MedicalResearch.com Interview with:
Patrik Brundin, M.D., Ph.D.
Director, Center for Neurodegenerative Science
Van Andel Research Institute
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: The investigational drug at the heart of our study, MSDC-0160, has been in development to treat type 2 diabetes by improving cellular metabolism.
In Parkinson’s, reductions in cellular metabolism slow down vital housekeeping processes that clear out toxic proteins that otherwise accumulate with age. If these proteins aren’t removed, they clump together, leading to the damage and cell death that causes Parkinson’s hallmark symptoms, such as rigidity and tremor. MSDC-0160 helps keep these housekeeping processes working, ultimately protecting the brain.
We demonstrated that MSDC-0160 has strong, reproducible, positive effects across multiple models of Parkinson’s disease—it rescued dopamine-producing cells, improved behavioral deficits in mouse models and reversed inflammation. Overall, we believe it is a strong candidate for repurposing as a potential treatment that actually may slow the disease’s progression, rather than only mitigating symptoms.
MedicalResearch.com: What should readers take away from your report?
Response: If MSDC-0160 demonstrates the same success in clinical trials as it did in laboratory studies, it could be a game-changer for millions of people with Parkinson’s. Currently, there are no therapies that actually slow the disease process, only treatments that mitigate symptoms. We are extremely hopeful that MSDC-0160 will change this, giving patients more years with better health.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: We are working to quickly move MSDC-0160 into rigorous clinical trials, which are absolutely critical for determining if the drug also has positive effects in humans. These efforts are underway in concert with our collaborators at Metabolic Solutions Development Company, which developed MSDC-0160, and The Cure Parkinson’s Trust, through its drug repurposing initiative Linked Clinical Trials.
We also hope to expand our studies to investigate the drug’s effect in diseases that have similar mechanisms to Parkinson’s, such as Lewy body dementia.
MedicalResearch.com: Is there anything else you would like to add?
Response: We are very hopeful and look forward to the results of the clinical trials, which will tell us if MSDC-0160 impedes Parkinson’s progression in humans. If so, this would be a huge step forward that could give millions of people an improved quality of life.
MSDC-0160 is one of several prototypes in a new class of insulin sensitizers that modulate the function of the mitochondrial pyruvate carrier (MPC). Unlike other drugs that target a cell surface receptor or a specific enzyme, these compounds work by regulating cellular metabolism. In addition to studies in Parkinson’s and diabetes, MSDC also is investigating MPC modulators in Alzheimer’s disease and NASH, a type of fatty liver disease.
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Anamitra Ghosh et al. Mitochondrial pyruvate carrier regulates autophagy, inflammation, and neurodegeneration in experimental models of Parkinson’s disease.
Science Translational Medicine, December 2016 DOI: 10.1126/scitranslmed.aag2210
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