Experimental diabetes drug shows promise in Parkinson’s disease

The drug, MSDC-0160, offers a new approach to treating Parkinson’s disease, targeting the underlying disease rather than alleviating symptoms.

Parkinson’s disease in MRI brain scan

An experimental drug originally developed for type 2 diabetes has shown early promise as a treatment for Parkinson’s disease, say researchers who are now planning a clinical trial in humans.

Known as MSDC-0160, the drug differs from current treatments for Parkinson’s disease in that rather than simply alleviating symptoms, it is designed to target the underlying disease.

Study leader Patrik Brundin, director of the Van Andel Research Institute’s Center for Neurodegenerative Science, Michigan, hopes the study will be a “watershed moment” for Parkinson’s disease research.

“All of our research in Parkinson’s models suggests this drug could potentially slow the disease’s progression in people as well,” he says.

He adds that, if successful in human trials, MSDC-0160 would be the first therapy in Parkinson’s disease to treat the underlying disease and potentially slow its progression.

Several studies have shown potential similarities in metabolic changes at the molecular level in diabetes and Parkinson’s disease. This has led to an increased interest in the effects of thiazolidinediones – a group of insulin sensitising drugs – in patients with Parkinson’s disease, the US researchers report in Science Translational Medicine
(online, 7 December 2016).

In the latest study, the researchers tested MSDC-0160 — developed to act in a similar way to thiazolidinediones but with fewer side effects — in several animal and cell models of Parkinson’s disease.

The laboratory experiments suggested that the new drug, which targets a transport protein in mitochondria, regulates mitochondrial function in brain cells and restores the cells’ ability to convert basic nutrients into energy. This led to reduced levels of inflammation and less nerve cell death in several different Parkinson’s models tested.

In mice genetically engineered to mimic Parkinson’s disease, long-term use of MSDC-0160 was found to preserve motor function and reduce neuroinflammation.

Tom Isaacs, a co-founder of The Cure Parkinson’s Trust, a charity based in London, which part-funded the study, says: “Our scientific team has evaluated more than 120 potential treatments for Parkinson’s disease, and MSDC-0160 offers the genuine prospect of being a breakthrough that could make a significant and permanent impact on people’s lives in the near future.” He says they are working to move the drug into clinical trials as quickly as possible and hope to start in 2017.

Claire Bale, head of research communications and engagement at Parkinson’s UK, a charity based in London, says to date no treatment had been found that can either stop or slow down the advancement of Parkinson’s.

“Although it has so far only been tested in mice models, the MSDC-0160 drug shows significant promise; indicating improvements in motor function, maintaining dopamine production and protecting the cells lost in a number of models of the condition,” she says.

“While other diabetes drugs have shown promise in Parkinson’s, they can have serious side effects.

“Encouragingly, this new drug appears to have a better safety profile, which we hope will translate into good long-term safety in people with the condition.”

Bale adds that it is essential that clinical trials are geared towards people in the early stages of the disease where the protective properties of the drug are likely to have the greatest impact.


[1] Ghosh A, Tyson T, George S et al. Mitochondrial pyruvate carrier regulates autophagy, inflammation, and neurodegeneration in experimental models of Parkinson’s disease. Science Translational Medicine 2016;8:368ra174. doi: 10.1126/scitranslmed.aag2210

Last updated
The Pharmaceutical Journal, Experimental diabetes drug shows promise in Parkinson’s disease;Online:DOI:10.1211/PJ.2016.20202062

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