Researchers at Northwestern Medicine have tested a new strategy for treating Parkinson’s disease with positive results. The study suggests the new approach could serve as viable treatment option for several forms of Parkinson’s disease.
Parkinson’s disease affects approximately 10 million individuals worldwide and each year, 60,000 Americans are diagnosed with the disease. Patients with Parkinson’s disease experience progressive damage to nerve cells in a region of the brain called the substantia nigra. The disease causes damage to neurons responsible for producing the neurotransmitter dopamine, which is required for movement. The reduction in dopamine levels results in involuntary shaking, stiff and inflexible muscles, and slow movement. While the disease is not fatal, patients can suffer complications. There is no cure, but with treatment patients can have a near-normal life expectancy.
One of the most common causes is a mutation in the gene GBA1, which codes for an enzyme called glucocerebrosidase (GCase). The mutation results in defective GCase enzymes, which leads to the accumulation of toxic proteins that kill dopamine-producing neurons.
Several approaches for treatment have been tested, such as targeting the mutated proteins and enzymes and limiting their harmful effects, but these strategies only work on limited types of Parkinson’s disease. Instead, the researchers tried amplifying healthy enzymes using a technique that had been shown to be effective in mouse models and in vitro studies in human brain cells on both genetic and idiopathic forms of the disease.
They activated wild-type GCase and believe this could be an effective strategy for treating several different forms of Parkinson’s disease. “Activating wild-type GCase may be more relevant for multiple forms of PD that exhibit reduced activity of wild-type GCase,” said Dr. Dimitri Krainc, chair of neurology and director of the Center for Neurogenetics at Northwestern University Feinberg School of Medicine.
The study is detailed in the paper – A modulator of wild-type glucocerebrosidase improves pathogenic phenotypes in dopaminergic neuronal models of Parkinson’s disease – which was recently published in the journal Science Translational Medicine. DOI: 10.1126/scitranslmed.aau6870