Additionally, studies with fly models indicated that pyrvinium pamoate significantly improved locomotive abilities, reinforcing its therapeutic potential in neurodegenerative diseases.

Zhu's previous findings revealed that the shape of nuclear speckles influences their function, with more spherical shapes linked to poorer proteostasis.

Senior author Bokai Zhu, Ph.D., emphasizes that rejuvenating nuclear speckles represents a novel concept that could be the next frontier in neurodegenerative research.

New research from the University of Pittsburgh suggests that targeting nuclear speckles could revolutionize treatment for neurodegenerative diseases like Alzheimer's and Parkinson's, which are driven by the accumulation of misfolded proteins.

Nuclear speckles are cellular structures that play a crucial role in regulating protein production, folding, and degradation, essential for maintaining a balance known as proteostasis.

The FDA-approved drug pyrvinium pamoate operates uniquely by lowering the surface tension of nuclear speckles, enhancing their interaction with chromosomes and promoting the expression of genes crucial for proteostasis.

In a series of experiments, pyrvinium pamoate was identified as effective in improving nuclear speckle shape and function, significantly reducing pathological tau protein levels by approximately 70% in mouse neurons.

The drug not only reduced tau protein accumulation in mouse models with human tau but also improved motor function in fly models of tauopathy, showcasing its potential efficacy in treating these conditions.

In models of frontotemporal dementia, pyrvinium pamoate restored the shape of nuclear speckles and significantly lowered tau levels without causing cellular stress.

Zhu aims to advance this research into clinical trials to evaluate the effectiveness of pyrvinium pamoate in humans suffering from proteinopathies.

The research also indicates potential applications of pyrvinium pamoate for various protein-related disorders, including retinitis pigmentosa, by restoring normal protein function.

The study highlights the importance of nuclear speckle dysregulation in neurodegeneration and proposes further clinical trials to test the drug's efficacy in humans.