Parkinson’s disease is the second most common neurodegenerative disorder, affecting approximately 10 million people worldwide. There are 100,000 Canadians living with this disease and 5,000 new cases each year. Parkinson's disease is due to the death of a structure in the brain called the substantia nigra that producing dopamine. Decreased dopamine results in tremors, slowness of movement, rigidity, declined ability to concentrate. There are many current treatments for Parkisons Disease but none are focused on curing the disease. Why the sustantia nigra dies is unknown. Dr. Chris MacMaster, a Dalhousie University researcher is working to understand this tragic disease and find a cure.
The majority of cases of Parkinson’s disease are idiopathic (no clear cause), however, hereditary forms of the disease account for approximately 10% of all patients. The most common inherited form of Parkinson’s disease is due to mutations in a gene called LRRRK2. In addition to the known role of LRRK2 in inherited Parkinson’s Disease, approximately 30% of idiopathic Parkinson’s disease patients display an increase in LRRK2. A therapy that works through inhibition of LRRK2 activity will be effective for the inherited form of Parkinson’s disease and will likely prove effective for the 30% of idiopathic Parkinson’s disease patients that display increased LRRK2 activity.
Dr. Chris McMaster and his team have tested several small molecules/drugs that inhibit LRRK2 activity. More than 5,000 FDA-approved drugs and 100,000 synthesized novel small molecules were tested. Six compounds were effective in the inhabitation of LRRK2 activity. One compound is an off-patent drug that is known to be able to cross the blood-brain barrier, which may allow for its direct repurposing for the treatment of Parkinson’s disease. The goal is to quickly move this class of compounds through trials in Parkinson’s disease models. Soon after this compound would be developed for first in human trials of a new class of drug for the treatment of Parkinson’s disease.
Dalhousie Medical Research Foundation is raising funds to support this critical and life-saving research.