  • Dwarfism / drug therapy*
  • Mice
  • Mice, Inbred C57BL
  • Nanoparticles / administration & dosage
  • Nanoparticles / chemistry*
  • Off-Label Use
  • Particle Size
  • Polyesters / chemistry*
  • Polyethylene Glycols / chemistry*
  • Polymers / chemistry
  • Tissue Distribution

Substances

  • Drug Carriers
  • Polyesters
  • Polymers
  • polyethylene glycol-poly(lactide-co-glycolide)
  • Polyethylene Glycols
  • Acetylcysteine

Supplementary concepts

  • Diastrophic dysplasia