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Validation of VPS41, a protein involved in lysosomal trafficking, as a target for Parkinson disease therapy

Two years ago, in an MJFF-funded search for proteins that are neuroprotective in a simple worm model of PD, the most promising target to emerge was VPS41, believed to be involved in systems that breakdown abnormal proteins. Our goal is to determine whether this protein is protective in human cell lines and mice, and therefore may be a useful target for treatments for human Parkinson disease.
Project Description:
The work will be conducted by two groups of investigators. At the University of Alabama (UA), Drs. Guy and Kim Caldwell will expand on their earlier studies in worms to identify the specific components of VPS41 that are protective. At the University of Alabama at Birmingham (UAB), Dr. Standaert and his team will translate the findings in worms into human cell lines, knockout mice, and viruses for gene therapy. Together, they will seek to determine the components of VPS41 responsible for its protective effects, and whether the protection extends to models of PD in cell lines and the intact brains of rodents.
Relevance to Diagnosis/Treatment of Parkinson’s Disease:
This project is directed at developing a novel approach to neuroprotective treatments to Parkinson disease. If VPS41 is as effective in mammals as it is in worms, it would have a powerful ability to reduce or prevent the degeneration of Parkinson disease. This could lead to new treatments that work in a variety of ways, from drugs to enhance the functions of VPS41, which is normally found in the brain, to gene-therapy approaches for delivery of more VPS41.
Anticipated Outcome:
We expect to learn whether VPS41, discovered in a worm model, is effective in models based on human cell lines or the intact brain of rodents. If VPS41 is effective, it will represent a new target for therapeutic development. In addition, successful neuroprotection by VPS41 would support the further use of the worm model to identify other targets and treatments.


  • David G. Standaert, MD, PhD

    Birmingham, AL United States

  • Kim A. Caldwell, PhD

    Tuscaloosa, AL United States

  • Guy A. Caldwell, PhD

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