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Drug Development.

Rijwan Uddin Ahammad1, Brian Spencer1, Robert A Rissman1

  • 1Alzheimer's Therapeutic Research Institute, Keck School of Medicine, University of Southern California, San Diego, CA, USA.

Alzheimer'S & Dementia : the Journal of the Alzheimer'S Association
|December 26, 2025
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Summary

A new treatment targeting alpha-synuclein (α-syn) in Dementia with Lewy bodies (DLB) showed promising results. Systemic delivery of ApoB11:ASO α-syn reduced α-syn aggregates, improved cognitive and motor functions, and enhanced neuronal survival in mice.

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Area of Science:

  • Neuroscience
  • Neurology
  • Pharmacology

Background:

  • Dementia with Lewy bodies (DLB) involves alpha-synuclein (α-syn) and Alzheimer's disease (AD) pathology, leading to neurodegeneration and cognitive decline.
  • Current treatments for DLB and related disorders (ADRD) are limited, with no disease-modifying therapies available.
  • α-syn oligomers and proto-fibrils are implicated in the neurodegenerative process of synucleinopathies.

Purpose of the Study:

  • To evaluate the safety and efficacy of systemically delivered ApoB11:2'-OMe ASO targeting α-syn in a mouse model of DLB.
  • To assess the pharmacokinetic, pharmacodynamic, and toxicological profile of the ASO treatment.
  • To determine the impact of the treatment on α-syn aggregation, neurodegeneration, and cognitive/motor deficits.

Main Methods:

  • Utilized 2'-OMe modified antisense oligonucleotides (ASOs) targeting α-syn, conjugated with ApoB11 for blood-brain barrier penetration.
  • Administered ApoB11:2'-OMe ASO peptides to DLB transgenic and non-transgenic mice.
  • Assessed treatment effects using qPCR, immunohistochemistry, Western blot, and behavioral tests.

Main Results:

  • ApoB11:2'-OMe ASO targeting α-syn was well-tolerated and safe in DLB mice, with successful brain delivery and no observed toxicity.
  • Significant reduction in α-syn aggregates was observed in the brains of treated DLB mice.
  • Improved spatial memory and motor performance were noted, accompanied by increased neuronal survival and reduced neurodegeneration.

Conclusions:

  • Systemically delivered ApoB11:ASO α-syn represents a safe, disease-modifying treatment strategy for DLB.
  • This approach shows potential as a future therapeutic for other synucleinopathies.
  • Targeting α-syn reduction offers a promising avenue for treating neurodegenerative diseases.