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鈥淲e hope this will be a watershed moment for millions of people living with Parkinson鈥檚 disease,鈥� says , director of Center for Neurodegenerative Science, chairman of The Cure Parkinson鈥檚 Trust鈥檚 , and the study鈥檚 senior author. 鈥淎ll of our research in Parkinson鈥檚 models suggests this drug could potentially slow the disease鈥檚 progression in people as well.鈥�

Until now, Parkinson鈥檚 treatments have focused on symptom management. If successful in human trials, would be the world鈥檚 first therapy to treat the underlying disease and slow its progression鈥攑otentially 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 Brundin.

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

, a co-founder of who has lived with Parkinson鈥檚 for 22 years, says MSDC-0160 represents one of the most promising treatment the Trust鈥檚 international consortium has seen to date.

鈥淥ur scientific team has evaluated more than 120 potential treatments for Parkinson鈥檚 disease, and MSDC-0160 offers the genuine prospect of being a breakthrough that could make a significant and permanent impact on people鈥檚 lives in the near future,鈥� says Isaacs. 鈥淲e are working tirelessly to move this drug into human trials as quickly as possible in our pursuit of a cure.鈥�

MSDC-0160 was developed by Kalamazoo, Michigan-based (MSDC) to treat type 2 diabetes. In 2012, Brundin recognized it as an exciting drug candidate because of its mode of action, proven safety in people, local availability and the start-up company鈥檚 interest in collaborating on initiatives. After four years of work, the effects of the drug in the laboratory exceeded Brundin鈥檚 expectations.

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

鈥淧arkinson鈥檚 disease and diabetes may have vastly different symptoms with unrelated patient outcomes; however, we鈥檙e discovering they share many underlying mechanisms at the molecular level and respond similarly to a new class of insulin sensitizers like MSDC-0160,鈥� says Jerry Colca, Ph.D., co-founder, president and chief scientific officer of MSDC.

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

鈥淭his is an immensely promising avenue for drug discovery,鈥� says Brundin. 鈥淲hatever the outcome of the upcoming trial for Parkinson鈥檚, we now have a new road to follow in search of better treatments that cut to the root of this and other insidious diseases.鈥漈he Cure Parkinson鈥檚 Trust and Van Andel Research Institute are currently working with MSDC to address regulatory issues and obtain funding to organize the clinical trial, which Brundin hopes can begin sometime in 2017.

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

The paper鈥檚 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.

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About Parkinson鈥檚 diseaseAlthough Parkinson鈥檚 symptoms vary widely among patients, they typically progress from a constellation of vague problems, including loss of smell, constipation, poor sleep, mood fluctuations, and changing blood pressures, to the disease鈥檚 hallmarks, such as tremors, affected speech and facial expressions, stooping, poor motor function, and memory loss. The gold standard treatment, levodopa, supplements the neurotransmitter dopamine, a chemical in the brain that is critical for motor function and one that decreases as the disease progresses. Though often effective early in Parkinson鈥檚 progression, levodopa鈥檚 effectiveness may decline and the need to endure increasingly heavy doses comes with debilitating side effects like confusion, anxiety, hallucinations, uncontrollable movements and tremors, and incontinence.

About MSDC-0160 is a novel mTOT modulator being developed to treat neurodegenerative diseases such as Parkinson鈥檚 and Alzheimer鈥檚 disease. It is a once-a-day, oral insulin sensitizer that works through a new drug target located in the inner mitochondrial membrane, called mTOT. The mTOT mechanism ties mitochondrial metabolism to aspects of the pathology associated with insulin resistance and certain neurodegenerative diseases.

MSDC-0160 has been shown to safe, well-tolerated, and efficacious in a 90-day, randomized, double-blind, comparator- and placebo-controlled, multi-dose Phase 2b clinical study in 258 patients with type 2 diabetes (; and in a Phase 2a study conducted by researchers at in Chicago in patients diagnosed with dementia due to Alzheimer鈥檚 disease, wherein this new therapeutic agent prevented the decline in a measure of brain glucose utilization in patients with mild to moderate Alzheimer鈥檚 disease ().