Respiratory Drug to be Evaluated in Parkinson's Disease

Ambroxol improves the function of a protein associated with Parkinson's disease development.

A clinical trial has been launched to determine whether an FDA approved drug for respiratory diseases can also treat Parkinson’s disease.

This trial is part of the Linked Clinical Trials (LCT) initiative under the collaboration of The Cure Parkinson’s Trust and Van Andel Research Institute (VARI).

"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 President and co-founder of The Cure Parkinson's Trust, Tom Isaacs. "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 researchers will analyze ambroxol, which is used to treat respiratory issues. In prior studies, it was found that the drug improves the function of a protein that is key in the cellular “trash removal” processes.

The data suggests that this impairment is associated with neurodegenerative disease onset and progression. Furthermore, gene mutations that encode this protein are one of the greatest risk factors for developing Parkinson’s disease.

"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," said principal investigator Anthony Schapira MD, DSc. "What is particularly interesting is the potential for ambroxol also to benefit Parkinson's patients without these genetic mutations."

Over the last 50 years, there has been scarce breakthroughs for treating Parkinson’s, with the exception of the standard treatment drug levodopa and the surgical option of deep brain stimulation.

These therapeutic options can improve quality of life but it does not slow or halt the progression of brain cell death.

"With this trial, we're moving beyond treating Parkinson's symptoms. We want to actually slow or stop disease progression," said the head of LCT's international scientific committee, Patrik Brundin, MD, PhD. "This drug -- ambroxol -- has performed exceptionally well in preclinical studies for Parkinson's and is already approved to treat other conditions."

LCT is also supporting clinical trials that study the effects of the cholesterol drug simvastatin and the diabetes drug exenatide as potential treatment options for Parkinson’s disease.

Future trials are also expected to focus on diabetes drugs and other medications that help remove the buildup of iron in the brain. Additionally, they will explore treatments that address mitochondrial function.