Characterization of novel conformation-selective α-synuclein antibodies as potential immunotherapeutic agents for Parkinson's disease

Neurobiol Dis. 2020 Mar:136:104712. doi: 10.1016/j.nbd.2019.104712. Epub 2019 Dec 16.

Abstract

Parkinson's disease (PD) and dementia with Lewy bodies (DLB) are progressive neurodegenerative diseases for which there is no disease-modifying treatment. PD and DLB are characterized by aggregation of the synaptic protein α-synuclein, and there is compelling evidence to suggest that progression of these diseases is associated with the trans-cellular spread of pathogenic α-synuclein through the brains of afflicted individuals. Therapies targeting extracellular, pathogenic α-synuclein may therefore hold promise for slowing or halting disease progression. In this regard, it has been suggested that highly-selective antibodies can be administered as therapeutic agents targeting pathogenic proteins. In the current study, we screened a series of antibodies using multiple selection criterion to identify those that selectively bind pathogenic α-synuclein and show potent inhibition of pathology seeding in a neuronal model of α-synucleinopathy. A lead antibody was tested in a mouse model of PD, and it was able to reduce the spread of α-synuclein pathology in the brain and attenuate dopamine reductions in the striatum. This study highlights the therapeutic potential of α-synuclein immunotherapy for the treatment of PD and DLB, and provides a framework for screening of α-synuclein antibodies to identify those with preferred properties.

Keywords: Animal model; Antibody; Immunotherapy; Intervention; Misfolded; Protein spread; SNCA; Transmission.

Publication types

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

MeSH terms

  • Animals
  • Antibodies, Monoclonal / administration & dosage*
  • Antibodies, Monoclonal / chemistry
  • Antibodies, Monoclonal / genetics
  • Cells, Cultured
  • Dose-Response Relationship, Immunologic
  • Female
  • Humans
  • Immunotherapy / methods*
  • Lewy Body Disease / genetics
  • Lewy Body Disease / immunology*
  • Lewy Body Disease / therapy*
  • Male
  • Mice
  • Mice, Inbred BALB C
  • Parkinson Disease / genetics
  • Parkinson Disease / immunology*
  • Parkinson Disease / therapy*
  • alpha-Synuclein / administration & dosage*
  • alpha-Synuclein / chemistry
  • alpha-Synuclein / genetics

Substances

  • Antibodies, Monoclonal
  • alpha-Synuclein