A landmark Phase 3 clinical trial has started in the UK to test whether a common cough medicine can be used to treat Parkinson’s disease. Early research shows that the drug, developed 50 years ago, penetrates the brain and helps remove toxic proteins known to cause Parkinson’s disease.
About ten years ago, researchers were investigating a new treatment for the condition known as Gaucher disease. A rare genetic disorder causes people to lack an enzyme called glucocerebrosidase (or GCase). Researchers who screened hundreds of already approved drugs found that a common ingredient in cough syrup called ambroxol increases the activity of GCase.
Following this discovery, Parkinson’s disease researchers took notice. Low levels of his GCase in the brain are thought to be involved in the pathology of Parkinson’s disease. This enzyme plays an important role in helping the brain clear out toxic proteins.
Up to 15% of Parkinson’s disease patients have specific genetic mutations that reduce GCase production, and many Parkinson’s disease patients without genetic mutations still exhibit abnormally low levels of GCase. If ambroxol increases her GCase activity, could it help treat Parkinson’s disease?
Preclinical studies were promising, and it quickly became plausible that the drug could help people with Parkinson’s disease. Is there enough of a drug to effectively cross the blood-brain barrier in humans, and can patients tolerate the doses necessary for the drug to have a therapeutic effect?
A Phase 2 clinical trial provides promising answers to these questions, finding that ambroxol is safely tolerated at doses high enough to enter the brain and raise GCase levels. . The study, published in 2020, also showed that ambroxol may reduce levels of the toxic protein alpha-synuclein and improve motor symptoms in patients with Parkinson’s disease.
All of this has paved the way for a pivotal Phase 3 trial to start in the UK this year. The trial will recruit 330 Parkinson’s disease patients and provide each subject with either ambroxol or placebo for his two years.
“This is the first time that a drug specifically directed against the genetic cause of Parkinson’s disease has reached this level of testing, and is the result of a decade of extensive and detailed research in the laboratory and a series of principled clinical trials. proof,” the principal investigator explained. Anthony Shapira on trial.
By 2025, it is hoped that the trial will provide some insight into whether this common cough medicine will help people with Parkinson’s disease. said it is working to fund such trials to rapidly introduce new treatments into the clinic.
“This trial is a major step forward in finding a new treatment for Parkinson’s disease. It will be one of the phase trials, Parkinson’s disease, worldwide.”
Source: UCL
