Can a drug that was originally designed to treat diabetes become the world’s first medication to slow the progress of Parkinson’s disease?
Researchers are confident enough in the drug’s ability that they are organizing human clinical trials to begin next year.
The announcement came after the researchers’ findings were published today in the journal Science Translational Medicine.
“We hope this will be a watershed moment for millions of people living with Parkinson’s disease,” Dr. Patrik Brundin, director of Van Andel Institute’s Center for Neurodegenerative Science, chairman of The Cure Parkinson’s Trust’s Linked Clinical Trials committee, and the study’s senior author, said in a statement.
“The American Parkinson Disease Association (APDA) is always optimistic about studies that seek to slow the progression of the disease,” added Leslie A. Chambers, APDA president and chief executive officer, in an email to Healthline. “Well-designed randomized controlled clinical trials that assess new therapies are key in supporting this aspiration. We hope that a trial like this might offer promise for the future.”
Researchers said if the Parkinson’s human trials go well, the drug could someday be tested for possible treatment of other neurodegenerative diseases such as Alzheimer’s.
How the drug works
The drug, MSDC-0160, was designed by Michigan-based Metabolic Solutions Development Company to treat type 2 diabetes.
The drug showed the potential to regulate mitochondrial function in brain cells and restore the cells’ ability to convert basic nutrients into energy.
That enhances the cells’ ability to handle potentially harmful proteins, which leads to reduced inflammation and less nerve cell death.
Researchers said recently revived revelations that Parkinson’s may originate, at least partially, in the body’s energy metabolism led them to test MSDC-0160 on that disease.
After four years of positive results, the researchers said they are ready for human trials.
“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, said in a statement.
The trials are also the latest in drug repurposing, a move in the scientific community to use medications that are effective on more than one ailment.
Search for a Parkinson’s cure
Parkinson’s disease affects more than 10 million people worldwide, including 1 million people in the United States.
There’s no cure for the ailment. Treatments now focus on symptom management.
Researchers say first-line treatment for Parkinson’s has remained relatively unchanged since the introduction of levodopa in the 1960s.
If it works, MSDC-0160 would be the first therapy to treat the underlying disease. That could result in fewer falls for patients as well as less cognitive decline.
In addition, it could delay the implementation of other Parkinson’s medications that can have serious side effects.
Tom Isaacs, a co-founder of The Cure Parkinson’s Trust who has lived with Parkinson’s for 22 years, said in a statement that 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,” said Isaacs. “We are working tirelessly to move this drug into human trials as quickly as possible in our pursuit of a cure.”
The Cure Parkinson’s Trust and Van Andel 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.