VAI researchers believe cough medication may hold the key to slowing Parkinson’s disease

GRAND RAPIDS, Michigan (WOOD) — The VanAndel Institute will continue his work with Curing Parkinson’s to facilitate a third trial of a drug that could help slow the progression of the neurological disease.

The focus will be on a drug called ambroxol. The drug, which was invented in the 1960s, is used in cough medicines in many European and South American countries. However, it has not been approved for sale in the United States by the Food & Drug Administration.

Ambroxol is one of the drugs that has priority in the International Linked Clinical Trials Program, which was created as part of the collaboration between VAI and Cure Parkinson’s. The organization focuses on medicines that have already been proven to be safe and that can be used in other conditions.

“In the beginning, the (ILCT) primarily evaluated all compounds previously approved by the FDA or other regulatory agency. And the goal behind that is to make sure these drugs can get into clinical trials quickly,” VAI assistant professor Michael Henderson told News 8. speeding up the process because clinical trials can take many years.

Research into the potential use of ambroxol to treat Parkinson’s began in 2014. The first two trial phases confirmed that the drug would be safe for Parkinson’s patients. Phase three will focus on how effective it will be.

“For cough medicine you only have to get to the throat, right? But for brain medicine you would have to reach the brain. And so one of the positive results of the (earlier trials) was that they found that ambroxol could actually get to the brain,” Henderson explained. “(The trials also showed) that it was able to alter the target gene, which is called glucocerebrosidase. This is the protein that we think is involved in (slowing down) Parkinson’s disease.”

Parkinson’s disease is the second most common neurodegenerative disorder behind Alzheimer’s disease, affecting approximately 10 million people worldwide. According to the National Health Institutes, the brain disease causes inadvertent or uncontrollable movements, including trembling and stiffness, and causes problems with balance and coordination. It is a progressive disease, meaning that the symptoms worsen over time.

Parkinson’s disease occurs when nerve cells in the basal ganglia — the part of the brain that controls movement — become damaged or die. The buildup of those dead or damaged cells leads to the symptoms we now associate with Parkinson’s. However, Ambroxol has shown that it can increase levels of glucocerebrosidase, commonly called GCase, a protein that removes those waste cells from the brain.

By increasing the amount of GCase, researchers believe it may delay the onset of severe symptoms.

“For Parkinson’s disease, we have symptomatic treatments. These are treatments that can improve quality of life while the patient is alive, but we don’t have anything that could slow the progression of the disease,” Henderson said. “What we mean by slowing progression is that since these are age-related diseases, people usually get them when they are older. So if you slow progression, even by a matter of five years or so, that can extend the person’s quality of life (beyond the point of Parkinson’s impact).”

Phase three will be carried out in a range of clinics across the UK. It will involve about 330 patients with Parkinson’s disease, one group taking ambroxol and the other a placebo. Then those patients will be monitored for levels of GCase and Parkinson’s symptoms. Henderson expects to have phase three results in two to three years.

“It depends on how quickly the trial can recruit and assess those results,” he said.

The phase three clinical trials are expected to cost approximately $6.6 million. Cure Parkinson is expected to cover approximately 40% of the costs, and VAI and two other partners will each cover 20%.

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