GRAND RAPIDS, Mich. (Dec. 7, 2016)—A new investigational drug originally developed for type 2 diabetes is being readied for human clinical trials in search of the world’s first treatment to impede the progression of Parkinson’s disease (PD) following publication of research findings today in the journal Science Translational Medicine.

We hope this will be a watershed moment for millions of people living with Parkinson’s disease. All of our research in Parkinson’s models suggests this drug could potentially slow the disease’s progression in people as well.

- Patrik Brundin, M.D., Ph.D., director Van Andel Research Institute’s Center for Neurodegenerative Science, chairman Linked Clinical Trials Committee, and the study’s senior author.

"Until now, Parkinson’s treatments have focused on symptom management. If successful in human trials, MSDC-0160 would be the world’s first therapy to treat the underlying disease and slow its progression—potentially improving quality of life and preventing the occurrence of falls and cognitive decline. It may also reduce or delay the need for medications that can have debilitating side effects," says Professor Brundin.

Tom Isaacs, 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 the Trust’s international consortium has 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 Tom. “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 Michigan-based company, Metabolic Solutions Development Company (MSDC), in Kalamazoo, US  to treat type 2 diabetes. In 2012, Professor Patrik Brundin recognised it as an exciting drug 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 Professor Brundin’s expectations.

The novelty 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. Consequently, the cells’ ability to handle potentially harmful proteins (alpha-synuclein) is normalised, which leads to reduced inflammation and less nerve cell death.

Parkinson’s disease and diabetes may have vastly different symptoms with unrelated patient outcomes; however, we’re discovering they share many underlying mechanisms at the molecular level and respond similarly to a new class of insulin sensitizers like MSDC-0160.

- Jerry Colca, Ph.D., co-founder, president and chief scientific officer of MSDC.

The Cure Parkinson’s Trust and Van Andel Research Institute are currently working with MSDC to address regulatory issues and obtain funding to organise the clinical trial, which Professor Brundin hopes can begin sometime in 2017.

Funding for the research was provided by Van Andel Research Institute, The Cure Parkinson’s Trust, the Campbell Foundation, and the Spica Foundation.

The paper’s authors include Anamitra Ghosh, Trevor Tyson, Sonia George, Erin N. Hildebrandt, Jennifer A. Steiner, Zachary Madaj, Emily Schulz, Emily Machiela, Martha L. Escobar Galvis, Jeremy M. Van Raamsdonk and Patrik Brundin, all of Van Andel Research Institute; William G. McDonald and Jerry R. Colca, both of Metabolic Solutions Development Company; and Jeffrey H. Kordower, of Rush University Medical Center and Van Andel Research Institute.

Research Article Source: Mitochondrial pyruvate carrier regulates autophagy, inflammation, and neurodegeneration in experimental models of Parkinson’s disease - Science Translational Medicine 7th December 2016