New Parkinson’s Clinical Trial Will Evaluate Drug Already Approved for Respiratory Conditions

A new clinical trial for Parkinson’s disease will focus on a drug already approved for the treatment of respiratory conditions that has revealed neuroprotective effects in preclinical studies.

The clinical trial is part of the Linked Clinical Trials (LCT) initiative, a program that identifies potential new treatments to slow, stop, or reverse Parkinson’s disease by repurposing drugs that have been approved to treat other conditions. LCT is spearheaded by The Cure Parkinson’s Trust in England in partnership with Van Andel Research Institute (VARI), based in Grand Rapids, Michigan.

“With this trial, we’re moving beyond treating Parkinson’s symptoms. We want to actually slow or stop disease progression,” Patrik Brundin, M.D., Ph.D., head of LCT’s international scientific committee and director of VARI’s Center for Neurodegenerative Science, said in a news release. “This drug — ambroxol — has performed exceptionally well in preclinical studies for Parkinson’s and is already approved to treat other conditions.”

The LCT scientific committee prioritizes new treatments once a year to identify the most promising drugs to fast-track into clinical trials, in the hope of not only proving efficacy but also shortening the time it takes for a potential treatment to move from the laboratory to clinical trials and, ultimately, to the patient. By selecting drugs that have already passed rigorous safety and toxicology trials, this approach can reduce the cost of bringing new Parkinson’s disease treatments to fruition.

“Linked Clinical Trials has real momentum and provides us with the prospect of having new, potentially breakthrough treatments being accelerated into the clinic within a relatively short period,” said Tom Isaacs, president and co-founder of The Cure Parkinson’s Trust. “This new trial provides real hope that we are on the cusp of something truly life-altering — new therapies that show promise to improve the quality of life for the 7 to 10 million people worldwide who have Parkinson’s.”

The clinical trial will be based at Royal Free Hospital in London and focuses on ambroxol, a secretolytic agent used in the treatment of respiratory diseases associated with viscid or excessive mucus. It will be headed by Anthony Schapira, M.D, D.Sc., whose previous work showed that ambroxol is able to improve the function of a protein implicated in the “trash removal” of cellular processes. Evidence from recent studies indicates that impairments of these cellular processes are associated with the onset and progression of neurodegenerative diseases. Moreover, the greatest genetic risk factor for developing Parkinson’s has been attributed to genetic mutations in the gene that encodes for this specific protein.

“Our preclinical work suggests ambroxol may be an effective Parkinson’s treatment, thanks to its ability to correct a dysfunctional protein that is prevalent in people carrying a genetic mutation associated with inherited Parkinson’s cases,” Schapira said. “What is particularly interesting is the potential for ambroxol also to benefit Parkinson’s patients without these genetic mutations.”

Levodopa is the current gold standard drug treatment for patients with Parkinson’s disease, who also have the option to undergo deep brain stimulation. These treatments have been found to improve patients’ quality of life, but they do not slow or stop gradual death of brain cells.

In addition to the clinical trial evaluating ambroxol’s potential, LCT is also supporting clinical trials investigating simvastatin, a cholesterol-lowering drug, and exenatide, a diabetes drug, as potentially treatments for patients with Parkinson’s disease.

Other clinical trials focusing on other diabetes treatments, drugs to remove iron build-up in the brain, and treatments to address mitochondrial function, are expected to launch in the coming months.