GRAND RAPIDS, Michigan (WOOD) – The VanAndel Institute will continue to work with Cure Parkinson’s disease 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 medicine 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 prioritized by the International Related Clinical Trials Program, which was created as part of the partnership between VAI and Cure Parkinson’s. The organization focuses on drugs that have already been proven safe and can be applied to other conditions.
“In the beginning, the (ILCT) was primarily evaluating all compounds that had previously received approval from the FDA or other regulatory body. And the purpose behind that is to facilitate rapid entry of these drugs into trials. clinics,” VAI assistant professor Michael Henderson told News 8. “If they’ve already been proven safe and have evidence that they could treat Parkinson’s disease, that could really speed up the process because that clinical trials can take many years.
Research into the potential use of ambroxol to treat Parkinson’s disease began in 2014. The first two trial phases confirmed that the drug would be safe for patients with Parkinson’s disease. The third phase will focus on its effectiveness.
“For a cough medicine, it just gets to the throat, right? But for brain medicine, you would need to reach the brain. And so one of the positive results from (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 modify the target gene, 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 disease behind Alzheimer’s disease and affects approximately 10 million people worldwide. According to National Institutes of Health, the brain disease causes involuntary or uncontrollable movements, including shaking and stiffness, and leads to difficulties with balance and coordination. It is a progressive disease, which means that the symptoms get worse over time.
Parkinson’s disease occurs when nerve cells in the basal ganglia, the part of the brain that controls movement, become impaired or die. The accumulation of these dead or damaged cells leads to the symptoms we now associate with Parkinson’s disease. However, ambroxol has been shown to increase the amount of glucocerebrosidase, commonly known as GCase, which is a protein that removes these waste cells from the brain.
By increasing the amount of GCase, the researchers believe it could slow the onset of serious symptoms.
“For Parkinson’s disease, we have symptomatic treatments. These are treatments that can improve the patient’s quality of life, but we don’t have anything that can slow disease progression,” Henderson said. “What we mean by slowing progression is that, as these are age-related diseases, people usually get them when they are older. So if you slow the progression even by about five years, it can prolong the person’s quality of life (beyond the point of impact of Parkinson’s disease).
Phase three will be conducted in a series of clinics in the UK. About 330 patients with Parkinson’s disease will be involved, with one group taking ambroxol and the other taking a placebo. Then these patients will be monitored for GCase levels and symptoms of Parkinson’s disease. Henderson expects to have phase three results in two to three years.
“It depends on how quickly the trial can recruit and look at those results,” he said.
Phase three clinical trials are expected to cost around $6.6 million. Cure Parkinson is expected to cover around 40% of the cost, and VAI and two other partners will each cover 20%.