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Experimental Drug Opens New Route to Slowing Parkinsonís Progression

A new study in laboratory animals finds that an experimental drug developed for diabetes may hold promise for slowing the progression of Parkinson’s disease (PD). Because the drug has already been tested for safety, clinical trials for its potential to treat Parkinson’s could begin within a year. The research appears in the December 7 edition of Science Translational Medicine.

Scientists have observed several shared features between Parkinson’s and diabetes, such as abnormalities in cell metabolism and links to inflammation. Because of these shared characteristics, scientists have tested several drugs for Type II diabetes, including pioglitazone and rosiglitazone, to see they might also have an effect on Parkinson’s. To date, the results have not been promising. However, as a new generation of diabetes drugs has been developed – drugs that target a newly-discovered protein called MPC (or mitochondrial pyruvate carrier) – this has presented new options.

Researchers at the Van Andel Institute in Grand Rapids, MI, led by Patrik Brundin, M.D., Ph.D., conducted studies to test one of these newer experimental drugs, called MSDC-0160, in PD. The drug reduces the activity of the MPC protein. They conducted studies in three models: human dopamine neurons (the type affected by PD) grown in the lab, roundworms (C. elegans) engineered to have PD-like levels of alpha-synuclein protein, and two different mouse models of PD in which the animals display movement difficulties similar to those in PD.


  • In two of the models - dopamine neurons in the lab and roundworms - pre-treatment with MSDC-0160 provided protection from exposure to the toxin MPTP, known to damage dopamine neurons.
  • In studies with mice, the researchers found that MSDC-0160 given by mouth was able to reach the brain.
  • In mice exposed to MPTP, treatment with the drug lessened both movement difficulties and damage to dopamine neurons.
  • In mice with a PD-like loss of dopamine neurons lessened, the drug movement symptoms, increased survival of dopamine neurons, helped maintain brain levels of dopamine and reduced inflammation.

What Does It Mean?

Current therapies for PD can help symptoms, but there is an urgent need for treatments that target the underlying disease and slow its progression. In this study, researchers demonstrated that an experimental drug, MSDC-0160, protected dopamine neurons and reduced inflammation in animal models of PD.

More research is needed to understand precisely how the drug works. For example, its effects were not perfect — even with treatment, some dopamine neurons still died and the animals did not regain completely normal activity. Nevertheless, scientists believe MSDC-0160 may improve the function of a cell’s metabolism and restart the cell’s ability to recycle damaged proteins and other cell components – both known to be affected by Parkinson’s.

Early testing of MSDC-0160 for treating diabetes and Alzheimer’s disease has already shown its potential safety in humans. MSDC-0160 appears to have a fewer side-effects than related diabetes drugs, like pioglitazone. Therefore, the drug could proceed quickly to clinical trials to test whether it can impact the progression of PD. The researchers suggest that this new class of drugs that target the newly identified MPC, like MSDC-0160 studied here, may ultimately benefit other neurodegenerative diseases as well, including dementia with Lewy bodies.

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Do you have additional questions about current medications for Parkinson's disease or potential new medications in the PD pipeline? We invite readers to learn more by accessing PDF's free resources below, or by contacting our National HelpLine at (800) 457-6676 or

Reference: Ghosh, A., Tyson, T., George, S., Hildebrandt, E. N., Steiner, J. A., Madaj, Z., et al. (2016). Mitochondrial pyruvate carrier regulates autophagy, inflammation, and neurodegeneration in experimental models of Parkinson's disease. Science Translational Medicine, 8(368), 368ra174.

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Source Date: Dec 15 2016