GRAND RAPIDS, Mich. (WOOD) — The Van Andel Institute will continue its work with Cure 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 medications 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 the drugs prioritized by the International Linked 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 proven to be safe and can be applied to other ailments.
“At the beginning the (ILCT) evaluated mostly all compounds that had previously had some FDA or other regulatory agency approval. And the purpose behind that is to facilitate these drugs getting into clinical trials quickly,” VAI assistant professor Michael Henderson told News 8. “If they’ve already been proven safe and they have evidence that they could treat Parkinson’s disease, this could really speed up the process because clinical trials can take many years.”
Research into ambroxol’s potential use to treat Parkinson’s started in 2014. The first two trial phases confirmed that the drug would be safe for Parkinson’s patients to take. Phase three will focus on how effective it will be.
“For cough medicine, you only have to reach the throat, right? But for brain medicine, you would need to reach the brain. And so, one of the positive outcomes from the (earlier trials) were that they found that ambroxol could actually reach the brain,” Henderson explained. “(The trials also showed) it was able to modify 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 and impacts around 10 million people worldwide. According to the National Institutes of Health, the brain disease causes unintended or uncontrollable movements, including shaking and stiffness and causes difficulty with balance and coordination. It’s a progressive disease, which means symptoms worsen over time.
Parkinson’s disease occurs when nerve cells in the basal ganglia — the section of the brain that controls movement — become impaired or die. The buildup of those dead or damaged cells lead to the symptoms we now associate with Parkinson’s. However, ambroxol has shown that it can increase the amount of glucocerebrosidase, commonly referred to as GCase, which is a protein that removes those waste cells from the brain.
By boosting the amount of GCase, researchers believe it could slow down the onset of severe symptoms.
“For Parkinson’s Disease, we have symptomatic treatments. These are treatments that can improve the quality of life while the patient is living, but we don’t have anything that would 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’re older. So, if you slow progression, even by a matter of five years or so, that may extend the person’s quality of life (past the point of Parkinson’s impact).”
Phase three will be conducted at a series of clinics in the United Kingdom. Approximately 330 Parkinson’s disease patients will be involved, one group taking ambroxol and the other taking a placebo. Then, those patients will be monitored for levels of GCase and Parkinson’s symptoms. Henderson expects to have the phase three results in two to three years.
“It depends on how quickly the trial can recruit and review those results,” he said.
The phase three clinical trials are expected to cost approximately $6.6 million dollars. Cure Parkinson’s is expected to cover around 40% of the cost, and VAI and two other partners will each cover 20%.