Science Translational Medicine reports that a new drug originally developed for type 2 diabetes is being prepared for human clinical trials in hope of finding the world's first treatment to slow Parkinson's disease.

The drug was developed by Kalamazoo-based Metabolic Solutions Development Company and supported by research from Van Andel Research Institute's Center for Neurodegenerative Science.

"We hope this will be a watershed moment for millions of people living with Parkinson's disease," says Patrik Brundin, M.D., Ph.D., director of Van Andel Research Institute's Center for Neurodegenerative Science, chairman of The Cure Parkinson's Trust's Linked Clinical Trials Committee, and the study's senior author. "All of our research in Parkinson's models suggests this drug could potentially slow the disease's progression in people as well."

Until now, Parkinson's treatments have focused on symptom management. If successful in human trials, the drug, named MSDC-0160, would be the world's first therapy to treat the underlying disease and slow its progression. It may also reduce or delay the need for medications that can have debilitating side effects, says Brundin.

Parkinson's disease afflicts between 7-10 million people worldwide, including an estimated 1 million Americans. The numbers are expected to increase dramatically as the average human lifespan increases. There is currently no cure, and treatment has remained relatively unchanged since the introduction of levodopa in the 1960s.

Tom Isaacs, a co-founder of The Cure Parkinson's Trust who has lived with Parkinson's for 22 years, says MSDC-0160 represents one of the most promising treatments seen to date.

"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," says Isaacs. "We are working tirelessly to move this drug into human trials as quickly as possible in our pursuit of a cure."

MSDC-0160 was developed by Metabolic Solutions Development Company to treat type 2 diabetes. In 2012, Brundin recognized it as a candidate because of its mode of action, proven safety in people, local availability and the start-up company's interest in collaborating on drug repurposing initiatives. After four years of work, the effects of the drug in the laboratory exceeded Brundin's expectations.

The promise of MSDC-0160 stems from a recently revived revelation that Parkinson's may originate, at least partially, in the body's energy metabolism. The new drug appears to regulate mitochondrial function in brain cells and restore the cells' ability to convert basic nutrients into energy. This helps the cells' ability to handle potentially harmful proteins and leads to reduced inflammation and less nerve cell death.

While Brundin says he is eager to see MSDC-0160 launched into a clinical trial in Parkinson's disease, he's equally excited about the possibility of testing the drug in Lewy body dementia and other cognitive decline conditions, such as Alzheimer's disease.

"This is an immensely promising avenue for drug discovery," says Brundin. "Whatever the outcome of the upcoming trial for Parkinson's, we now have a new road to follow in search of better treatments that cut to the root of this and other insidious diseases."

Regulatory issues and funding still need to be arranged to begin the clinical trial which Brundin hopes can begin sometime in 2017.

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