Proteomimetic polymer blocks mitochondrial damage, rescues Huntington's neurons, and slows onset of neuropathology in vivo

Sci Adv. 2024 Nov;10(44):eado8307. doi: 10.1126/sciadv.ado8307. Epub 2024 Nov 1.

Abstract

Recently, it has been shown that blocking the binding of valosin-containing protein (VCP) to mutant huntingtin (mtHtt) can prevent neuronal mitochondrial autophagy in Huntington's disease (HD) models. Herein, we describe the development and efficacy of a protein-like polymer (PLP) for inhibiting this interaction in cellular and in vivo models of HD. PLPs exhibit bioactivity in HD mouse striatal cells by successfully inhibiting mitochondrial destruction. PLP is notably resilient to in vitro enzyme, serum, and liver microsome stability assays, which render analogous control oligopeptides ineffective. PLP demonstrates a 2000-fold increase in circulation half-life compared to peptides, exhibiting an elimination half-life of 152 hours. In vivo efficacy studies in HD transgenic mice (R6/2) confirm the superior bioactivity of PLP compared to free peptide through behavioral and neuropathological analyses. PLP functions by preventing pathologic VCP/mtHtt binding in HD animal models; exhibits enhanced efficacy over the parent, free peptide; and implicates the PLP as a platform with potential for translational central nervous system therapeutics.

MeSH terms

  • Animals
  • Disease Models, Animal*
  • Humans
  • Huntingtin Protein* / genetics
  • Huntingtin Protein* / metabolism
  • Huntington Disease* / drug therapy
  • Huntington Disease* / metabolism
  • Huntington Disease* / pathology
  • Mice
  • Mice, Transgenic*
  • Mitochondria* / drug effects
  • Mitochondria* / metabolism
  • Neurons* / drug effects
  • Neurons* / metabolism
  • Neurons* / pathology
  • Peptides / chemistry
  • Peptides / pharmacology
  • Polymers / chemistry
  • Polymers / pharmacology
  • Valosin Containing Protein / antagonists & inhibitors
  • Valosin Containing Protein / metabolism

Substances

  • Huntingtin Protein
  • Polymers
  • Valosin Containing Protein
  • Peptides