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April 3, 2026Annals of Neurology1 citations

Combination Disease‐Modifying Therapy for Neurodegenerative Diseases Using Repurposed Drugs

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ASAlexander ShtilbansALAnthony E. Lang

Key Points

  • To evaluate the effectiveness of repurposed drugs on neurodegenerative diseases and propose a combinatory therapy strategy.
  • Reviewed existing literature on drug effects in preclinical models and humans.
  • Examined various drug classes including GLP-1 RAs and c-Abl TKIs.
  • Discussed the concept of combinatory therapy targeting neurodegeneration processes.
  • Identified positive effects of multiple drug classes in neurodegenerative disease models.
  • Demonstrated potential for combination therapies to enhance neuroprotection.
  • Outlined ideal strategies for designing effective drug combinations.

Abstract

We review the positive effects of several existing drugs from different classes, such as chemical chaperones, glucagon‐like peptide‐1 receptor agonists (GLP‐1 RAs), iron chelators, and cluster‐Abelson tyrosine kinase inhibitors (c‐Abl TKIs), in preclinical disease models and in available published human data following use of these drugs in individuals with common neurodegenerative diseases (NDs), including Parkinson's disease (PD), Alzheimer's disease (AD), amyotrophic lateral sclerosis (ALS), and Huntington's disease (HD). A concept of combinatory neuroprotective therapy using a drug‐repurposing approach is then discussed. Finally, we propose a strategy to design an ideal combination of drugs able to address multiple pathogenic processes involved in neurodegeneration to achieve clinically meaningful results. ANN NEUROL 2026 ANN NEUROL 2026

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Cite This Study

Shtilbans et al. (2026) studied this question.

synapsesocial.com/papers/69cf5eee5a333a821460da42https://doi.org/10.1002/ana.78190
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